scholarly journals Real-World Treatment Patterns and Outcomes in Patients with Hemophagocytic Lymphohistiocytosis (HLH) and Other Clinical Conditions Treated with Emapalumab: The Real-HLH Study Design

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 4991-4991
Author(s):  
Carl E. Allen ◽  
Jennifer Leiding ◽  
Shanmuganathan Chandrakasan ◽  
Kelly J. Walkovich ◽  
Abiola Oladapo ◽  
...  
Keyword(s):  
Board Of Directors ◽  
Real World ◽  
Clinical Characteristics ◽  
Index Date ◽  
Treatment Patterns ◽  
Patient Identification ◽  
Advisory Committees ◽  
The Real ◽  
The Us ◽  
Clinical Conditions

Abstract Introduction: Hemophagocytic lymphohistiocytosis (HLH) is a rare, life-threatening hyperinflammatory syndrome characterized by overactivation of the immune system due to systemic release of proinflammatory cytokines, especially of interferon gamma (IFNγ), and persistent activation of macrophages/histiocytes and T cells. Emapalumab, a fully human, anti-IFNγ monoclonal antibody that binds to both free and receptor-bound IFNy, neutralizing its biologic activity, was approved by the Food and Drug Administration (FDA) in 2018 for the treatment of adult and pediatric patients with primary HLH with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. The safety and efficacy of emapalumab was based on results from a pivotal phase 2/3 trial which reported a 63% overall response in patients treated with emapalumab. Since approval, no study has evaluated the use of emapalumab in a larger cohort of patients in the real-world clinical setting. Aim: To assess real-world treatment patterns and outcomes, clinical and demographic characteristics among patients treated with emapalumab. Method: This retrospective, non-interventional, observational medical chart review study will include patients treated across treatment centers in the US with emapalumab in a non-clinical trial setting. The study aims to include more than 100 patients who have been treated with at least one dose of emapalumab between November 20, 2018 and December 31, 2020 (patient identification period). The date that the patient initiates treatment with emapalumab within the patient identification period is defined as the index date. The post-index date is defined as the period from the index date through to the study end date (June 30, 2021), end of data availability for the patient, or date of death, whichever occurs first. Patients will be classified into three groups (i.e. primary HLH, secondary HLH, or non-HLH) based on the information obtained from their charts, the HLH 2004 diagnostic criteria, and adjudication by the REAL-HLH Steering Committee. Results: The primary objective of the study is to describe treatment patterns in patients with HLH treated with emapalumab in a real-world clinical setting, including emapalumab dose, treatment duration, and reasons for initiating or discontinuing treatment. The secondary objectives are to describe demographic and clinical characteristics of patients with HLH treated with emapalumab, and their outcomes. The exploratory objectives include detailing the demographic, clinical characteristics, treatment patterns, and outcomes of patients with non-HLH clinical conditions treated with emapalumab (Table 1). Demographics and clinical characteristics will be analyzed at the time of diagnosis. Treatment patterns and outcomes will be analyzed during the post-index date. Conclusion: The study aims to assess treatment patterns and outcomes, clinical and demographic characteristics among patients treated with emapalumab, a novel IFNγ blocking agent, in real-world clinical settings in the US. Figure 1 Figure 1. Disclosures Allen: Sobi: Consultancy. Leiding: Sobi: Consultancy. Chandrakasan: Sobi: Consultancy. Walkovich: X4 Pharmaceuticals: Other: Local PI for clinical trial involving mavorixafor and patients with neutropenia; Swedish Orphan Biovitrum AB (Sobi): Consultancy, Honoraria; Pharming: Honoraria, Membership on an entity's Board of Directors or advisory committees; Horizon Pharmaceuticals: Honoraria, Membership on an entity's Board of Directors or advisory committees. Oladapo: Sobi: Current Employment. Yee: Sobi: Current Employment. Pednekar: Sobi: Consultancy; PRECISIONheor: Current Employment. Raza: Sobi: Consultancy; PRECISIONheor: Current Employment. Jordan: Sobi: Consultancy.

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

Blood ◽  
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.

scholarly journals The Grndad Registry: Contemporary Natural History Data and an Analysis of Real-World Patterns of Use and Limitations of Disease Modifying Therapy in Adults with SCD

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 34-36
Author(s):  
Alexandra Boye-Doe ◽  
Elizabeth Brown ◽  
Charu Puri-Sharma ◽  
Anjulika Chawla ◽  
Joshua J Field ◽  
...  
Keyword(s):  
Board Of Directors ◽  
Real World ◽  
Research Funding ◽  
Clinical Characteristics ◽  
Reticulocyte Count ◽  
Principal Investigator ◽  
Advisory Committees ◽  
Disease Modifying Therapy ◽  
Disease Modifying ◽  
The Impact

Incremental improvement in care for children with sickle cell disease (SCD), arising from government-funded research over the last 4 decades, resulted in a dramatically reduced childhood mortality. However, the impact of iterative research and disease modifying therapy (DMT) on adults with SCD has not been as strong. Until now, there has been no coordinated, longitudinal, generalizable, natural history study of SCD that allowed for an assessment of the contemporary adult population. Here, we describe demographics at enrollment and cross-sectional clinical characteristics of 570 adults with SCD (SCA, homozygous HbSS or HbSb0 N=387, 68%, and compound heterozygous SCD variant, HbSC or HbSb+ N=183, 32%, Table I) on whom we have evaluable data. These data are from the multi-site REDCap-based prospective Globin Research Network for Data and Discovery (GRNDaD) registry, comprising 11 centers with over 1100 consented adults and children. The objective of this work was to evaluate the cohort at year of entry, including the use and clinical associations with DMT, and to explore indicators of disease progression as patients age. 16% of adults with SCA and 9.6% with variant disease stroke; 60.9% of adults with SCA and 41% with variant disease had a history of acute chest syndrome. Albuminuria was prevalent in both SCA (39.5%) and variant disease (19.4%). 185 adults (185/387, 47.8%) with SCA, previously referred for symptoms in clinic, had recorded tricuspid regurgitant jet velocity measurements, with a significantly abnormal result (>2.7 m/s), in 92 (92/185, 49.7%). At enrollment, 45% of adults with SCA (175/387) and 14% of adults with variant disease (25/183) were on hydroxyurea (HU); 20.4% of adults with SCA were on chronic transfusions (79/387) compared with 7% of adults with variant disease (13/183). One third of all adults with SCA were not on or were not consistently on DMT, and had laboratory evidence for increased hemolysis (Table 1). Adults with SCA who were on HU had a higher MCV and higher HbF than other treatment states (Table 1). However, only 34% (60/175) of adults with SCA on HU were at maximally tolerated dose (MTD), per guideline-based recommendations, i.e. ANC ≤4.0 x109/L. On HU, those in the lowest quartile for ANC (<3.2 x109/L) were older (mean age 35.9 years (95% Confidence Limit (CL) 32.5-39.3) vs. 31.2 (95% CL 28.2 to 34.4) years, P=0.04), had a lower mean reticulocyte count (119 x109/L (95% CL 76-162) vs. 203 (95% CL 129-278), P=0.05), and a higher mean MCV (104.4 fL (95% CL 100.2-108.7) vs. 92.5 (95% CL 87.2-97.8), P=0.0007), compared to those in the highest quartile for ANC (>5.7 x109/L, N=34), but did not otherwise differ (including mean HbF, which was not measured in a standardized way). In older adults with SCD (Table 2), fewer people with SCA than with variant disease were >54 years old, (26/387 HbSS, 7%, vs. 34/183, 19%, respectively). The older adult with SCA had a depressed reticulocyte count and a trend towards a higher creatinine. 45% of adults with SCA were on HU, and only a minority were at MTD, highlighting the challenges to optimal long-term therapy in chronic illness. Those patients not stably on DMT had laboratory evidence for worse anemia and hemolysis, without an evident increase in hospital admissions, perhaps due to a hyper hemolytic phenotype. Despite a more intensive regimen, SCA patients on transfusions had a higher Hgb but did not have hemolysis labs that differed from SCA patients on HU. Further, there was no difference in hospitalizations amongst treatments for SCA, although a decrease in hospitalizations was detectable in variant disease (Table 1). Successful use of DMTs in SCA was challenging even in academic centers, and there was evidence for ongoing hemolysis in treated and untreated patients. These real world data provide useful information about adults (>17 years) with SCD. These data highlight opportunities to improve adherence to therapy (patient-centered) and to prescribing guidelines (provider-centered), and to consider less-burdensome alternatives. Importantly, we found that a large proportion of people with SCA were not on DMT, and with HU often not at MTD. In future, the GRNDaD registry will enable prospective longitudinal real-world analyses of the impact of DMTs and/or newer therapies on clinical outcomes, will enhance quality improvement, and will allow us to more fully explore clinical characteristics, of SCA and variant disease, in the aging adult. Disclosures Puri-Sharma: Bluebird Bio: Current Employment. Chawla:Bluebird Bio: Current Employment. Field:Shires: Research Funding; Ironwood: Research Funding. Neumayr:Emmaus: Consultancy; Bayer: Consultancy; CTD Holdings: Consultancy; Pfizer: Consultancy; ApoPharma: Consultancy, Membership on an entity's Board of Directors or advisory committees; Micelle: Other: Site principal investigator; GBT: Other: Site principal investigator; PCORI: Other: site principal investigator; Novartis: Other: co-investigator; Bluebird Bio: Other: co-investigator; Sangamo Therapeutics: Other; Silarus: Other; Celgene: Other; La Jolla Pharmaceuticals: Other; Forma: Other; Imara: Other; National Heart, Lung, and Blood Institute: Other; Health Resources and Services Administration: Other; Centers for Disease Control and Prevention: Other; Seattle Children's Research: Other. Desai:Pfizer, Inc.: Membership on an entity's Board of Directors or advisory committees, Research Funding; GBT, Inc.: Membership on an entity's Board of Directors or advisory committees, Research Funding; Ironwood Pharmaceuticals, Inc.: Membership on an entity's Board of Directors or advisory committees; Rockpointe Continuing Medical Education Company: Consultancy. Lanzkron:GBT: Research Funding; HRSA: Research Funding; Ironwood: Research Funding; NHLBI: Research Funding; PCORI: Research Funding; Pfizer: Research Funding; Pharmacy Times Continuing Education: Honoraria; Prolong: Research Funding. Little:Hemex Health, Inc.: Patents & Royalties: Microfluidic electropheresis (patent, no royalties); BioChip Labs: Patents & Royalties: SCD Biochip (patent, no royalties); GBT: Research Funding; GBT: Membership on an entity's Board of Directors or advisory committees; Bluebird Bio: Research Funding; NHLBI: Research Funding.

scholarly journals Streamline - Retrospective Cohort Study of Relapsed or Refractory (R/R) FLT3-Mutated Acute Myeloid Leukemia (AML): Real-World Treatment, Testing Patterns, and Outcomes

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 30-31
Author(s):  
Amer M. Zeidan ◽  
Adrienne M. Gilligan ◽  
Santosh Gautam ◽  
David L. Grinblatt ◽  
Dina Elsouda ◽  
...  
Keyword(s):  
Tyrosine Kinase ◽  
Board Of Directors ◽  
Real World ◽  
Targeted Therapies ◽  
Research Funding ◽  
Treatment Patterns ◽  
Advisory Committees ◽  
Mutation Status ◽  
Flt3 Mutation ◽  
Flt3 Mutations

Introduction: The past decade in AML research has led to increased emphasis on disease-related mutations and associated targeted therapies. FMS-like tyrosine kinase 3 (FLT3) mutations (FLT3+) are found in about 30% of AML patients and confer a poorer prognosis. Also, the availability of targeted therapies increases the value of testing for FLT3+ AML. A paucity of data exists regarding the real-world FLT3 testing rates in AML patients in both the newly diagnosed and R/R settings. As the treatment landscape for patients with R/R FLT3+ AML expands, it is important to understand how utilization of these therapies and trends in FLT3 testing impact patient outcomes in real-world settings. These updated results examined FLT3 testing trends, treatment patterns, and overall survival (OS) in patients with R/R FLT3+ AML. Patients were grouped based on the FDA approval of the second-generation FLT3 inhibitor gilteritinib (pre-gilteritinib approval vs. post-gilteritinib approval). Methods: This ongoing (01/01/2015-4/17/2020) retrospective study uses electronic medical record data of US patients from a network of 400+ oncology practices maintained in the Definitive Oncology Dataset, including practices affiliated with CancerLinQ. Eligible adult (≥18 years) patients who had a confirmed diagnosis of AML, FLT3+ status, and had ≥1 R/R event between 01/01/2015 and 02/20/2020 were included. FLT3 testing trends included testing performed at initial diagnosis, re-testing performed in the R/R setting, and changes in FLT3 mutation status. Treatment patterns included all systemic anticancer therapies received (including supportive care) for R/R FLT3+ AML. OS was measured from the first R/R event; patients without evidence of death were censored at the last observed visit. Kaplan-Meier analysis was applied to evaluate differences in OS by subsequent hematopoietic stem cell transplant (HSCT) status. Results: Data from 175 patients (50.9% male, n=89) with R/R FLT3+ AML were evaluated (n=124 pre-gilteritinib; n=51 post-gilteritinib). Most patients were White (72.0%; n=126) with a median age of 62 years (range: 20-86) at first R/R event. Median length of follow-up was limited for the post-gilteritinib cohort (9.2 months vs. 15.0 months for pre-gilteritinib, P<0.001). Patients tested for FLT3 mutations at initial AML diagnosis increased from 84.7% (n=105/124) in the pre-gilteritinib cohort to 98.0% (n=50/51) in the post-gilteritinib cohort, and the rates of re-testing increased from 29.5% (n=31/105) to 46.0% (n=23/50) across the two cohorts (Figure 1). After the first R/R event, 18.9% (n=33/175) reported changes in their FLT3 mutation status. Seventy-four different treatment combinations were utilized at the first R/R event. Use of FLT3 tyrosine kinase inhibitors (TKIs), either alone or in combination with chemotherapy, increased from 30.6% (n=38/124) in the pre-gilteritinib cohort to 49.0% (n=25/51) in the post-gilteritinib cohort (Table 1). In the pre-gilteritinib cohort midostaurin (52.6%, n=20/38) and sorafenib (32.4%, n=13/38) were the most commonly prescribed FLT3 TKIs. In the post-gilteritinib cohort, the most common FLT3 TKI was gilteritinib (52.0%, n=13/25). Median OS (95% CI) across all patients at the first R/R event was 13.4 (9.0-17.6) months in the pre-gilteritinib cohort and 12.9 (7.0, NA) months in the post-gilteritinib cohort. Median OS was significantly shorter among patients that did not receive HSCT among all patients and the subset of treated patients (P<0.05 for both values). Median OS for patients with no HSCT was 3.9 months longer in the post-gilteritinib cohort versus the pre-gilteritinib cohort (Table 2). Conclusion: An increase in FLT3 TKI use (both monotherapy and in combination) and a decrease in high-intensity chemotherapy was observed across the two cohorts. In the 18-month post approval period, FLT3 TKI use increased 60% with gilteritinib accounting for more than half of FLT3 TKIs used in the R/R setting. FLT3 re-testing increased by 55% in the R/R setting between the two cohorts; however, re-testing is suboptimal and there is a need to re-test as patients progress. Among pre- and post-treated patients that did not receive HSCT, there was an improvement in OS by almost 4 months, possibly due to increased use of targeted FLT3 TKIs. With recent approval of these targeted therapies, it will be important to continue to monitor FLT3 testing, treatment patterns, and clinical outcomes. Disclosures Zeidan: Celgene / BMS: Consultancy, Honoraria, Research Funding; Daiichi Sankyo: Consultancy, Honoraria; Agios: Consultancy, Honoraria; Ionis: Consultancy, Honoraria; BeyondSpring: Consultancy, Honoraria; Abbvie: Consultancy, Honoraria, Research Funding; Otsuka: Consultancy, Honoraria; Pfizer: Consultancy, Honoraria, Research Funding; CCITLA: Other; Astex: Research Funding; Cardinal Health: Consultancy, Honoraria; Trovagene: Consultancy, Honoraria, Research Funding; Seattle Genetics: Consultancy, Honoraria; Leukemia and Lymphoma Society: Other; Boehringer-Ingelheim: Consultancy, Honoraria, Research Funding; Jazz: Consultancy, Honoraria; Novartis: Consultancy, Honoraria, Research Funding; Taiho: Consultancy, Honoraria; Aprea: Research Funding; ADC Therapeutics: Research Funding; Takeda: Consultancy, Honoraria, Research Funding; Epizyme: Consultancy, Honoraria; MedImmune/Astrazeneca: Research Funding; Acceleron: Consultancy, Honoraria; Astellas: Consultancy, Honoraria; Incyte: Consultancy, Honoraria, Research Funding; Cardiff Oncology: Consultancy, Honoraria, Other. Gilligan:ConcertAI: Current Employment. Grinblatt:Astellas: Membership on an entity's Board of Directors or advisory committees; Bristol Myers Squibb: Membership on an entity's Board of Directors or advisory committees; Alexion: Speakers Bureau. Elsouda:Astellas: Current Employment. Sullivan:Astellas Pharma: Current Employment. Pandya:Astellas Pharma, Inc.: Current Employment.

scholarly journals Real-World Use of FLT3-TKIs in R/R FLT3-Mutated AML in the United States

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 24-25
Author(s):  
David L. Grinblatt ◽  
Bhavik J. Pandya ◽  
Wei Han ◽  
Loretta Sullivan ◽  
Qi Feng ◽  
...  
Keyword(s):  
Tyrosine Kinase ◽  
Board Of Directors ◽  
Real World ◽  
Treatment Duration ◽  
The United States ◽  
Treatment Patterns ◽  
Prescription Data ◽  
Concomitant Chemotherapy ◽  
Advisory Committees ◽  
Specialty Pharmacy

Background: Targeted inhibition of the FLT3 tyrosine kinase is a treatment strategy for patients with relapsed or refractory, FLT3 mutation-positive acute myeloid leukemia (R/R FLT3mut+ AML). While the multikinase inhibitors, sorafenib and midostaurin, were not approved specifically for R/R AML, they are used off-label as treatment. Since FDA approval on 11/28/2018, gilteritinib has become the first globally approved targeted therapy for the treatment of R/R FLT3mut+ AML in adults. This study aims to understand the treatment landscape for R/R FLT3mut+ AML patients following the introduction of a new therapeutic agent. Aim/Objective: To describe the characteristics and treatment patterns of patients initiating FLT3 tyrosine kinase inhibitors (TKIs) for R/R FLT3mut+ AML in the real-world setting. Methods: This US-based retrospective cohort study was conducted using two separate closed-claims databases (MarketScan, 1/1/2007-2/29/2020; IQVIA, 1/1/2006-4/30/2020) and a specialty-pharmacy claims database (ProMetrics, 12/6/2018-3/12/2020). Patients ≥18 years of age, with an AML diagnosis and newly initiated FLT3-TKI (ie, gilteritinib, midostaurin, or sorafenib) therapy for R/R diagnosis or episode ("R/R AML") on or after gilteritinib market availability were eligible for inclusion. Patients were indexed on the first newly initiated FLT3-TKI for R/R AML observed on or after 12/1/2018. Patients' baseline clinical and demographic characteristics, AML-related treatments preceding FLT3-TKI initiation for R/R AML, and concomitant therapies (defined as any AML-related treatment overlapping with FLT3-TKI use after R/R AML) were identified. Because individual patients could have received more than one FLT3-TKI during the study period, treatment sequencing was analyzed by FLT3-TKI-containing regimens. Given the larger sample size available within the ProMetrics Specialty Pharmacy database, gilteritinib dose patterns and treatment duration were analyzed using de-identified dispensed prescription data from a separate gilteritinib-only cohort for greater precision. Descriptive statistics were used to evaluate all study outcomes, except for treatment duration, which was estimated using Kaplan-Meier life table analysis. Results: A total of 52 and 51 patients initiating FLT3-TKIs for R/R AML were identified in the MarketScan and IQVIA databases, respectively. The mean age of patients was 53.1 (MarketScan) and 52.3 years (IQVIA); patients had a mean ± SD Quan-Charlson Comorbidity Index of 3.6 ± 2.4 (MarketScan) and 3.7 ± 2.2 (IQVIA). Most patients treated with FLT3-TKIs for R/R AML during the study period were first initiated on gilteritinib (MarketScan: n=35 [67.3%], IQVIA: n=36 [70.6%]); sorafenib was the second most common FLT3-TKI (MarketScan: n=9 [17.3%], IQVIA: n=8 [15.7%]), followed by midostaurin (MarketScan: n=8 [15.4%], IQVIA: n=7 [13.7%]). During the study period, 61 and 55 FLT3-TKI-containing regimens among 52 and 51 R/R AML patients were identified across the MarketScan and IQVIA databases, respectively. Most FLT3-TKI-containing regimens for R/R AML included gilteritinib (MarketScan: n=43 [70.5%], IQVIA: n=38 [69.1%]). Prior exposure to other FLT3-TKIs for new onset and/or R/R AML indications was observed in up to 63% of gilteritinib-, 56% of sorafenib-, and 13% of midostaurin-containing regimens (Table). Following onset of R/R AML, gilteritinib was given without concomitant chemotherapy in 47% (Marketscan: 20/43) to 55% (IQVIA: 21/38) of gilteritinib regimens; in a smaller sample of sorafenib regimens, sorafenib was given without concomitant chemotherapy in up to 56% (IQVIA: 5/9) of regimens. Of 748 patients identified in the ProMetrics database with gilteritinib claims, 714 (95%) patients were initiated on a 120-mg daily dose, with less than 5% (n=34) incurring a downward dosage titration. The median treatment duration was 150 days (95% CI: 129-172 days). Conclusions: Among FLT3-TKI-containing regimens initiated for R/R AML after gilteritinib market approval, close to 70% included gilteritinib, an agent approved for R/R FLT3mut+ AML. Sample sizes for midostaurin- and sorafenib-containing regimens limited characterization of treatment patterns. Gilteritinib was given without concomitant chemotherapy in approximately 50% of regimens prescribed; the dose and treatment durations were similar to those previously reported by the ADMIRAL trial. Disclosures Grinblatt: Astellas: Membership on an entity's Board of Directors or advisory committees; Bristol Myers Squibb: Membership on an entity's Board of Directors or advisory committees; Alexion: Speakers Bureau. Pandya:Astellas Pharma, Inc.: Current Employment. Sullivan:Astellas Pharma: Current Employment. Feng:Astellas: Current Employment. Hedlund:Astellas: Current Employment.

scholarly journals Comparative Effectiveness of Triplets Containing Bortezomib (V), Carfilzomib (K), or Ixazomib (I) Combined with a Lenalidomide and Dexamethasone Backbone (Rd) in Patients with Relapsed/Refractory Multiple Myeloma (RRMM) in Routine Care in the United States (US)

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 1827-1827
Author(s):  
Ajai Chari ◽  
Dorothy Romanus ◽  
Aditya Raju ◽  
Lauren Cain ◽  
Marlo Blazer ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Board Of Directors ◽  
Real World ◽  
Comparative Effectiveness ◽  
Research Funding ◽  
Routine Care ◽  
Advisory Committees ◽  
The Us ◽  
Frailty Score

BACKGROUND: Proteasome inhibitors (PIs) combined with an Rd backbone constitute commonly used regimens in RRMM in the US. Lack of data from head-to-head clinical trials comparing the efficacy of these PI-Rd triplets and the extent to which efficacy translates to benefit in routine care highlight the need for real-world evidence. We conducted a comparative effectiveness analysis of time to next therapy (TTNT) of these PI-Rd triplet combinations in a real-world representative cohort of RRMM patients. METHODS: In this retrospective cohort study, RRMM patients who initiated an index regimen with IRd, KRd, or VRd in ≥2nd line of therapy (LOT 2+) were followed between 1/2014-9/2018 in Optum's deidentified national electronic health records database. This database captures information on all patients treated by >140,000 providers across 50 states in the US. It is representative of the US general population. Baseline data included: a modified frailty score utilizing age and Charlson Comorbidity Index1, ECOG performance status, CRAB symptoms, ISS stage, cytogenetic risk (high: del[17p], t[4;14], or t[14;16]), history of: cardiovascular disease, peripheral neuropathy, stem-cell transplant, and uncontrolled hypertension, prior PI and/or IMID exposure and refractory status, time from diagnosis to start of index LOT, time from start of frontline therapy to start of LOT 2, and treatment-free interval prior to index LOT. Duration of therapy (DOT) and TTNT were estimated using Kaplan-Meier methods and compared using stratified (by LOT) ± baseline-adjusted Cox proportional hazard models with a repeated measures analysis of pt-LOTs with robust sandwich estimators to account for inclusion of patients in multiple LOTs. The main analysis was repeated in subgroups a priori defined by frailty score, cytogenetic risk, prior PI and/or IMID exposure and LOT. Observations were censored at time of loss to follow up/end of study period (9/30/2018). RESULTS: Overall, 650 patients with 733 pt-LOTs (IRd, n=168; KRd, n=208; VRd, n=357) were included; 20% of the RRMM patients in this cohort were treated at academic centers. In the I-/K-/V-Rd groups, respectively, median age was 71/65/71 years and 42/22/38% patients were ≥75 years old; 39/55/46% and 28/14/29% patients had a modified frailty score of intermediate and frail, respectively; 71/88/80% had a symptomatic relapse with at least 1 CRAB symptom; 20/28/13% patients had high cytogenetic risk; 69/72/90% patients were treated in 2-3 LOT, and 63/70/32% patients had prior exposure to a PI+IMID (P<0.05 for all). The median follow-up was 14.4 months. Unadjusted median DOT of index LOT was 12.3/7.2/10.0 months, with a lower risk of discontinuation of the oral PI component for I in comparison to both K and V (HR[I vs K] of 0.66 and HR[I vs V] of 0.68) and R component in the IRd vs KRd and VRd groups (HR[I vs K] of 0.64 and HR[I vs V] of 0.75) (P<0.05 for all). Unadjusted median TTNT was 12.7/8.6/14.2 months for I-/K-/V-Rd in LOT ≥2; with HR for TTNT of 0.76 (IRd vs KRd; P<0.05); 1.01 (IRd vs VRd; P=0.96); 1.33 (KRd vs VRd; P<0.05). The adjusted HRs for TTNT I-/K-/V-Rd in LOT ≥2 were 0.82 (IRd vs KRd); 0.93 (IRd vs VRd); 1.13 (KRd vs VRd; P>0.05 for all), and were comparable between all 3 groups. A significant TTNT benefit for IRd vs KRd was observed among patients with high cytogenetic risk (HR: 0.47) and with intermediate frailtymodified score (HR: 0.60; P<0.05 for all). CONCLUSIONS: In this real-world representative RRMM cohort, in unadjusted analyses, the IRd group had a longer DOT of the PI and R components and a longer TTNT in comparison with KRd. After adjusting for baseline confounders to separate the treatment effect, the I/K/V-Rd triplet combinations were comparable in TTNT, but IRd was significantly better than KRd in patients with high-risk cytogenetics and with an intermediate frailty score. Residual confounding is always a potential limitation of any non-randomized study. Conversely, the results of randomized, clinical trials are limited in generalizability as 40-60% of patients are excluded due to inability to meet eligibility criteria. This is especially pertinent in an elderly RRMM population as demonstrated in the real-world effectiveness results in this study. Ref: 1. Palumbo, et al. Blood. 2015;125:2068-2074 Figure Disclosures Chari: Sanofi: Membership on an entity's Board of Directors or advisory committees; Seattle Genetics: Membership on an entity's Board of Directors or advisory committees, Research Funding; Pharmacyclics: Research Funding; Oncoceutics: Research Funding; Novartis Pharmaceuticals: Research Funding; GlaxoSmithKline: Research Funding; Array Biopharma: Research Funding; Karyopharm: Consultancy, Membership on an entity's Board of Directors or advisory committees; Janssen: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Millennium/Takeda: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Celgene: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Bristol-Myers Squibb: Consultancy; Amgen: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding. Romanus:Takeda: Employment. Raju:Millennium Pharmaceuticals, Inc., a wholly owned subsidiary of Takeda Pharmaceutical Company Limited: Other: Xcenda received funding from Takeda to complete this analyses., Research Funding; Xcenda, LLC: Employment. Cain:Takeda: Employment. Blazer:Xcenda: Employment; Takeda: Other: Xcenda received funding from Takeda for completion of this analysis, Research Funding. Farrelly:Xcenda: Employment; Takeda: Other: Xcenda received funding from Takeda for completion of this analysis, Research Funding. Stull:Takeda: Employment. Ailawadhi:Pharmacyclics: Research Funding; Cellectar: Research Funding; Celgene: Consultancy; Takeda: Consultancy; Janssen: Consultancy, Research Funding; Amgen: Consultancy, Research Funding.

scholarly journals Real-World Treatment Patterns and Transfusion Burden Among US Patients with Newly Diagnosed Myelodysplastic Syndromes (MDS)

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 3499-3499
Author(s):  
Sudipto Mukherjee ◽  
S. Lane Slabaugh ◽  
Ronda Copher ◽  
Jonathan Johnson ◽  
Paul Buzinec ◽  
...  
Keyword(s):  
Board Of Directors ◽  
Treatment Duration ◽  
Research Funding ◽  
Index Date ◽  
Treatment Patterns ◽  
Newly Diagnosed ◽  
Advisory Committees ◽  
Median Treatment ◽  
Medical Claims ◽  
Median Treatment Duration

Introduction: Symptomatic anemia transfusion support represents a significant problem for patients with MDS. The principal strategy in managing these patients remains supportive care with erythropoiesis-stimulating agents (ESAs) and red blood cell (RBC) transfusions. However, there is paucity of real-world data regarding patterns of use of ESA and other therapies, and its impact on transfusion needs in these patients early in their clinical course. To address this question, we performed a retrospective claims-based study to characterize treatment patterns and transfusion burden in patients with MDS. Methods: In this retrospective claims analysis of a large national health insurance plan, all patients aged ≥ 18 years with newly diagnosed MDS (≥ 2 medical claims with an International Classification of Diseases, 9th or 10th Revision [ICD-9 or ICD-10] diagnosis codes ≥ 30 days apart) identified between January 2012 and May 2018 were included. Index date was defined as date of first diagnosis. Continuous health plan enrollment for ≥ 6 months pre- and 3 months post-index date was required. RBC transfusion status was evaluated during the 16 weeks prior to first diagnosis as well as 16 weeks prior to and immediately following line of therapy (LOT) 1 and LOT2. Transfusion burden categories were adapted and modified from the proposed International Working Group 2018 revised criteria (Platzbecker U, et al., Blood. 2019;133(10):1096-1107). Categories included transfusion independence (TI), defined as patients receiving no transfusions during the observation period; low (LTB), moderate (MTB), and high transfusion burden (HTB) were defined by patient's having 1-3, 4-7, and ≥ 8 unique dates for a transfusion during the observation periods, respectively. Therapies in each LOT were captured using pharmacy and medical claims data. The end of a LOT was defined after ≥ 60-day gap in therapy, a claim for any new or additional MDS therapy, or patient death; LOT durations are described for non-censored patients. Results: Among the 3,587 patients identified (mean age = 74.9 years, 44.3% female), transfusion burden during 16 weeks prior to index was: 78.8% TI, 19.2% LTB, 1.9% MTB, and 0.2% HTB. Among the 1,935 patients who received LOT1, transfusion burden in the 16 weeks preceding LOT1 was: 57.0% TI, 36.3% LTB, 5.6% MTB, and 1.1% HTB. The top 5 regimens in LOT1 were ESA monotherapy (61.9%), hypomethylating agent (HMA) monotherapy (19.2%), white blood cell growth factor (WBCGF) monotherapy (3.5%), immunomodulators (3.3%), and HMA + ESA (2.7%) (Figure). Of 824 patients who received LOT2, transfusion burden prior to LOT2 was: 49.4% TI, 28.6% LTB, 16.5% MTB, and 5.5% HTB. The top 5 regimens in LOT2 were ESA monotherapy (44.2%), HMA monotherapy (12.1%), HMA + ESA (9.2%), WBCGF + ESA (6.9%), and WBCGF monotherapy (6.4%) (Figure). In LOT1, the median treatment duration for ESA monotherapy was 2.8 months (mean = 5.2 months, standard deviation [SD] = 6.8) whereas in LOT2, the median treatment duration was 2.0 months (mean = 3.7 months, SD = 5.0). Amongst patients receiving ESA monotherapy as LOT2, 90.4% had prior ESA monotherapy as LOT1. In patients treated with HMA monotherapy in LOT1 that also experienced a LOT2, 39.1% moved on to a variety of LOT2 regimens (Table), while in 15.5% ESAs were combined with HMA (Table). Conclusions: Our results show that at the time of diagnosis, 20% of MDS patients were transfusion dependent, but up to 50% require treatment. The high rate of ESA use is likely due to anemia-related symptoms. In those treated with ESA monotherapy, approximately 50% have a LOT1 duration that is &lt; 3 months, and interestingly, the majority of patients restart a second LOT with an ESA as the most common regimen. Additional analyses are necessary to determine whether this indicates a switch in ESA agent, escalation in dose of prior ESA, treatment cycling due to elevated hemoglobin, or other reasons. Considering the short median treatment duration and worsening of transfusion dependency beyond first line, there remains a high unmet need for MDS therapy that more effectively slows the progression of transfusion dependence. Disclosures Mukherjee: Bristol-Myers Squibb: Speakers Bureau; Takeda: Membership on an entity's Board of Directors or advisory committees; Pfizer: Honoraria; Novartis: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Projects in Knowledge: Honoraria; Celgene Corporation: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Partnership for Health Analytic Research, LLC (PHAR, LLC): Consultancy; McGraw Hill Hematology Oncology Board Review: Other: Editor. Slabaugh:Celgene Corporation: Employment, Equity Ownership. Copher:Celgene Corporation: Employment. Johnson:Optum: Employment; Celgene Corporation: Consultancy. Buzinec:Optum: Employment. Mearns:Celgene Corporation: Employment.

Treatment Patterns from 2009 to 2015 in Patients with Newly Diagnosed Multiple Myeloma in the United States: A Report from the Connect® MM Registry

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 4489-4489 ◽  
Author(s):  
Robert M. Rifkin ◽  
Rafat Abonour ◽  
Brian G.M. Durie ◽  
Cristina J. Gasparetto ◽  
Sundar Jagannath ◽  
...  
Keyword(s):  
Multiple Myeloma ◽  
Board Of Directors ◽  
The United States ◽  
Treatment Patterns ◽  
Second Line ◽  
Newly Diagnosed ◽  
First Line ◽  
Advisory Committees ◽  
Frequency Of Use ◽  
The Us

Abstract Introduction: Between 2009 and 2015, use of novel therapies (immunomodulating drugs and proteasome inhibitors) in multiple myeloma (MM) increased. Regimens initiated during this time frame may help project near-term future treatment patterns. Connect® MM is the first and largest prospective, observational, US-based, multicenter disease registry designed to characterize treatment patterns and outcomes for patients (pts) with newly diagnosed MM (NDMM). Pts with NDMM were enrolled in 2 sequential cohorts from Sep 2009 to Apr 2016. This noninterventional registry did not prescribe or limit therapy choices. Study sites represented all census regions, with 89% and 11% split between community and academic sites, respectively. This allowed a reasonable generalizability to patterns for the US. Methods: Connect® MM enrollment was initiated in Sep 2009 at 250 community and academic sites. Pts were enrolled within 2 months of diagnosis. Cohort 1 enrolled 1493 NDMM pts from Sep 2009 to Dec 2011, and Cohort 2 enrolled 1518 NDMM pts from Dec 2012 to Apr 2016. Data were collected at a baseline visit and quarterly visits thereafter until death or discontinuation. The current analysis was conducted for the population of treated pts (N=2848) as of May 2016. This study examined recorded treatment choice of first-line regimen, maintenance therapy, and second-line regimen in 6-month intervals. Trends in regimens were graphically represented using "Tepee" plots (Srinivasan, Shankar. Resource Tepee. Patent US 7,495,673 B1. 24 Feb 2009). Briefly, all pts who initiated treatment during each 6-month interval are represented horizontally, with each horizontal line indicating 100% of all treatment used in that period. The regimens are represented by gray shading with wider bands signifying the more frequently used regimens at each time interval. Results: Median follow‐up for all pts was 39.3 months (0.03‐78.4) in Cohort 1 and 15.4 months (0.2-40.1) in Cohort 2. For the treated population, the median age was 67 years (range 24‐94), 58% were male, 83% were white, and 38% of those reporting International Staging System stage had stage III MM. By US geographical region, 329 (11.6%) pts were from the Northeast, 1036 (36.4%) from the Midwest, 1117 (39.2%) from the South, 360 (12.6%) from the West, 4 (0.1%) from Puerto Rico, and 2 missing (0.05%). Most pts (2285; 80.2%) were from community sites, and 397 (13.9%) were from academic sites with the remaining from government sites. A total of 1416 (47.4%) reported an intent to transplant (stem cell transplant [SCT]) at the initiation of therapy. A total of 666 (25.8%) have progressed and entered second line. Tepee plots of treatment patterns for start of induction for those pts with and without SCT intent are provided in Figure 1A and 1B, respectively. The year 2012 does not feature in these induction plots, as this period corresponds to a time when pts were not enrolled-Cohort 1 had been completed and Cohort 2 had not yet opened. The 4 most common induction regimens for SCT intent, from left to right, in order of decreasing frequency of use, were lenalidomide (R), bortezomib (V), dexamethasone (D) combined (RVD); VD; cyclophosphamide plus VD (CyBorD); and RD. The 5 most common induction regimens for those without SCT intent, from left to right, in order of decreasing frequency of use, were VD, RD, RVD, CyBorD, and V. Triplet therapy in first-line induction pts increased in frequency from 2009 to 2014. The 4 most frequent maintenance regimens for those with SCT intent were R monotherapy, V monotherapy, RD, and RVD. The 4 most common maintenance regimens for pts who did not intend to receive SCT were R monotherapy, RD, VD, and V monotherapy. The most prevalent regimens in the second line were VD, RD, V, and RVD. Additional graphs including treatment patterns by age group (≤ 70 vs > 70 years) and maintenance by conduct of first-line SCT will be presented. Conclusions: Our work utilizes Tepee plots to outline induction and maintenance treatment patterns over time, for both SCT and non-SCT intent pts, using the largest, prospective, noninterventional registry study in the US. Triplet therapy use increased in the time period studied, with RVD being the most frequently used triplet for pts with or without SCT intent. The most common maintenance regimens included R as monotherapy or in combination. Disclosures Rifkin: Takeda: Membership on an entity's Board of Directors or advisory committees; Celgene: Membership on an entity's Board of Directors or advisory committees; Amgen/ONYX: Membership on an entity's Board of Directors or advisory committees. Abonour:Celgene: Membership on an entity's Board of Directors or advisory committees. Durie:Amgen: Consultancy; Janssen: Consultancy; Takeda: Consultancy. Gasparetto:Celgene: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Takeda: Consultancy; Janssen: Honoraria; Bristol-Myers Squibb: Honoraria. Jagannath:Bristol-Myers Squibb: Consultancy; Celgene: Consultancy; Merck: Consultancy. Terebelo:Celgene: Membership on an entity's Board of Directors or advisory committees. Toomey:Celgene: Consultancy. Kitali:Celgene: Employment, Equity Ownership. Zafar:Celgene: Employment. Srinivasan:Celgene: Employment; Individual Patent: Patents & Royalties: US7,495,673B1 Used for MM-Connect Treatment Patterns Abstract.. Hardin:Celgene: Consultancy, Membership on an entity's Board of Directors or advisory committees.

scholarly journals Treatment Patterns and Outcomes of Relapsed/Refractory Peripheral T-Cell Lymphoma (RR-PTCL) Patients Treated in the Community Oncology Setting

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 1656-1656
Author(s):  
Chadi Nabhan ◽  
Jalyna Laney ◽  
Joseph Feliciano ◽  
Choo Hyung Lee ◽  
Andrew J Klink
Keyword(s):  
Clinical Trials ◽  
Real World ◽  
Patient Characteristics ◽  
Treatment Patterns ◽  
Novel Therapies ◽  
Employment Equity ◽  
The Real ◽  
Community Oncology ◽  
The Us ◽  
Equity Ownership

Abstract Introduction: The majority of PTCL cases are pathologically heterogeneous, aggressive and chemorefractory; nearly 70% of patients who undergo induction chemotherapy develop RR disease. Data on how RR-PTCL patients are managed outside of clinical trials and academic settings have not been adequately studied. Understanding how PTCL patients are treated in the real world allows better design of future clinical trials and refined development of novel therapies. This study aimed to describe contemporary treatment patterns, clinical characteristics, and overall survival (OS) among RR-PTCL patients treated in the US community oncology setting. Methods: We conducted a retrospective, observational cohort study of RR-PTCL patients treated between 1/1/2010-12/31/2016. Data were collected from patient medical records abstracted from 30 community oncologists across all geographic areas in the US (38% South, 22% West, 7% Midwest, 26% Northeast, and 7% unknown). Patient characteristics, treatment patterns, adverse events (AEs), physician-reported response to first- and second-line (1L, 2L) treatments, and date of death were collected. Descriptive statistics were calculated to summarize patient characteristics and study outcomes. Median (95% confidence interval [CI]) OS from RR disease was calculated by the Kaplan-Meier method. Results: We analyzed 200 RR PTCL patients (71% received CHOP, 20% received CHOEP, and 9% received another regimen as 1L systemic therapy). The majority (58%) were PTCL not otherwise specified (NOS), 61% were males, and median age was 62 years (range: 20-90) (Table). Overall response rate (ORR) to 1L treatment was 80% (CR, 54%; PR, 26%), and ORR to 2L treatment was 55% (CR, 31%, PR, 24%). Among all PTCL patients and among those with PTCL NOS, the OS (95% CI) from RR disease was 13.0 months (95% 7.0-not estimable) and 9.0 months (95% 6.0-not estimable), respectively (Figure). Median time to RR disease from 1L initiation was 12.9 months. Median durations of 1L and 2L treatments were 4.4 months and 3.7 months, respectively. Among those who continued to 2L therapy, 27% received brentuximab vedotin (2% in combination), 19% received ICE (carboplatin/cisplatin, ifosfamide, etoposide), 13% received romidepsin (2% in combination with pralatrexate), 10% received pralatrexate monotherapy, 7% received GEMOX (gemcitabine, oxaliplatin), and the rest (22%) received other 2L regimens or an unknown regimen (2%). After 1L initiation, only 14/200 (7%) received hematopoietic stem cell transplant (HSCT) and 28/151 (19%) after 2L. AEs resulting in a dose reduction, discontinuation, or hospitalization during 1L treatment and occurring among ≥10% included anemia (39%), thrombocytopenia (27%), neutropenia (23%), mouth sores (16%), diarrhea (15%), neuropathy (14%), febrile neutropenia (13%), and vomiting (10%). Conclusions: While response to 1L treatment in this cohort was high (80%), all developed RR disease (or died), for whom the prognosis remains poor. Very few patients underwent HSCT in any line of therapy. Our data demonstrate the unmet medical need for RR-PTCL patients and the need for novel therapies in the 1L setting. It also underscores the need to better understand factors leading to treatment selection and optimal treatment sequencing in the real world. Disclosures Nabhan: Cardinal Health: Employment, Equity Ownership. Laney:Cardinal Health: Employment, Equity Ownership. Feliciano:Seattle Genetics: Employment, Equity Ownership. Lee:Cardinal Health: Employment. Klink:Cardinal Health: Employment, Equity Ownership.

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