Demographic and Comorbidity Characteristics of Newly Diagnosed Multiple Myeloma Patients in the United States: A Real World Data Analysis

Abstract Background Multiple myeloma (MM) remains an incurable plasma cell neoplasm with >20,000 new cases diagnosed annually in the United States (US). The presentation of MM is diverse and the management is guided by disease- and patient- related factors. Real world evidence regarding disease presentation is sparse for newly diagnosed MM (NDMM) patients. The goal of this study was to describe the baseline demographic and clinical characteristics of NDMM patients by linking multiple large claims databases in the US. Methods A retrospective cohort study was conducted by linking the records between 01/01/2006-12/31/2013 from three longitudinal IMS databases (Medical Claims, Pharmacy Prescriptions, and Hospital Charge Master), to reflect a complete treatment continuum for patients in all ages and by all payer types across healthcare settings. Included patients met the following criteria: ≥2 claims with a diagnosis of MM (ICD-9-CM 203.0x) at least 30 days apart between 01/01/2007-12/31/2012 (first MM diagnosis date = index date); provider stability for at least 6 months before and 3 months after index date; aged 18+ as of index date. NDMM patients were identified as those with no MM diagnosis in the 6 months pre-index. Patient baseline characteristics were assessed using data at index for age, gender, geographic region and payer type; 6-month pre-index plus index day data for Charlson Comorbidity Index (CCI); 6-month pre-index data for 32 comorbidities (e.g., cardiac arrhythmia, cerebrovascular disease, chronic kidney disease, diabetes, hypertension, among others); and 5 comorbidity categories (cardiovascular, renal, metabolic, skeletal-related events, and other). Autologous stem cell transplant (ASCT) status was also assessed based on the available follow-up data after initial diagnosis. Patient baseline characteristics were compared between the patients with versus without ASCT after MM diagnosis. Chi-square tests for categorical variables and t-tests for continuous variables (alpha=0.05) were conducted. Results The study cohort of 8,239 NDMM patients had a mean (SD) age of 66.2 (11.3) years at diagnosis, with 56.7% aged 65+; 51.8% were female; and the majority were with commercial (43.3%) or Medicare (42.8%) as payer. Patients with post-index ASCT (n=1,429; 17.3%) were distinctly different from those without (n=6,810; 82.7%): they were younger with a mean (SD) age of 58.1 (8.7) vs. 67.9 (11.1) years, fewer aged 65+, 24.1% vs. 63.5%; fewer female, 47.6% vs. 52.6% (all p<0.001); and the majority (67.5%) of patients who later had ASCT were covered by commercial plans, compared to Medicare as the biggest payer (48.2%) for those who did not have post-index ASCT. NDMM patients had an overall mean (SD) CCI score of 3.1 (1.6). Patients who later received ASCT were healthier than those who did not, with mean (SD) CCI scores of 2.7 (1.4) vs. 3.2 (1.6) (p<0.001). Close to half (47.9%) of all NDMM patients had >1 type of comorbidities at baseline. Figure 1 depicts the baseline comorbidity categories among all NDMM patients as well as the two subsets (with versus without post-index ASCT). Comorbidities were prevalent even before MM diagnosis: 43.9% of patients presented with metabolic comorbidities, 21.4% with cardiovascular diseases, 11.5% with renal conditions, 5.9% with skeletal-related events, and 45.0% with other comorbidities. The patient subset with post-index ASCT had lower comorbidity occurrences compared to those without: 10.5% vs. 23.6% in cardiovascular diseases, 5.9% vs. 12.7% in renal conditions, 32.7% vs. 46.3% in metabolic diseases (all p<0.001), consistent with literature showing that ASCT candidates tend to be younger and have less severe comorbidities. Conclusions This study provided baseline demographic and comorbidity profiles of NDMM patients and showed that the NDMM patient population presented with various comorbidities prior to the first MM diagnosis, suggesting the needs for efficacious, tolerable and safe treatment options for a heterogeneous patient population with complex disease profiles. As expected, patients who had ASCT after initial diagnosis were younger and healthier than those who did not have ASCT. The study findings portray a clearer picture of NDMM patients in the US, and provide clinicians with valuable real world evidence to identify appropriate treatment strategies for these patients. Figure 1 Figure 1. Disclosures Chen: Onyx Pharmaceuticals: Research Funding. De:Onyx Pharmaceuticals: Research Funding. Cong:Onyz Pharmaceuticals: Employment. Aggarwal:Onyx Pharmaceuticals: Employment. Wade:Onyx Pharmaceuticals: Research Funding.

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Real World Evidence in Relapsed/Refractory Classical Hodgkin Lymphoma Patients Who Are Ineligible for Stem Cell Transplant in the United States (US)

Blood ◽

10.1182/blood-2018-99-113682 ◽

2018 ◽

Vol 132 (Supplement 1) ◽

pp. 2268-2268

Author(s):

Joseph Feliciano ◽

Nate Way ◽

Gerald Engley ◽

Nilanjan Ghosh

Keyword(s):

Real World ◽

Systemic Therapy ◽

Research Funding ◽

Patient Characteristics ◽

The United States ◽

Treatment Patterns ◽

Cell Transplant ◽

Real World Evidence ◽

The Us ◽

Line Of Therapy

Abstract Introduction: Treatment of relapsed or refractory classical Hodgkin lymphoma (R/R cHL) in patients considered ineligible for stem cell transplant (SCT) in the United States (US) has evolved since 2011. It is important to understand the current treatment landscape and the outcomes associated with current standards of care as new treatment options have been introduced. This analysis provides recent real-world evidence on patient characteristics, treatment patterns, and outcomes in R/R cHL patients in the US who are initially considered ineligible for SCT and are treated with or without brentuximab vedotin (BV). Methods: Hematologists and oncologists (N=205) from the US retrospectively identified patients diagnosed with R/R cHL who received at least two lines of therapy and received their most recent line of therapy between January 2014 and May 2018. The physicians were responsible for abstracting data and completing response forms for variables of interest. The current analysis focused on patients who were considered ineligible for SCT by their physician: descriptive statistics on patient demographics/clinical characteristics, treatment patterns, and outcomes by line of therapy; bivariate analyses (chi-square) comparing treatment modalities by line of therapy. Results: Physicians retrospectively identified 297 patients that they considered ineligible for SCT. Mean (SD) age at initial cHL diagnosis was 53.0 (18.5), most patients were male (69.4%) and Caucasian (61.3%). The most common cHL subtype at diagnosis was nodular sclerosis HL (40.4%), and patients had either Stage I/II (45.8%) or Stage III/IV (54.2%) cHL at initial diagnosis. Median follow-up time for the cohort included here was 15.96 months from initiation of 1L treatment. The majority of the cohort (N = 297) received systemic therapy alone (84.5%) compared to those who received systemic therapy in combination with radiation therapy (RT) (15.5%) in 1L. 1L systemic regimens included regimens that contained ABVD alone or ABVD in combination with other regimens (69.4%). Of those who used ABVD alone or in combination with another regimen (N = 206), 24.8% used a PET adapted approach and deescalated to AVD (N = 51) and 11% escalated to be BEACOPP (N = 18). Other systemic regimens included AVD (10.1%), BEACOPP (7.4%) and ICE (5.7 %). The majority of patients achieved a complete response (CR) or partial remission (PR) after 1L therapy (41.4%, 38% respectively) while 34.1% (N = 61) failed to achieve remission or progressed while on therapy. The most common systemic regimens in 2L (N = 293) were BV monotherapy or in combination with bendamustine (34.6%), salvage regimens [including ICE, DHAP, ESHAP or gemcitabine based combinations] (33%), re-challenge with a previous 1L regimen (19.5%), and PD-1 inhibitors (10.8%). Very few patients received systemic therapy in combination with RT (6.7%) in 2L.The most common systemic regimens used in 3L (N = 21) for the selected cohort of patients not eligible for SCT were BV monotherapy (28.6%) and PD-1 inhibitors (33.3%). Median (range) number of cycles in 2L and 3L was four (1-18) and two (1-14), respectively. Treatment outcomes were variable for patients in 2L and 3L. In 2L, 27.6% achieved a CR, 25.6% achieved a PR, while 24.2% and 15.8% were refractory or progressed on treatment. There were no CRs reported in 3L (N = 21). 26 patients died in 2L and 3L combined. Conclusion/Summary: Given the rapid evolution of therapies used to treat R/R cHL, these findings fill a crucial data gap in real-world evidence on patient characteristics, treatment patterns, and outcomes of patients deemed SCT ineligible in the US. Disclosures Feliciano: Seattle Genetics: Employment. Way:Kantar Health: Employment; Seattle Genetics: Research Funding. Engley:Seattle Genetics: Employment. Ghosh:Juno: Consultancy, Research Funding; SGN: Consultancy, Research Funding, Speakers Bureau; PCYC: Consultancy, Research Funding, Speakers Bureau; Celgene: Consultancy; Spectrum: Consultancy; Gilead: Consultancy, Speakers Bureau; Pharmacyclics, an Abbvie Company: Consultancy, Research Funding, Speakers Bureau; Genentech: Research Funding; F. Hoffman-La Roche Ltd: Research Funding; Abbvie: Consultancy, Speakers Bureau; Forty seven Inc: Research Funding; TG Therapeutics: Honoraria, Research Funding.

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Baseline Characteristics of CML Patients Accross Europe - Comparing Real-World Patients with Patient Collectives Included in Clinical Trials

Blood ◽

10.1182/blood.v124.21.3160.3160 ◽

2014 ◽

Vol 124 (21) ◽

pp. 3160-3160 ◽

Cited By ~ 1

Author(s):

Michele Baccarani ◽

Verena Sophia Hoffmann ◽

Gianantonio Rosti ◽

Fausto Castagnetti ◽

Susanne Saussele ◽

...

Keyword(s):

Real World ◽

Clinical Studies ◽

Research Funding ◽

Chronic Phase ◽

Population Based ◽

Costal Margin ◽

Spleen Size ◽

Newly Diagnosed ◽

Baseline Characteristics ◽

Population Based Registry

Abstract Introduction: Most of the knowledge about treatments and outcome of CML patients originates from clinical studies. To get new and unbiased insights in the epidemiology, treatment and outcome of CML, the EUTOS population-based registry of newly diagnosed CML patients was established, - as part of the European Treatment and Outcome Study (EUTOS) for CML. The aim was to collect the data of all adults with newly diagnosed CML, irrespective of treatment and of enrolment in studies. Patients and Methods: The EUTOS population-based registry collected data of newly diagnosed CML patients, 18 years or older, over a specified period of time from 2008 till 2012 living in defined regions. The data were collected by 22 study groups in 20 European countries. Data were gathered via a web-based CRF-system. For comparison we used the already published data from five Company-sponsored registration studies IRIS (O’Brien et.all, NEJM, 2003), TOPS (Cortes et al, JCO, 2009) ENESTnd (Saglio et al, NEJM, 2010), DASISION (Kantarjian et al, NEJM, 2010) and BELA (Cortes et al, JCO, 2012), from three Investigator-sponsored studies GIMEMA (Castagnetti et al, JCO, 2010 and Gugliotta et al, Blood, 2011), French SPIRIT (Preudhomme et al, NEJM, 2010) and German CML IV (Hehlmann et al, JCO, 2011) and from two single referral centers HAMMERSMITH (De Lavallade et al, JCO, 2008) and MDA (Jain et al, Blood, 2013). Results: Till 15.05.2014 2978 patients were registered in the EUTOS Population-based registry. 94.3% of the patients were diagnosed in chronic phase (CP), 3.6% in accelerated phase (AP), and 2.2% in blastic phase (BP). For the calculation of the prognostic scores 361 patients had to be excluded because they were pretreated. For the comparison we used 2350 patients in Chronic Phase with laboratory values before any treatment. 54% of the patients in the EUTOS Population-based registry were male, less than in all studies (56.6 - 60.6%). The median age at diagnosis was 56 years, higher than in all studies (46 - 55). In EUTOS the proportion of patients more than 60 years and more than 65 years old was 40.4 % and 21.9 % respectively. Similar data were rarely reported in all other studies. Median value of the spleen size below costal margin was 0. 46.1% of the patients had a palpable spleen and 15.2% had a spleen size ≥ 10 (spleen size is always reported in cm under costal margin in this abstract). The % of palpable spleen is only reported by IRIS, 25.0% and by the FRENCH Spirit group, 49.8%. The median spleen is only reported by GIMEMA, 2.0. Spleen size ≥ 10 is reported by IRIS, 6.0%, ENESTnd, 12.4% and HAMMERSMITH 25.5%. While the median values for Platelets and Hemoglobin show no big differences, the median WBC in EUTOS is 83.9 x109/l and in the Company-sponsored registration studies: IRIS 18-20 x109/l , in ENESTnd 23-26 x109/l, in DASISION 23-25 x109/l , and in BELA 22-23 x109/l, in the Investigator-sponsored studies: GIMEMA 55 x109/l , in the FRENCH SPIRIT 83-104 x109/l , in the GERMAN CML IV 75-91 x109/l , and in the single referral center study HAMMERSMITH 140 x109/l, clearly indicating that in company-sponsored, registration studies, the reported values of the WBC were not recorded prior to any treatment. The median values for Blasts, Basophils and Eosinophils show also not so big differences. The % of Sokal low risk patients is in EUTOS with 34.5% lower than in all studies (35.2 - 60%) with the exception of HAMMERSMITH 28.9%. Discussion: The EUTOS Population-based registry provides the first European wide real-world series of patients with newly diagnosed Ph+, BCR-ABL+ CML. The age and sex distribution and some baseline characteristics such as Sokal Score as well as median WBC count in the EUTOS population-based registry are different from many prospective studies. This should be taken in due consideration before extrapolating the results of treatment studies to real life. Spleen size, which is known as an important value for prediction, is only very rarely reported in clinical studies. With further follow-up, this registry will provide a population-based insight on treatment, survival, and causes of death. Disclosures Baccarani: Novartis, BMS, Pfizer, Ariad: Consultancy, Honoraria, Speakers Bureau. Hoffmann:Novartis: Research Funding. Rosti:Novartis: Consultancy, Honoraria; Bristol Myers Squibb: Consultancy, Honoraria. Castagnetti:Novartis: Consultancy, Honoraria; Bristol Myers Squibb: Consultancy, Honoraria; Pfizer: Consultancy. Saussele:Novartis: Honoraria, Research Funding; Bristol Myers Squibb: Honoraria, Research Funding; Pfizer: Honoraria. Steegmann:Novartis, BMS, Pfizer: Honoraria, Research Funding. Mayer:Ariad: Consultancy, Research Funding; Bristol Myers Squibb: Consultancy, Research Funding; Novartis: Consultancy, Research Funding. Turkina:Novartis: Consultancy, Honoraria; Bristol Myers Squibb: Consultancy, Honoraria. Zaritskey:Novartis: Consultancy. Clark:Novartis Pharmaceuticals Corporation: Honoraria, Research Funding, Speakers Bureau; Bristol Myers Squibb: Honoraria, Research Funding; Pfizer: Honoraria, Research Funding. Porkka:BMS: Honoraria; BMS: Research Funding; Novartis: Honoraria; Novartis: Research Funding; Pfizer: Research Funding. Hehlmann:Novartis: Research Funding; Bristol-Myers Squibb: Research Funding. Hasford:Novartis: Research Funding. Lindoerfer:Novartis: Research Funding.

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Treatment Patterns, Adverse Events, and Economic Burden in a Privately Insured Population of Newly Diagnosed Patients with Chronic Lymphocytic Leukemia in the United States

Blood ◽

10.1182/blood-2018-99-114735 ◽

2018 ◽

Vol 132 (Supplement 1) ◽

pp. 3557-3557 ◽

Cited By ~ 1

Author(s):

Shaum Kabadi ◽

Ravi K Goyal ◽

Saurabh P Nagar ◽

James A Kaye ◽

Keith L Davis ◽

...

Keyword(s):

Health Care ◽

Adverse Events ◽

Systemic Therapy ◽

Research Funding ◽

The United States ◽

Treatment Patterns ◽

Newly Diagnosed ◽

The Us ◽

Privately Insured

Abstract Introduction: Contemporary data describing treatment patterns, adverse events (AEs) and outcomes in CLL patients in clinical practice is lacking. We conducted a retrospective cohort study and assessed treatment patterns, AEs, health care resource use (HCRU), and costs in patients with newly diagnosed CLL. Methods: Using a nationally representative population of privately insured patients in the US, adult patients with CLL were selected if they had continuous health plan enrollment for ≥12 months before the first CLL diagnosis without any evidence of any CLL-directed treatment. Treatment patterns up to 4 lines of therapy (LOT) and occurrence of AEs during CLL therapies were assessed. Mean per-patient monthly HCRU and costs were assessed overall and by number of unique AEs. Results: Of all patients meeting the selection criteria (n=7,639; median age, 66 years), 18% (n=1,379) received a systemic therapy during study follow-up. The most common systemic therapy regimens, regardless of therapy line, were bendamustine/rituximab (BR) (32%), rituximab monotherapy (24% [including maintenance]), ibrutinib monotherapy (15%), and fludarabine/cyclophosphamide/ rituximab (FCR) (14%). Of these, BR was the most common LOT-1 regimen (28.1%), while ibrutinib was the most common regimen in LOT-2 (20.8%) and in LOT-3 (25.5%). Use of idelalisib was limited to 1.6% of all patients receiving systemic therapy; however, an increasing trend was observed as patients moved from first to fourth LOT (<1% in LOT-1, 3.1% in LOT-2, 4.7% in LOT-3, and 8.6% in LOT-4). AEs identified during the most common treatments for CLL are presented by treatment regimen in Table 1. The mean monthly all-cause and CLL-related costs, among patients treated with a systemic therapy, were $7,943 (SD=$15,757) and $5,185 (SD=$9,935), respectively. Figure 1 depicts mean monthly costs by care setting and number of AEs, among all patients. Mean (SD) monthly all-cause costs during the post-index date follow-up were $905 ($1,865) among those with no AEs, $1,655 ($5,364) among those with 1-2 AEs, $2,883 ($8,483) among those with 3-5 AEs, and $6,032 ($13,290) among those with ≥6 AEs. Conclusions: This population-based study yielded recent real-world evidence on treatment patterns, AEs, HCRU, and costs in patients enrolled in health plans in the US. Immunochemotherapy, particularly BR, remained the preferred frontline therapy for CLL, whereas ibrutinib was the preferred therapy in LOT-2 and LOT-3, during the study period. This study demonstrates that the AE burden associated with current treatment alternatives for CLL is substantial, and the management of AEs occurring during treatments may have a significant impact on the overall health care costs. Disclosures Kabadi: AstraZeneca: Employment. Goyal:RTI Health Solutions: Employment, Research Funding. Nagar:RTI Health Solutions: Employment, Research Funding. Kaye:RTI Health Solutions: Employment, Research Funding. Davis:RTI Health Solutions: Employment, Research Funding. Mato:Celgene: Consultancy; AstraZeneca: Consultancy; Pharmacyclics: Consultancy, Honoraria, Research Funding; Abbvie: Consultancy; Sunesis: Honoraria, Research Funding; TG Therapeutics: Research Funding; Janssen: Consultancy, Honoraria; Acerta: Research Funding; Prime Oncology: Speakers Bureau; Regeneron: Research Funding.

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Treatment Patterns Among Adults with Newly Diagnosed Primary Immune Thrombocytopenia in the United States

Blood ◽

10.1182/blood-2018-99-117252 ◽

2018 ◽

Vol 132 (Supplement 1) ◽

pp. 497-497

Author(s):

Karynsa Cetin ◽

Leah J McGrath ◽

Robert Overman ◽

Diane Reams ◽

Anjali Sharma ◽

...

Keyword(s):

Cumulative Incidence ◽

Research Funding ◽

Rescue Therapy ◽

The United States ◽

Advisory Board ◽

Oral Steroid ◽

Newly Diagnosed ◽

Employment Equity ◽

The Us ◽

Equity Ownership

Abstract Introduction: Immune thrombocytopenia (ITP) is a rare platelet disorder that can lead to an increased tendency to bleed. Recommended first-line therapies include corticosteroids, intravenous immunoglobulin (IVIg) and intravenous (IV) anti-D. An estimated two-thirds of adult patients with ITP will develop persistent or chronic disease (ITP lasting 3-12 months or >12 months, respectively). Several evidence-based options for second-line treatment exist, but no randomized trials have directly compared one therapy to another. Patterns of treatment in routine clinical practice therefore vary. There is a paucity of data on current real-world treatment dynamics in ITP, and such data could help identify gaps in care and inform future studies of real-world comparative effectiveness and safety. We described the types of treatments administered following an ITP diagnosis, as well as the subsequent occurrence of bleeding and requirement for rescue therapy among adults being managed in routine practice in the United States (US). Methods: We used electronic health record data from hematology clinics across the US (Flatiron Health, Inc.) linked to MarketScan® employer-based and Medicare Supplemental administrative health insurance claims databases (Truven Health Analytics, Inc.). We included patients aged 18 years or older with a new ITP diagnosis from January 1, 2011 through June 30, 2016, continuous enrollment in MarketScan prior to diagnosis, and no previous diagnosis of a secondary cause of thrombocytopenia. The cumulative incidence of each ITP treatment after diagnosis was estimated using competing risk models to account for deaths occurring before initiation. Estimates were provided specifically for 90 days and 1 year following diagnosis to describe treatment uptake in the newly diagnosed and persistent phases, respectively. The incidence of bleeding events and rescue therapy was quantified after the start of the more prevalent second-line therapies: rituximab, splenectomy, and thrombopoietin receptor agonists (TPO RAs) - eltrombopag and romiplostim. Rescue therapies (those that rapidly increase platelet counts in the setting of severe thrombocytopenia or active bleeding) included IV anti-D, IVIg, IV steroids, and platelet transfusions. Results: Among the cohort of 447 adults diagnosed with primary ITP, 47% were male, 61% were white, 32% were 65 years or older, and the median lowest platelet count in the 60 days prior to diagnosis was 85x109/L (IQR: 39, 125). Use of each ITP therapy by 90 days and 12 months post-diagnosis are provided in the Table. Oral corticosteroids were the most commonly used; the cumulative incidence of initiation was 41% by 90 days and 50% by 1 year following ITP diagnosis. IV steroids and rituximab were the next most frequently used medications (16% and 11% at 90 days; and 26% and 16% by 1 year, respectively). The cumulative incidence of the TPO RAs, eltrombopag and romiplostim, by 90 days was 3% and 7%, respectively, and by 1 year was 5% and 9%, respectively. Splenectomy was relatively rare (<4% by 1 year) as was use of all other non-rescue ITP medications (≤1% by 1 year). At 180 days post-ITP treatment initiation, rituximab initiators (N = 84) had a slightly lower incidence of bleeding overall (12% [6, 20]) than the other treatment groups (17% [6, 33] among 31 eltrombopag initiators; 19% [9, 31] among 49 romiplostim initiators; and 19% [6, 38] among 21 splenectomized patients). However, rituximab initiators had the highest cumulative incidence of rescue therapy use (48% [36, 58] compared with 29% [14, 46] for eltrombopag, 26% [14, 39] for romiplostim, and 19% [6, 39] for splenectomized patients). Subsequent oral steroid use was less frequent among TPO RA initiators than rituximab initiators or patients who underwent splenectomy. Conclusions: In this descriptive study of patients with primary ITP receiving care in the US, oral steroids were the most commonly used medication after diagnosis, reflecting their continued role as a frontline therapy. By 1 year after diagnosis, approximately 15% received rituximab, nearly 10% received romiplostim, and 5% received eltrombopag. Splenectomy was less common. Among the medical treatments, although bleeding risk overall appeared lowest in rituximab patients, oral steroid and rescue therapy use were lowest among the patients who initiated TPO RAs. Table. Table. Disclosures Cetin: Amgen Inc: Employment, Equity Ownership. Sharma:Amgen: Employment, Equity Ownership. Brookhart:Amgen Inc: Consultancy, Research Funding; NoviSci: Equity Ownership; Union Chimique Belge: Consultancy; GlaxoSmithKline: Consultancy; Merck: Consultancy; Genentech: Consultancy; TargetPharma: Consultancy; RxAnte: Consultancy; AstraZeneca: Research Funding. Altomare:Genentech: Consultancy; Ipsen: Other: Advisory Board Member; Amgen: Consultancy; Celgene: Other: Advisory Board Member; Novartis: Consultancy; Bayer: Consultancy; Incyte: Consultancy. Wasser:Amgen Inc: Consultancy; Novartis: Consultancy; Becton Dickinson: Equity Ownership; Abbott Labs: Equity Ownership; Biogen: Equity Ownership; Allergan: Equity Ownership; Eli Lilly: Equity Ownership; Incyte: Research Funding; Merck: Equity Ownership, Research Funding; Pfizer: Equity Ownership, Research Funding; Guardant: Research Funding.

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Economic Burden of Disease Progression Among Transplant Eligible Multiple Myeloma Patients Who Have Received at Least One Line of Therapy in the US

Blood ◽

10.1182/blood-2019-123743 ◽

2019 ◽

Vol 134 (Supplement_1) ◽

pp. 2211-2211

Author(s):

Rafael Fonseca ◽

May Hagiwara ◽

Sumeet Panjabi ◽

Emre Yucel ◽

Jacqueline Buchanan ◽

...

Keyword(s):

Disease Progression ◽

Economic Burden ◽

Burden Of Disease ◽

Research Funding ◽

Index Date ◽

Economic Burden Of Disease ◽

The Us ◽

Event Date ◽

Baseline Characteristics ◽

Line Of Therapy

Introduction Data on the economic burden of disease progression among multiple myeloma (MM) patients who have had a stem cell transplant (SCT) in the US are limited. This study evaluated the impact of disease progression on healthcare resource utilization (HRU) and costs among these patients in the US. Methods Adult patients (pts) with a confirmed MM diagnosis (Dx), defined as ≥1 inpatient or ≥2 non-diagnostic outpatient claims 30-365 days apart who initiated a course of MM therapy (Rx) during the study period (Jan 1, 2006 to Dec 31, 2016) and received SCT during the first year after the initial diagnosis of MM were identified from the Truven Analytics MedStat MarketScan® Commercial Claims and Encounters and Medicare and Coordination of Benefits databases. The first date with Dx for MM was defined as the "Dx date" and line of therapy (LOT) was identified based on an adaptation of a previously-developed algorithm. Pts were excluded if they had a gap in enrollment during the period beginning 6-month before the Dx date through 30-days after the first LOT (1L) initiation date, received MM Rx, unclassified antineoplastic medications, or SCT before the Dx date, or had missing information required to identify LOT or assess baseline characteristics or study outcomes. The 12 months after initiation of each LOT was designated the "event identification period" for progression. Disease progression was defined as advancement to next LOT, diagnosis or procedure codes indicative of bone metastasis, hypercalcemia, soft tissue plasmacytoma, skeletal related events, or acute kidney disease while off MM Rx, or death. The "event date" for progression was defined as date with evidence of progression. For each pt with progression, one pt without progression was randomly selected and assigned an event date to ensure that the distribution of time from LOT initiation to event date for pts without progression was the same as the distribution of time from LOT initiation to event date for pts with progression. For each LOT (L1-L3), annual HCRU and costs from index date, defined as 30-day before the event date, through disenrollment or end of the study period were compared for pts with vs without progression using inverse probability of treatment weighting (IPTW) to adjust for differences between groups in baseline characteristics including year of index date, age, gender, region, plan type, Medicare status, comorbidities, and pre-index HRU and healthcare costs. All costs were adjusted to 2016 US $. In a subgroup analysis, HCRU and costs were evaluated for LOTs initiated from 2013 to 2016 to reflect patterns of treatment in current era. Results Of the 28,184 adult MM pts who received ≥1 MM Rx during the study period, 3226 qualified for the study, including 837 pts with progression and 2389 without progression during 1L. The corresponding numbers for 2L, and 3L were 412 and 589, and 226 and 196, respectively. Mean age at index date was 57 years; 57% were male. After IPTW, all baseline characteristics were well balanced in all LOTs with standardized mean differences <0.1 for most baseline characteristics. The mean number of hospitalizations per year was greater for pts with progression in all LOTs, with differences ranging from 1.11 (95%CI 0.85, 1.37) for 2L to 0.34 (95%CI 0.21, 0.47) for 1L. In all LOTs, annual costs of inpatient and outpatient services were greater for pts with progression while costs of outpatient prescriptions were greater among patient without progression. Total healthcare costs were greater for pts with vs without progression in all LOTs with hospitalizations and outpatient visit costs being key drivers. Total incremental annual costs in those who progressed were higher by $13,386 (1L), $105,004 (2L), and $70,206 (3L). When the analysis was restricted to LOTs initiated between 2013 and 2016, the incremental costs in pts who progressed were generally greater in the most recent era: $43,381 (1L), $113,797 (2L), and $115,327 (3L). Conclusions For MM pts receiving drug Rx and SCT the economic burden of disease progression is substantial across all LOTs. Incremental costs of progression are largely attributable to hospitalizations and outpatient visits. Treatments that prevent or delay progression are likely to yield important reductions in downstream disease management costs. These savings should be considered in frameworks assessing the value of innovative treatments for MM. Disclosures Fonseca: AbbVie, Amgen, Bayer, Celgene, Kite, Janssen, Juno, Merck, Pharmacylics, Sanofi, Takeda: Other: Consultant/Advisor; Prognosticatin of MM based on Genetic Categorization by FISH: Patents & Royalties; Adaptive Biotechnologies: Other: Scientific Advisory Board. Hagiwara:Amgen Inc.: Research Funding. Panjabi:Amgen Inc.: Employment, Other: Owns Amgen stock, Research Funding. Yucel:Amgen Inc.: Employment, Other: Owns Amgen stock, Research Funding. Buchanan:Amgen Inc.: Employment, Other: Owns Amgen stock, Research Funding. Delea:Amgen Inc.: Research Funding.

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Burden of Disease Progression in Patients with Multiple Myeloma Who Have Received at Least One Line of Therapy in the US

Blood ◽

10.1182/blood-2018-99-117230 ◽

2018 ◽

Vol 132 (Supplement 1) ◽

pp. 4754-4754 ◽

Cited By ~ 2

Author(s):

May Hagiwara ◽

Thomas Delea ◽

Sumeet Panjabi ◽

Emre Yucel ◽

Rafael Fonseca

Keyword(s):

Disease Progression ◽

Economic Burden ◽

Burden Of Disease ◽

Research Funding ◽

Index Date ◽

Economic Burden Of Disease ◽

The Us ◽

Event Date ◽

Baseline Characteristics ◽

Line Of Therapy

Abstract Introduction Previous studies have documented substantial economic burden for multiple myeloma (MM) patients (pts). However, data on the economic burden of disease progression in the US are limited. In this study, we estimated effects of disease progression on healthcare resource utilization (HRU) and costs among pts with MM who have received at least one line of therapy (LOT) in the US. Methods Data from the Truven Analytics MedStat MarketScan® Commercial Claims and Encounters and Medicare and Coordination of Benefits databases were used. A retrospective incident user cohort of adult pts with a confirmed diagnosis (Dx), defined as ≥1 inpatient or ≥2 non-diagnostic outpatient claims 30-365 days apart with diagnosis, of MM who initiated a course of MM therapy during the study period (Jan 1, 2006 to Dec 31, 2016) was identified. The first date with Dx for MM was defined as the "Dx date" and LOT was identified based on a published algorithm. Pts were excluded if they had a gap in enrollment during the period beginning 6-month before the Dx date through 30-days after the first LOT (1L) initiation date, received MM therapy or unclassified antineoplastic medications before the Dx date or stem cell transplant (SCT) at any time during the study period, or had missing information required to identify LOT or assess baseline characteristics or study outcomes. The 12 months after initiation of each LOT was designated the "event identification period" for progression. Disease progression was defined as advancement to the next LOT, ICD-9/10 or CPT/HCPCS diagnosis or procedure codes indicative of bone metastasis, hypercalcemia, soft tissue plasmacytoma, skeletal related events, or acute kidney disease while off MM therapy, or death. The "event date" for progression was defined as date with evidence of progression. For each pt with progression, one pt without progression was randomly selected and assigned an index date to ensure that distribution of time from LOT initiation to event date for pts with progression was the same as that for pts without progression. For each LOT (L1-L4), annual HRU and costs from index date through disenrollment or the end of the study period were compared for pts with vs without progression using inverse probability of treatment weighting (IPTW) to adjust for differences between groups in baseline characteristics including year of index date, age, gender, region, plan type, Medicare status, comorbidities, and pre-index HRU and healthcare costs. All costs were adjusted to 2016 US dollars. In a subgroup analysis, HRU and costs were evaluated for LOTs initiated from 2013 to 2016 to reflect patterns of treatment in current era. Results Of the 28,184 adult MM pts who received one or more MM therapy during the study period, 11,179 qualified for the study, including 4468 and 6598 pts with and without progression during 1L, respectively. The corresponding numbers for 2L, 3L, and 4L were 1563 and 2207, 618 and 714, and 244 and 260, respectively. Mean age at index date was 68 years; 57% were male. After IPTW, all baseline characteristics were well balanced in all LOTs with standardized mean differences <0.1 for all characteristics. The mean number of hospitalizations per year was greater for patents with progression in all LOTs, with differences ranging from 0.46 (95%CI 0.40, 0.52) for 1L to 0.89 (95%CI 0.67, 1.11) for 3L. In all LOTs, annual costs of inpatient and outpatient services were greater for pts with progression while costs of outpatient prescriptions were greater among patient without progression. Total healthcare costs were greater for pts with vs without progression in all LOTs with hospitalizations and outpatient visit costs being key drivers. Total incremental annual costs in those who progressed were higher by $30,381 (1L), $33,064 (2L), $34,495 (3L), and $25,163 (4L). When the analysis was restricted to LOTs initiated between 2013 and 2016, the incremental costs in patients who progressed were greater in the most recent era: $52,739 (1L), $49,012 (2L), $25,329 (3L), and $65,400. Conclusions For MM pts receiving drug therapy, the economic burden of disease progression is substantial across all LOTs and this burden is greater in the most recent era (2013 to 2016). Treatments that prevent or delay progression are likely to yield important reductions in downstream disease management costs. These savings should be considered in frameworks assessing the value of innovative treatments for MM. Disclosures Hagiwara: Jazz Pharmaceuticals: Research Funding; Jazz Pharmaceuticals: Consultancy; Amgen: Research Funding; Amgen: Consultancy; Policy Analysis, Inc: Employment. Delea:Novartis: Consultancy, Research Funding; Amgen: Consultancy, Research Funding; Pfizer: Consultancy, Research Funding; Sanofi: Consultancy, Research Funding; Takeda: Research Funding; Seattle Genetics: Research Funding; Policy Analysis Inc.: Employment. Panjabi:Amgen: Employment, Equity Ownership. Yucel:Amgen: Employment, Equity Ownership. Fonseca:Mayo Clinic: Employment; Amgen: Consultancy.

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855. Persistence on F/TAF versus F/TDF for HIV Pre-Exposure Prophylaxis: A Real-World Evidence Analysis in the United States

Open Forum Infectious Diseases ◽

10.1093/ofid/ofab466.1050 ◽

2021 ◽

Vol 8 (Supplement_1) ◽

pp. S518-S519

Author(s):

Li Tao ◽

Valentina Shvachko ◽

Moupali Das ◽

Christoph C Carter ◽

Jared Baeten ◽

...

Keyword(s):

United States ◽

Real World ◽

The United States ◽

Odds Ratios ◽

Preexposure Prophylaxis ◽

Hazard Ratios ◽

Real World Evidence ◽

The Us ◽

Evidence Analysis ◽

Exposure Prophylaxis

Abstract Background Persistence to preexposure prophylaxis (PrEP) is an important determinant of its efficacy, but evidence on real-world persistence is lacking. This study assesses adherence to F/TDF and F/TAF for PrEP both in terms of discontinuation and re-initiation patterns. Methods We identified HIV-negative individuals in the United States who initiated F/TDF or F/TAF for PrEP between October 2019 and December 2020 from a de-identified prescription claims database; users taking generic F/TDF were excluded. Non-persistence was defined as a prescription fill gap of >30 days; discontinuation included switch from F/TDF to F/TAF or F/TAF to F/TDF. We used survival analyses to estimate persistence, Cox regressions to compare the hazard ratios (HR) of discontinuation, and logistic regression to compare the odds ratios of re-initiation after discontinuation. Results Among F/TAF users (N=82,402) median age at PrEP initiation was 35 years (interquartile range [IQR] 28−47) and median PrEP persistence was 4 months (IQR 1.8-8.9), compared to 31 years (IQR 25−40) and 2 months (IQR 1.0-3.8) for F/TDF users (N=48,501). PrEP persistence at 60 and 90 days was higher among F/TAF users than F/TDF users (Figure). F/TDF users were 2.5 times more likely to discontinue than F/TAF users, with more marked differences in older users than that in younger users (p for interaction between discontinuation and age group < 0.01, Table). We also observed a higher rate of discontinuation of F/TDF versus F/TAF if PrEP was prescribed by internal medicine or infectious disease physicians than by family medicine physicians (data not shown). After discontinuation, F/TAF users were 1.7 times more likely than F/TDF users to re-initiate PrEP; the association was not different by age. Persistence rates of F/TAF and F/TDF for PrEP by time of PrEP initiation Hazard ratios (HR) and corresponding 95% confidence intervals (CI) of non-persistence and odds ratios (OR) of re-initiation after discontinuation for users of F/TAF and F/TDF for PrEP in the US, Oct 2019 – Dec 2020 Conclusion In this real-world analysis, the F/TAF for PrEP regimen was associated with higher persistence and re-initiation than F/TDF for PrEP. These findings underscore the dynamic nature of PrEP utilization in the real-world and the importance of interventions aimed at improving PrEP persistence and re-initiation in people who would benefit from PrEP. Disclosures Li Tao, MD, PhD, Gilead Sciences Inc (Employee, Shareholder) Valentina Shvachko, MS, Gilead Sciences Inc (Employee, Shareholder) Moupali Das, MD, Gilead Sciences Inc. (Employee, Shareholder) Christoph C. Carter, MD, Gilead Sciences Inc. (Employee, Shareholder) Jared Baeten, MD, PHD, Gilead Sciences Inc. (Employee, Shareholder) David Magnuson, PharmD, Gilead Sciences Inc (Employee, Shareholder)

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The lines of loco-regional therapies received and the healthcare economic burden of patients newly diagnosed with hepatocellular carcinoma in the United States.

Journal of Clinical Oncology ◽

10.1200/jco.2021.39.3_suppl.286 ◽

2021 ◽

Vol 39 (3_suppl) ◽

pp. 286-286

Author(s):

Brian Seal ◽

Abdulla M. Abdulhalim ◽

Melissa Lingohr-Smith ◽

Jay Lin

Keyword(s):

Radiation Therapy ◽

Economic Burden ◽

Healthcare Costs ◽

Index Date ◽

The United States ◽

Newly Diagnosed ◽

Ablative Therapy ◽

Regional Therapy ◽

The Us

286 Background: Hepatocelluar carcinoma (HCC) is one of the fastest growing causes of cancer-related deaths in the US. The objectives of this study were to examine the lines of loco-regional therapies received and the healthcare economic burden of patients newly diagnosed with HCC in the US. Methods: Patients (≥18 years of age) diagnosed with HCC who received ≥1 loco-regional therapy (index date) after diagnosis were identified from the MarketScan Commercial and Medicare Supplemental databases (July 1, 2013-May 31, 2018). Patients were required to have ≥6 months of continuous insurance enrollment before the index date and ≥1 month after (follow-up period). The follow-up period was censored when patients received any systemic therapy. During the follow-up periods, the number of patients who received radiation therapy, ablative therapy, and embolization procedures (transarterial embolization/chemoembolization [TAE] and radioembolization [TARE]) were examined. All-cause and HCC-related healthcare costs (total and patient out-of-pocket [OOP] payments per patient per month [PPPM]) were also measured. Results: Among the 2,101 patients newly diagnosed with HCC who received ≥1 loco-regional therapy, median age was 64 years and 75.0% were male; the mean follow-up duration was 11.5 months. During the follow-up periods, 28.1% (n = 590) received radiation therapy, 27.3% (n = 574) ablative therapy, and 77.3% (n = 1,623) embolization therapy (TAE: 68.7% [n = 1,443]; TARE: 20.7% [n = 434]); 30.9% of patients received multiple loco-regional therapies. During the follow-up periods, total all-cause healthcare costs were a mean of $20,316 (OOP: $378) PPPM, of which 70.7% ($14,359; OOP: $227 PPPM) were HCC-related. The breakdown of healthcare costs is shown in the table. Conclusions: According to the findings of this large-scale real-world analysis of patients newly diagnosed with HCC in the US, a vast majority received at least one embolization procedure. The monthly healthcare economic burden of patients with HCC treated with local-regional therapies is relatively high. [Table: see text]

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Real-World Effectiveness of Voxelotor for Treating Sickle Cell Disease in the US

Blood ◽

10.1182/blood-2020-140525 ◽

2020 ◽

Vol 136 (Supplement 1) ◽

pp. 25-25

Author(s):

Ahmar Urooj Zaidi ◽

Thokozeni Lipato ◽

Ofelia A. Alvarez ◽

Alexander Lonshteyn ◽

Derek Weycker ◽

...

Keyword(s):

Board Of Directors ◽

Real World ◽

Research Funding ◽

Index Date ◽

Health Claims ◽

Transfusion Rate ◽

Publicly Traded ◽

Advisory Committees ◽

The Us

Background: Until late 2019, few treatments had been approved by the FDA for treating sickle cell disease (SCD). Voxelotor (Oxbryta®) is a sickle hemoglobin-polymerization inhibitor approved by the FDA in November 2019 for treatment of SCD in adults and adolescents aged ≥12 years under an accelerated approval based on results of the pivotal HOPE study. In HOPE, voxelotor increased average hemoglobin (Hb) by 1.1 g/dL from baseline in patients with 1-10 vaso-occlusive crises (VOCs) in the previous year and a Hb level between 5.5 and 10.5 g/dL who were not transfusion dependent. Of the participants on voxelotor 1500 mg, 51% had a Hb response >1.0 g/dL at week 24 (Vichinsky et al, NEJM 2019). This study sought to assess the real-world effectiveness of voxelotor based on data during the first 6 months post FDA approval. Methods: Data on medical and pharmacy claims for patients who were aged ≥12 years and receiving voxelotor from December 2019 to May 2020 were obtained from the Symphony Health claims dataset. For each patient, the date of the first voxelotor claim was defined as the "index date." Patients with at least 1 year's data prior to the index date were included in the analyses. Patients' demographic and clinical characteristics were summarized descriptively. Standardized annualized rates of transfusions and VOCs per patient per year (PPPY) prior to and after voxelotor initiation were compared. A subset of patients in the Symphony Health claims dataset had Hb lab data available. Patients with at least 1 Hb result within 30 days prior to the index date and at least 1 Hb result after the index date were included for Hb analyses. For these patients, change in Hb and the percentage of patients achieving a >1 g/dL increase in Hb were summarized. Confidence intervals and P values for changes in outcomes were based on bootstrapping. Results: As of May 31, 2020, 1275 patients from the Symphony Health claims datasets were identified who received voxelotor (40% male, mean age 35.7 years). In the year prior to voxelotor initiation, 715 (56.1%) of these patients received hydroxyurea, 121 (9.5%) received L-glutamine, 166 (13.0%) received at least 1 transfusion, 17 (1.3%) were on chronic transfusion (≥8 transfusions per year), and 681 (53.4%) had 1 or more VOCs. Mean (SD) follow-up was 64.9 (40.7) days. Among 1275 patients, 175 and 52 patients had at least 1 Hb level measurement during the 1 year prior to and after voxelotor initiation, respectively. Among the subset of patients with their Hb level tested within 30 days prior to the index date and at least 1 Hb level after index date (n=22), the baseline average Hb level was 8.0 g/dL (SD 1.4, median 7.9 g/dL, range 5.0-11.8 g/dL). Mean increase in Hb from baseline was 1.1-1.3 g/dL (Table 1) depending on the approach used to calculate Hb levels after voxelotor initiation; 55% (95% CI 32%-77%) of patients achieved a Hb increase >1 g/dL after voxelotor initiation. Among all 1275 patients, mean (SD) overall transfusion rates declined from 0.45 (1.67) PPPY pre-index to 0.31 (1.88) post-index, a change of -0.14 PPPY (P=0.005). Among 169 patients who received at least 1 transfusion in the year prior to initiation of voxelotor, the transfusion rate dropped from 3.39 (3.34) to 1.75 (4.30) PPPY, a change of -1.64 PPPY (P<0.001). Among 17 patients receiving chronic transfusions, the transfusion rate dropped from 11.29 (3.12) to 6.74 (7.37) PPPY, a change of -4.56 PPPY (P=0.013) (Table 2). After voxelotor initiation, the annualized rates of VOC were numerically reduced from 3.86 (6.69) to 3.64 (8.54) (P=0.248). To address the potential bias from the relatively short follow-up duration, similar results of transfusion and VOC rates were observed among patients with at least 30 days of follow-up or when only events within 3 months prior to index date were considered. Conclusions: Based on the first 6 months' data after the approval of voxelotor in the US, in real-world practice, voxelotor increases Hb by at least 1 g/dL, consistent with the HOPE randomized controlled trial results. Evidence suggests that transfusion rates decreased after voxelotor initiation. A favorable downward trend in VOC rates was also observed. This real-world evidence provides additional support for the use of this novel therapy in the treatment of hemolytic anemia and its associated complications in the SCD population. Further evaluation with a larger sample size and longer follow-up will help to confirm these findings. Disclosures Zaidi: Global Blood Therapeutics: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Novartis: Consultancy, Honoraria; Cyclerion: Consultancy, Honoraria; Imara: Consultancy, Honoraria; bluebird bio: Consultancy, Honoraria; Emmaus Life Sciences: Consultancy, Honoraria. Alvarez:Novartis: Membership on an entity's Board of Directors or advisory committees; Forma Therapeutics: Membership on an entity's Board of Directors or advisory committees. Lonshteyn:Policy Analysis Inc.: Current Employment; Global Blood Therapeutics: Research Funding; Novartis: Research Funding. Weycker:Policy Analysis Inc.: Current Employment, Current equity holder in publicly-traded company; Novartis: Research Funding; Global Blood Therapeutics: Research Funding. Pham:Policy Analysis Inc.: Current Employment; Global Blood Therapeutics: Research Funding; Novartis: Research Funding. Delea:Global Blood Therapeutics: Research Funding; Novartis: Research Funding; Policy Analysis Inc.: Current Employment, Current equity holder in private company. Agodoa:Global Blood Therapeutics: Current Employment, Current equity holder in publicly-traded company. Cong:Global Blood Therapeutics: Current Employment, Current equity holder in publicly-traded company. Shah:Novartis: Consultancy, Research Funding, Speakers Bureau; Global Blood Therapeutics: Consultancy, Research Funding, Speakers Bureau; CSL Behring: Consultancy; Alexion: Speakers Bureau; Bluebird Bio: Consultancy.

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Epidemiologic and Clinical Characteristics of Thalassemia (Thal) Intermedia (TI) in the United States

Blood ◽

10.1182/blood.v126.23.3279.3279 ◽

2015 ◽

Vol 126 (23) ◽

pp. 3279-3279

Author(s):

Elliott P. Vichinsky ◽

Janet Kwiatkowski ◽

Patricia J. Giardina ◽

Carole Paley ◽

Francis Vekeman ◽

...

Keyword(s):

Serum Ferritin ◽

Research Funding ◽

Clinical Characteristics ◽

Index Date ◽

Growth Failure ◽

The United States ◽

Compound Heterozygous ◽

Foreign Born ◽

Liver Iron ◽

The Us

Abstract Introduction TI is increasingly prevalent in the US due to changing immigration patterns. It is underdiagnosed, leading to inadequate or delayed management. This study reviewed the prevalence, epidemiology, and clinical characteristics of TI in patients (pts) in the US. Methods Medical records from 1/1997- 4/2014 at 4 US hematology centers were reviewed. Index date was the 1st TI visit at a center on or after 1/1/1997. Eligible pts had a TI diagnosis (≤8 mean packed red blood cell transfusions per year (yr) over a ≥3-y period after index date) and ≥12 months of follow-up. Data spanned from index date to death or last record. Descriptive analyses of demographic and clinical data were done by TI subtype. Results Of 138 pts enrolled, 84 had α-thal, 39 had β-thal, and 15 had E/β-thal. 74% of α-thal pts had deletional (del) Hb H, and 26% had non-deletional (ndel) Hb H. 59% of β-thal pts had homozygous or compound heterozygous β-globin mutations (8% with α deletion, 51% without), 20% had a single β mutation with α-gene triplication, 13% had autosomal dominant β thal, and 8% had other β-globin mutations. Of the E/β-thal pts, 80% had E/β0 and 20% had E/β+. Median age at index date was 2.3 yr (1.64 del; 6.1 ndel) in the α-thal group, 9.2 yr in the β-thal group, and 2.2 yr in the E/β-thal group. Most α-thal (77%) and E/β-thal (87%) pts were Asian; most β-thal pts were White (46%) or African-American (36%). Most α-thal (56%) and E/β-thal (53%) pts were of Southeast Asian origin; most β-thal pts were of Mediterranean (31%) or African (21%) origin. 21%, 10%, and 20% of α, β, and E/β-thal pts, respectively, were foreign-born, and 5%, 3% and 7%, respectively, were transfused outside of the US. Observation length was similar across subtypes (median: 5.3 yr). Clinical comorbidities are shown in Table 1. 22% of pts received ≥1 transfusion, while 7% of pts received ≥8 transfusions in any 1 yr to treat anemia, acute hemolysis, or cardiac abnormality; increased transfusions were initiated due to growth failure, acute hemolysis, or unspecified reasons. β-thal pts had a higher mean number of transfusions per pt per yr (PPPY) (α: 0.4 (0.0 del; 1.5 ndel), β: 0.9, E/β: 0.2) and higher mean serum ferritin (ng/mL) (204.3; 511.7; 362.4), and more often had iron chelation therapy (ICT) (11%; 28%; 7%). There was an association between higher serum ferritin, more frequent transfusions, and older age. In pts <10 yr, mean number of transfusions PPPY for regularly and ever transfused was 4.0 and 2.1, and serum ferritin for regularly, ever, and never transfused was 723.7, 438.0, and 146.4 ng/mL. In pts >18 yr, these values were 9.8 and 4.6 transfusions PPPY and 1166.8, 1042.2, and 521.4 ng/mL. An association also existed between ICT and higher mean serum ferritin (ICT: 769.9; no ICT: 463.7 ng/mL). 22% of pts had ≥1 liver iron test, and 18% had ≥1 cardiac iron test. Tested pts were older than those not tested (median yr, liver iron: 26.1 vs 7.4; cardiac: 20.1 vs 4.6). Among tested pts, 78% (25/32) had abnormal liver iron results. Median (range) LIC based on R2 or SQUID was 10.8 (2.5-18.2) mg/g dw, with corresponding within-12-month serum ferritin of 494 (127-1770) ng/mL. 49% (17/35) of pts tested had abnormal cardiac results based on electrocardiogram or echocardiogram. Table 1. Clinical comorbidities All α-thal α-thal del α-thal ndel β-thal E/β-thal N=138 N=84 N=62 N=22 N=39 N=15 Splenomegaly 54% 55% 39% 100% 51% 60% Extramedullary hematopoiesis 28% 24% 13% 55% 36% 33% Growth retardation 21% 23% 18% 36% 13% 33% Hepatomegaly 21% 24% 13% 55% 15% 20% Infections needing hospitalization/IV antibiotics 21% 25% 19% 41% 15% 13% Hypoparathyroidism/hypocalcemia 15% 17% 8% 41% 10% 20% Osteopenia/osteoporosis 13% 11% 5% 27% 21% 7% Bone deformities 9% 6% 5% 9% 21% 0% Splenectomy and/or cholecystectomy 9% 5% 2% 14% 21% 0% Cardiomegaly 4% 6% 2% 18% 0% 7% Conclusion TI in the US affects a diverse population. Our data showed a higher prevalence in African-Americans than previously documented. Rates of comorbidity and transfusion frequency increased with age. 18% and 4% of pts were born and transfused outside of the US, potentially leading to additional transfusion-related morbidity. Consistent with extant data, serum ferritin in TI often underestimated actual LIC, rendering more pts potentially eligible for ICT than observed. Morbidities observed in this study underscore the need for better and earlier diagnosis, substantiating the need for nationwide TI screening/surveillance to optimize management. Disclosures Vichinsky: Novartis: Research Funding. Kwiatkowski:Novartis: Research Funding; Sideris Pharmaceuticals: Consultancy; Shire Pharmaceuticals and Sideris Pharmaceuticals: Consultancy; ISIS: Membership on an entity's Board of Directors or advisory committees. Paley:Novartis: Employment. Vekeman:Novartis: Research Funding. Cheng:Novartis: Research Funding. Damron:Novartis: Research Funding. McCormick:Novartis: Research Funding. Sasane:Novartis: Employment. Qiu:Novartis: Employment. Duh:Novartis: Research Funding. Thompson:Bluebird bio: Consultancy, Research Funding; Novartis: Consultancy, Research Funding; Mast: Research Funding; Apopharma: Consultancy; Baxter: Consultancy, Research Funding.

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