Real-World Persistence with and Adherence to Emicizumab Prophylaxis in Persons with Hemophilia a: A Secondary Claims Database Analysis

Introduction: Emicizumab is a subcutaneously administered, humanized bispecific monoclonal antibody, approved for routine prophylaxis in persons with hemophilia A (PwHA) with or without factor VIII inhibitors. Little is known about patterns of emicizumab use in clinical practice. We aimed to evaluate real-world persistence with and adherence to emicizumab treatment for PwHA on prophylaxis. Methods: This retrospective study used de-duplicated commercial insurance claims data from IBM® MarketScan® Commercial Research and IQVIA PharMetrics® Plus databases from 16 November 2017 to 31 December 2019. Individuals included met the following criteria: 1) evidence of emicizumab use (at least two prescription fills) and 2) continuous enrollment for at least three months pre-emicizumab initiation. Individuals were followed until the end of study period or continuous enrollment. Persistence was defined as the proportion of individuals continuing treatment with emicizumab during the study period. Time to discontinuation and proportion of individuals who restarted emicizumab prophylaxis were reported for patients who discontinued emicizumab. Discontinuation was defined as 60 days without a prescription fill. Adherence was measured over the post-index persistence period using the proportion of days covered (PDC), and the proportion of individuals adherent at ≥80% PDC threshold was reported. Results: We identified 328 unique individuals who met the inclusion criteria. All patients were male (100%); the average age was 23 years (standard deviation [SD] ±16; range=1-64); and the average follow-up post-index was eight months (245±147 days). The vast majority of individuals (92%) were persistent with emicizumab prophylaxis during the study period. Among those who discontinued emicizumab (n=25), the average time to discontinuation was approximately three months (84±124 days); 40% restarted emicizumab prophylaxis after discontinuation, with an average time to restart of approximately four months (126±56 days). During the period for which individuals were persistent to treatment, adherence to emicizumab was high (81%); with 70% individuals meeting the ≥80% PDC threshold. Among those with at least one-year post-index enrollment (n=48), adherence during the first year was also high (85%), with 77% individuals adherent at the ≥80% PDC threshold. Conclusions: In this study, the vast majority of individuals treated with emicizumab had high rates of adherence and persistence to treatment. This is one of the first studies to report real-world persistence with and adherence to emicizumab prophylaxis in PwHA. Future evaluations should examine the association of persistence and adherence with health outcomes in this population. Disclosures Mahajerin: Spark Therapeutics, Alexion, Genentech, Inc.: Speakers Bureau. Khairnar:Genentech, Inc.: Current Employment, Current equity holder in publicly-traded company; F Hoffmann-La Roche Ltd: Current equity holder in publicly-traded company. Meyer:F. Hoffmann-La Roche Ltd: Current equity holder in publicly-traded company; Genentech, Inc.: Current Employment, Current equity holder in publicly-traded company. Abbass:Genentech, Inc.: Current Employment, Current equity holder in publicly-traded company; F Hoffmann-La Roche Ltd: Current equity holder in publicly-traded company. Wang:Genentech, Inc.: Current Employment, Current equity holder in publicly-traded company; F Hoffmann-La Roche Ltd: Current equity holder in publicly-traded company; Acumen, LLC: Ended employment in the past 24 months. Lee:Genentech, Inc.: Current Employment; F. Hoffmann-La Roche Ltd: Current equity holder in publicly-traded company. Tzeng:Genentech, Inc.: Current Employment, Current equity holder in publicly-traded company; F Hoffmann-La Roche Ltd: Current equity holder in publicly-traded company. Raimundo:Genentech, Inc.: Current Employment, Current equity holder in publicly-traded company; F Hoffmann-La Roche Ltd: Current equity holder in publicly-traded company.

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Real-World Clinical Management of Patients with Congenital Hemophilia and Inhibitors: Interim Analysis of the FEIBA Global Outcome Study (FEIBA GO)

Blood ◽

10.1182/blood-2020-139298 ◽

2020 ◽

Vol 136 (Supplement 1) ◽

pp. 23-24

Author(s):

Cedric Hermans ◽

Claude Négrier ◽

Pål A Holme ◽

Carmen Escuriola ◽

Ana R Cid ◽

...

Keyword(s):

Clinical Practice ◽

Real World ◽

Interim Analysis ◽

Research Funding ◽

Hemophilia A ◽

Outcome Study ◽

Publicly Traded ◽

On Demand ◽

The Past

Introduction and Objective: Development of inhibitory antibodies is a major complication of factor replacement therapy in patients with congenital hemophilia A or B. The FEIBA Global Outcome study (FEIBA GO) assessed the long-term safety and real-world effectiveness of activated prothrombin complex concentrate (aPCC; Baxalta US Inc, a Takeda company, Lexington, MA, USA) as prophylaxis or on-demand treatment in patients with congenital hemophilia A or B with inhibitors (PwHI) across different clinical settings. Methods: FEIBA GO (EUPAS6691) is a post-authorization, prospective, observational, multicenter cohort study. Male PwHI diagnosed before study entry and prescribed treatment with aPCC as part of routine clinical practice were followed over 4 years; treatment regimens were prescribed at the physician's discretion. Ethics approval and patient consent were obtained. These data report on an interim analysis (data cutoff, May 16, 2019) on the annualized bleeding rate (ABR) calculated per patient-year of follow-up and aPCC consumption in patients receiving aPCC prophylaxis. Results: Enrollment was started on September 3, 2014 and completed on December 31, 2017. Fifty-one enrolled PwHI have received aPCC prophylaxis (n=37) or on-demand (n=14) treatment from 26 sites in 11 countries (hemophilia A: n=50, hemophilia B: n=1; median [range] age at baseline: 17 [2-71] years). As of May 2019, mean±SD (median, range) ABR was 7.2±8.2 (5.7, 0-30) per patient-year for the 14 patients with ≥2 to <4 years' study follow-up (mean±SD: 3.0±0.6 years). In the 3 patients with ≥4 years of study follow-up (mean±SD: 4.0±0.03 years), mean±SD (median, range) ABR was 14.7±25.3 (0.2, 0-44) per patient-year. Patients received a variety of prophylaxis regimens; data on dosages per infusion and number of weekly infusions administered are provided in the Table. Conclusions: This interim analysis describes the real-world use and effectiveness of aPCC prophylaxis and supports previous data on the prevention of bleeding events in PwHI. The data also highlight the variety of dosing regimens reflecting the real-world use of aPCC prophylaxis in clinical practice. Although patient numbers were small, these data suggest that increased weekly doses are used in patients with more severe bleeding phenotypes. Figure 1 Disclosures Hermans: Kedrion:Speakers Bureau;Octapharma:Consultancy, Speakers Bureau;Roche:Consultancy, Speakers Bureau;Pfizer:Consultancy, Research Funding, Speakers Bureau;Novo Nordisk:Consultancy, Speakers Bureau;LFB:Consultancy, Speakers Bureau;Shire, a Takeda company:Consultancy, Research Funding, Speakers Bureau;Sobi:Consultancy, Research Funding, Speakers Bureau;Bayer:Consultancy, Research Funding, Speakers Bureau;EAHAD:Other;WFH:Other;CSL Behring:Consultancy, Speakers Bureau;CAF-DCF:Consultancy, Speakers Bureau;Biogen:Consultancy, Speakers Bureau.Négrier:CSL Behring, Octapharma, Shire/Takeda, Sobi:Research Funding;CSL, F. Hoffmann-La Roche Ltd, Sobi:Other: Travel support;Bayer, Biomarin, CSL Behring, Freeline, LFB, Novo Nordisk, Octapharma, Pfizer, F. Hoffmann-La Roche Ltd, Sanofi, Shire/Takeda, Sobi, Spark:Consultancy.Holme:Sobi:Honoraria, Research Funding;Bayer:Honoraria, Research Funding;CSL Behring:Honoraria;Novo Nordisk:Honoraria;Octapharma:Research Funding;Pfizer:Honoraria, Research Funding;Shire, a Takeda company:Honoraria, Research Funding.Escuriola:Grifols:Honoraria;CSL Behring:Consultancy, Honoraria, Research Funding;Biotest:Honoraria, Research Funding;Biomarin:Honoraria;Bayer:Honoraria;Kedrion:Honoraria;Shire, a Takeda company:Honoraria;Octapharma:Consultancy, Honoraria, Research Funding;Novo Nordisk:Consultancy, Honoraria;Roche:Honoraria;Sobi:Honoraria, Research Funding.Cid:Roche:Consultancy, Honoraria;Sobi:Consultancy, Honoraria;Novo Nordisk:Honoraria;Shire, a Takeda company:Honoraria.Kemenyash:Takeda Pharmaceutical International AG:Current Employment, Current equity holder in publicly-traded company.Botha:Takeda Pharmaceutical International AG:Current Employment, Current equity holder in publicly-traded company.Cano-Garcia:Sanofi Genzyme:Current Employment, Current equity holder in publicly-traded company;Shire, a Takeda company:Divested equity in a private or publicly-traded company in the past 24 months, Ended employment in the past 24 months.Windyga:Sanofi:Honoraria, Research Funding;Alexion:Honoraria, Research Funding;Alnylam:Honoraria, Research Funding;Baxalta/Shire, a Takeda company:Honoraria, Research Funding;Bayer:Honoraria, Research Funding;Siemens:Honoraria, Research Funding;Sobi:Honoraria, Research Funding;Werfen:Honoraria, Research Funding;CSL Behring:Honoraria, Research Funding;Ferring Pharmaceuticals:Honoraria, Research Funding;Novo Nordisk:Honoraria, Research Funding;Octapharma:Honoraria, Research Funding;Rigel Pharmaceuticals:Honoraria, Research Funding;Roche:Honoraria, Research Funding.

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Cardiovascular risk factors associated with acute myocardial infarction and stroke in the MADIABETES cohort

Scientific Reports ◽

10.1038/s41598-021-94121-8 ◽

2021 ◽

Vol 11 (1) ◽

Author(s):

M. A. Salinero-Fort ◽

F. J. San Andrés-Rebollo ◽

J. Cárdenas-Valladolid ◽

M. Méndez-Bailón ◽

R. M. Chico-Moraleja ◽

...

Keyword(s):

Atrial Fibrillation ◽

Blood Pressure ◽

Antihypertensive Drugs ◽

Hdl Cholesterol ◽

First Year ◽

Backward Elimination ◽

One Year ◽

Time Varying Covariates

AbstractWe aimed to develop two models to estimate first AMI and stroke/TIA, respectively, in type 2 diabetes mellitus patients, by applying backward elimination to the following variables: age, sex, duration of diabetes, smoking, BMI, and use of antihyperglycemic drugs, statins, and aspirin. As time-varying covariates, we analyzed blood pressure, albuminuria, lipid profile, HbA1c, retinopathy, neuropathy, and atrial fibrillation (only in stroke/TIA model). Both models were stratified by antihypertensive drugs. We evaluated 2980 patients (52.8% women; 67.3 ± 11.2 years) with 24,159 person-years of follow-up. We recorded 114 cases of AMI and 185 cases of stroke/TIA. The factors that were independently associated with first AMI were age (≥ 75 years vs. < 75 years) (p = 0.019), higher HbA1c (> 64 mmol/mol vs. < 53 mmol/mol) (p = 0.003), HDL-cholesterol (0.90–1.81 mmol/L vs. < 0.90 mmol/L) (p = 0.002), and diastolic blood pressure (65–85 mmHg vs. < 65 mmHg) (p < 0.001). The factors that were independently associated with first stroke/TIA were age (≥ 75 years vs. < 60 years) (p < 0.001), atrial fibrillation (first year after the diagnosis vs. more than one year) (p = 0.001), glomerular filtration rate (per each 15 mL/min/1.73 m2 decrease) (p < 0.001), total cholesterol (3.88–6.46 mmol/L vs. < 3.88 mmol/L) (p < 0.001), triglycerides (per each increment of 1.13 mmol/L) (p = 0.031), albuminuria (p < 0.001), neuropathy (p = 0.01), and retinopathy (p = 0.023).

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Mortality in older adults following a fragility fracture: real-world retrospective matched-cohort study in Ontario

BMC Musculoskeletal Disorders ◽

10.1186/s12891-021-03960-z ◽

2021 ◽

Vol 22 (1) ◽

Cited By ~ 2

Author(s):

Jacques P. Brown ◽

Jonathan D. Adachi ◽

Emil Schemitsch ◽

Jean-Eric Tarride ◽

Vivien Brown ◽

...

Keyword(s):

Cohort Study ◽

Hip Fracture ◽

Fragility Fracture ◽

Mortality Risk ◽

Real World ◽

First Year ◽

Matched Cohort ◽

Matched Cohort Study ◽

Fracture Cohort ◽

One Year

Abstract Background Recent studies are lacking reports on mortality after non-hip fractures in adults aged > 65. Methods This retrospective, matched-cohort study used de-identified health services data from the publicly funded healthcare system in Ontario, Canada, contained in the ICES Data Repository. Patients aged 66 years and older with an index fragility fracture occurring at any osteoporotic site between 2011 and 2015 were identified from acute hospital admissions, emergency and ambulatory care using International Classification of Diseases (ICD)-10 codes and data were analyzed until 2017. Thus, follow-up ranged from 2 years to 6 years. Patients were excluded if they presented with an index fracture occurring at a non-osteoporotic fracture site, their index fracture was associated with a trauma code, or they experienced a previous fracture within 5 years prior to their index fracture. This fracture cohort was matched 1:1 to controls within a non-fracture cohort by date, sex, age, geography and comorbidities. All-cause mortality risk was assessed. Results The survival probability for up to 6 years post-fracture was significantly reduced for the fracture cohort vs matched non-fracture controls (p < 0.0001; n = 101,773 per cohort), with the sharpest decline occurring within the first-year post-fracture. Crude relative risk of mortality (95% confidence interval) within 1-year post-fracture was 2.47 (2.38–2.56) in women and 3.22 (3.06–3.40) in men. In the fracture vs non-fracture cohort, the absolute mortality risk within one year after a fragility fracture occurring at any site was 12.5% vs 5.1% in women and 19.5% vs 6.0% in men. The absolute mortality risk within one year after a fragility fracture occurring at a non-hip vs hip site was 9.4% vs 21.5% in women and 14.4% vs 32.3% in men. Conclusions In this real-world cohort aged > 65 years, a fragility fracture occurring at any site was associated with reduced survival for up to 6 years post-fracture. The greatest reduction in survival occurred within the first-year post-fracture, where mortality risk more than doubled and deaths were observed in 1 in 11 women and 1 in 7 men following a non-hip fracture and in 1 in 5 women and 1 in 3 men following a hip fracture.

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Use of Trained Mothers to Teach Interviewing Skills to First-Year Medical Students: A Follow-up Study

PEDIATRICS ◽

10.1542/peds.60.2.165 ◽

1977 ◽

Vol 60 (2) ◽

pp. 165-169 ◽

Cited By ~ 1

Author(s):

Paula L. Stillman ◽

Darrell L. Sabers ◽

Doris L. Redfield

Keyword(s):

Medical Students ◽

School Curriculum ◽

Control Group ◽

First Year ◽

Feedback Session ◽

Optimal Learning ◽

Interviewing Skills ◽

One Year ◽

First Year Medical Students

This report describes an attempt to evaluate the effectiveness of "trained mother" interviews early in the medical school curriculum. As an adjunct to a first-year course that teaches interviewing techniques, half of the students were exposed to an interview with one of three trained mothers early in the course. This treatment interview was immediately followed by a feedback session which concentrated on the content and process of interviewing. At the end of the course, all students had an evaluative interview. Those students who had an initial interview and feedback session with a trained mother scored significantly higher on both the content and process of their interviews than the control group. This technique is an effective and efficient way to teach interviewing skills to medical students prior to entering any of their clinical clerkships. A follow-up assessment conducted one year later indicated that one interview with a trained mother is sufficient for optimal learning and that the skills learned are retained over at least that period of time.

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Combined hormonal contraception and risk of venous thromboembolism within the first year following pregnancy

Thrombosis and Haemostasis ◽

10.1160/th13-09-0797 ◽

2014 ◽

Vol 112 (07) ◽

pp. 73-78 ◽

Cited By ~ 1

Author(s):

Thomas Bergholt ◽

Anne Nielsen ◽

Michael J. Paidas ◽

Ellen Christine L. Løkkegaard ◽

Jesper Petersen

Keyword(s):

Venous Thromboembolism ◽

Hormonal Contraception ◽

Hormonal Contraceptives ◽

First Year ◽

The Difference ◽

Combined Hormonal Contraception ◽

One Year ◽

First Time ◽

Combined Hormonal Contraceptives

SummaryEstimating the risk of venous thromboembolism (VTE) associated with combined hormonal contraceptives following early terminated pregnancies or birth, a Danish nationwide retrospective cohort observing a one-year follow-up was defined using three unique registries. All Danish women with confirmed pregnancies aged 15–49 during the period of 1995–2009 were included. The main outcomes were relative and absolute risks of first time venous thromboembolism in users as well as non-users of combined hormonal contraceptives. In 985,569 person-years, 598 venous thromboembolisms were recorded. After early terminated pregnancies and births, respectively, 113 and 485 events occurred in 212,552 and 773,017 person-years. After early terminated pregnancies, the crude VTE incidence ratios were similar, and the numbers needed to harm were equal between groups that did or did not use combined hormonal contraceptives throughout the follow-up year. After childbirth, individuals that used combined hormonal contraceptives were more likely than non-users to experience VTE depicted by crude incidence ratios; however, the difference was only significant after 14 weeks. This implied that the numbers needed to harm were lower for those that used compared to those that did not use combined oral contraceptives in the initial 14 weeks postpartum. In conclusion, the use of combined hormonal contraceptives after early terminated pregnancies was not detrimental, but during the puerperal period, they should be used with caution.

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Nonfocal Symptoms in Patients with Transient Ischemic Attack and Association with Stroke Risk

Current Neurovascular Research ◽

10.2174/1567202619666211217124919 ◽

2021 ◽

Vol 19 ◽

Author(s):

Shuxiang Yang ◽

Lu Zhao ◽

Lulu Pei ◽

Shuang Cao ◽

Yuan Gao ◽

...

Keyword(s):

Transient Ischemic Attack ◽

Stroke Risk ◽

First Year ◽

Kaplan Meier ◽

Predictive Outcome ◽

One Year ◽

Ischemic Attack ◽

Cumulative Risks

Background and Objective: Patients with transient ischemic attack（TIA）occasionally showed nonfocal symptoms, such as decreased consciousness, amnesia and non-rotatory dizziness. This study intended to evaluate the effect of nonfocal symptoms on the prognosis of patients with TIA. Methods: Data from the prospective hospital-based TIA database of the First Affiliated Hospital of Zhengzhou University were analyzed. The predictive outcome was stroke occurrence at 1 year. Cumulative risks of stroke in patients with and without nonfocal symptoms were estimated with Kaplan-Meier models. Results: We studied 1384 patients with TIA (842 men; mean age, 56±13 years), including 450 (32.5%) with nonfocal symptoms. In the ﬁrst year after TIA, stroke occurred in 168（12.1%） patients. There was no difference in the risk of stroke between patients with both focal and nonfocal symptoms and patients with focal symptoms alone (11.8% vs 12.4%, log-rank; P=0.691). Conclusions: The occurrence of nonfocal symptoms did not increase the risk of stroke at one-year follow-up compared to the occurrence of focal symptoms alone.

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Persistence with Antihypertensives Related to Formulation: The Case of Nifedipine

Annals of Pharmacotherapy ◽

10.1345/aph.1e163 ◽

2005 ◽

Vol 39 (2) ◽

pp. 237-242 ◽

Cited By ~ 10

Author(s):

Nancy S Breekveldt-Postma ◽

Ron MC Herings

Keyword(s):

Cardiovascular Diseases ◽

Antihypertensive Drugs ◽

First Year ◽

Proportional Hazard ◽

Once Daily ◽

Gastrointestinal Therapeutic System ◽

Dosing Frequency ◽

Nifedipine Gastrointestinal Therapeutic System ◽

One Year

BACKGROUND: Controlled-release dosage forms may enhance persistence with therapy because of reduced dosing frequency and fewer adverse effects. OBJECTIVE: To assess the differences in persistent use of nifedipine between once-daily nifedipine gastrointestinal therapeutic system (GITS) and twice-daily nifedipine retard formulations. METHODS: Incident nifedipine users were selected from January 1992 to December 2001 from the PHARMO database, including drug-dispensing records and hospital records of more than one million subjects in the Netherlands. Patients with unaltered formulation and dosing frequency of nifedipine in the first year of follow-up with at least 2 prescriptions were included in the cohort. Persistence with different formulations was assessed using Cox's proportional hazard analyses. Covariates included in the analysis were, among others, hospitalizations and comedication for cardiovascular diseases. RESULTS: In total, 5889 incident users of nifedipine were included. The median duration of the first treatment episode was 133 days for nifedipine retard and 262 days for nifedipine GITS. One-year persistence with nifedipine increased from 32% in patients using retard formulations to 44% in patients using nifedipine GITS. Multivariate analyses showed that patients using nifedipine GITS were 1.3 times (RR 1.33; 95% CI 1.22 to 1.46) more persistent than those using retard formulations of nifedipine. Persistent patients more often used other antihypertensive drugs and were more often hospitalized for cardiovascular diseases. CONCLUSIONS: Patients using once-daily nifedipine GITS are more persistent with therapy than patients using twice-daily retard formulations.

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Therapy persistence in newly diagnosed non-valvular atrial fibrillation treated with warfarin or NOAC

Thrombosis and Haemostasis ◽

10.1160/th15-04-0350 ◽

2016 ◽

Vol 115 (01) ◽

pp. 31-39 ◽

Cited By ~ 96

Author(s):

Anja Katholing ◽

Christopher Wallenhorst ◽

Saul Benedict Freedman ◽

Carlos Martinez

Keyword(s):

Atrial Fibrillation ◽

Guideline Adherence ◽

Practice Research ◽

First Year ◽

Clinical Practice Research Datalink ◽

Supplementary Material ◽

One Year ◽

The Uk ◽

Physician Adherence

SummaryEfforts to reduce stroke in atrial fibrillation (AF) have focused on increasing physician adherence to oral anticoagulant (OAC) guidelines, but high early vitamin K antagonist (VKA) discontinuation is a limitation. We compared persistence of non-VKA OAC (NOAC) with VKA treatment in the first year after OAC inception for incident AF in real-world practice. We studied 27,514 anticoagulant-naïve patients with incident non-valvular AF between January 2011 and May 2014 in the UK primary care Clinical Practice Research Datalink, with full medication use linkage: mean age 74.2 ± 12.4, 45.7 % female, mean follow-up 1.9 ± 1.1 years. After treatment initiation and follow-up until 1/2015, the proportion remaining on OAC at one year (persistence) was estimated using competing risk survival analyses. OAC was commenced ≤90 days after incident AF in 13,221 patients (48.1 %): 12,307 VKA and 914 NOAC (apixaban, dabigatran, rivaroxaban). Amongst those treated with OAC, the proportion commencing NOAC increased from zero in 1/2011 to 27.0 % in 5/2014, and OAC prescriptions for CHA2DS2VASc score ≥2 (guideline adherence) increased from 41.2 % to 65.5 %. Persistence with OAC declined over 12 months to 63.6 % for VKA and 79.2 % for NOAC (p< 0.0001). Persistence for those with CHA2DS2VASc ≥2 was significantly greater for NOAC (83.0 %) than VKA (65.3 %, p< 0.0001) at one year and all earlier time points. Comparison of VKA and NOAC cohorts matched on individual CHA2DS2VASc components showed consistent results. In conclusion, persistence was significantly higher with NOAC than VKA, and could alone lead to fewer cardioembolic strokes. Increased guideline adherence following NOAC introduction could further decrease AF stroke burden.Supplementary Material to this article is available online at www.thrombosis-online.com.

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A Prospective Study on the Feasibility of Related and Unrelated Donor Search and Hematopoietic Cell Transplantation for Adults with Acute Leukemia at 31 European Transplant Centers

Blood ◽

10.1182/blood.v112.11.2385.2385 ◽

2008 ◽

Vol 112 (11) ◽

pp. 2385-2385

Author(s):

Vanderson Rocha ◽

Mutlu Arat ◽

Vladimir Koza ◽

Norbet C Gorin ◽

Augustin Ferrant ◽

...

Keyword(s):

Acute Leukemia ◽

Cord Blood ◽

Hla Typing ◽

First Year ◽

Unrelated Donor ◽

Alternative Donor ◽

Family Donor ◽

Unrelated Cord Blood ◽

One Year

Abstract Currently, a donor can be virtually found for all patients with an indication for allo-transplantation due to the increased number of hematopoietic stem cell (HSC) donors and increasing use of umbilical cord blood and haplo-transplants. In this study, we have addressed the question of the feasibility of searching HSC donors grafts [HLA-matched sibling donor (MSD), and alternative donors] and performing such transplants or other treatments (such as autologous HSC transplant or chemotherapy) for adults with acute leukemia for whom an indication of allo-HSCT could be planned during the course of their disease. The second objective was to compare in an “intent to treat” analysis, LFS according to planned strategy treatment. Patients were included at HLA typing test. A specific questionnaire was completed specifying the initial strategies planned for each patient and their changes over the period: at HLA typing, after 3, 6, 9, 12 months after the registration, and twice a year for the following 2 years. From 2003 to 2006, 702 adults were enrolled by 31 EBMT centres, 490 had AML, 212 ALL. HLA typing was performed for 443 patients at diagnosis, 172 after first CR, 11 after CR2, and 64 in more advanced phase. Median follow-up was 31 months, median age was 42-years (18–75); 207 patients were aged more than 50 years. A MSD was found for 309 patients (44%). Of 309 patients, 290 patients had 1 MSD, 17 patients 2 MSD and only 2 patients more than 2 donors. In 40 cases where the MDS was found the transplant centres did not planned to perform the transplant. A transplant using a haploidentical donor was planned in 4 cases. At the end, 273 patients were planned to be transplanted from a family donor (269 from a MSD and 4 from an haplo). For the remaining 429 patients, the indication of an auto-HCT was made in 85 (20%) patients, use of chemotherapy as post-remission therapy in 142 (33%) and indication of an allo-HSCT with an alternative donor in 202 (47%). Of those 202 patients, the transplant centres were keen to search for a cord blood donor in 72 cases and for a haplo donor in 11 cases. Analysing the treatment received at the last follow-up and comparing with the strategy planned at the registration, 215 of 270 (80%) patients were transplanted with a family donor, 112 of 142 (79%) patients were still receiving chemotherapy, 53 of 85 (62%) received an autograft and 118 of 202 (58%) received an unrelated transplant. At last follow-up, 448 patients of 702 patients included received an HSCT (64%). Probability of survival at 2 years for 702 patients was 55%. Interval from HLA typing and HSCT was 125 days for MSD, 148 days for unrelated donor, 167 days for unrelated cord blood and 149 days for autologous transplantation. Cumulative incidence function at one year (CIF; using death as a competing event) for receiving an allograft was 81% if the strategy planned was an allograft with a MSD, 57% for those patients for whom an alternative donor was searched and 6% for those patients for whom an allo-transplant was not planed at inclusion. However, CIF at 1-year for really receiving an allograft was 74% in case of MSD, and only 30% for those patients not having an HLA identical siblingAt 2-years, LFS by initial planned strategy at HLA typing was 43% for those patients in whom a donor search was planned, 47% for those with a planned autologous HSCT, 46% for those with planned chemotherapy and 49% for those with a family transplant. In conclusion, the majority of patients (80%) with AL in European centres are HLA typed at diagnosis or CR1. The majority of patients received a MSD as planned treatment in the first year after HLA typing, in the absence of MSD an alternative donor was searched only for 51% of patients and CIF at one year for performing an allotransplant was 30% however in case of searching for alternative donor, transplantation could be performed in 57% of the cases in the first year. Surprisingly, searching for other alternative donors such as unrelated cord blood or haplo-identical donors, was only done for a small proportion of patients, in spite of encouraging results with both strategies. Therefore, in retrospective studies comparing outcomes of HLA identical sibling, and other alternative donor transplants there is a potential bias linked to the decision to search or not in half of the cases, that is probably linked to patients-, disease- and centre-related factors.

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Real World Data In Myeloma: Experiences From The Swedish Population-Based Registry On 2494 Myeloma Patients Diagnosed 2008-2011

Blood ◽

10.1182/blood.v122.21.1972.1972 ◽

2013 ◽

Vol 122 (21) ◽

pp. 1972-1972 ◽

Cited By ~ 3

Author(s):

Cecilie Blimark ◽

Erik Holmberg ◽

Gunnar Juliusson ◽

Hareth Nahi ◽

Forsberg Karin ◽

...

Keyword(s):

Plasma Cell ◽

Real World ◽

Clinical Intervention ◽

Population Based ◽

Plasma Cell Leukemia ◽

Cell Leukemia ◽

First Report ◽

One Year ◽

The Impact

Abstract Introduction The Swedish Myeloma Registry (SMR) is a prospective observational registry designed to document real-world management and outcomes in newly diagnosed myeloma, with the purpose to improve the quality of the management of patients in Sweden. Population-based registries may provide complementary information on the management of patients to that of clinical intervention trials. With high representation and excellent data quality we can present valuable information in a whole population and reduce the impact of selection on outcome and reduce the subsequent problem with extrapolating data from clinical intervention studies on non-study populations. Methods The registry comprises web-reported data on all patients diagnosed with myeloma, plasmocytoma, and plasma cell leukemia from 2008 in Sweden, at time of diagnosis and after one year of follow-up. Coverage is analyzed through the compulsory Swedish Cancer Registry. Survival is achieved from the Swedish Tax Agency. Missing data are actively requested. This first report contains data on patients diagnosed between 2008 and 2011 with follow-up after one year on patients with symptomatic disease 2008-2010, with a follow-up through the end of 2012. Analyses of incidence, patient characteristics at baseline, proportion of patients given intensive treatment, obtaining very good partial remission (VGPR) and overall survival (OS) were estimated. Results Clinical data at baseline was available for 2494 patients (96% coverage)and 1- year follow-up data on 1427 patients (90% of all symptomatic cases initially reported), from 70 different centers in Sweden. The age adjusted incidence was 6.5 myeloma cases per 100 000 inhabitants and year. The median age was 70 years for men, and 73 years for women (34% younger than 66 years). At diagnosis, 76% were reported as symptomatic myeloma, 18% as smouldering myeloma, 5% plasmocytoma and 1% plasma cell leukemia. IgG-myeloma was most common (59%), followed by IgA (21%), Bence-Jones (13%), non-secretory (4%), IgD and IgM both less than 1%. Among symptomatic myeloma (n=1910), 76% had osteolytic lesions or compression fractures at diagnosis. Anemia (defined as hemoglobin levels below 10 g/dl) was seen in 33%, impaired kidney function (s-creatinin levels above 173 mmol/l) in 18%, and hypercalcemia in 21% at the time of diagnosis. In patients were ISS was available, 23%, 45% and 32% were in stage I, II, and III, respectively. Previous MGUS was known in 13 % of patients. Overall, 81 % of patients 65 years or younger received autologous stem cell transplantation (ASCT) and 4% of the elderly population. In the patients aged 65 years and younger, 63% of patients received one of the newer drugs in the first year of treatment, for the patients 66 to 80 years the number was 56%, and 25% of patients above 80 years. Throughout the study period, an increase in VGPR-rate on initial treatment was observed, more pronounced in younger patients (<66 years), from 35% in 2008 to 46% in 2010. For patients >65 years, the VGPR-rate increased from 17 to 27%. After a median of follow-up time of three years, OS was 63%. There was a significant difference in absolute and relative survival between younger and older patients. In symptomatic myeloma, patients 65 years or younger had an expected 3-year survival of 76% and in patients 66 years and above it was 50% (Figure). The relative 3-year survival for patients with asymptomatic patients was 81%. Discussion SMR is an instrument for increased quality in the management of plasma cell neoplasms in Sweden. This first report from the registry shows very high coverage and good adherence to guidelines in all regions of Sweden, both in diagnostics and treatment. A great effort is made to make the SMR complete and to present population-based data on management and outcome in Sweden. Longer follow-up is needed to address the question of the impact of new treatment options on the survival. The registry gives a great opportunity to perform population-based research of high quality based on the acceptance of the registry among treating physicians. Disclosures: Turesson: Celgene Corp: Honoraria.

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