Cure Sickle Cell Initiative (CureSCi) - Hematopoietic Cell Transplant for Sickle Cell Disease (HCT for SCD)

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Accession Number
HLB02422123b

Study Type
Epidemiology Study

Collection Type
Open BioLINCC Study See bottom of this webpage for request information

Study Period
1991 -

NHLBI Division
DBDR

Dataset(s) Last Updated
November 13, 2023

Primary Publication URLs
N/A

Consent

Commercial Use Data Restrictions No

Data Restrictions Based On Area Of Research No

Available Data

Data available for request include allogeneic hematopoietic cell transplants for sickle cell disease (Hb SS and Hb Sβ thalassemia) in the United States from January 1991 to December 2022. Follow-up data through May of 2023 are available.

Objectives

The Center for International Blood and Marrow Transplant Research (CIBMTR) is a hematopoietic cell transplant registry that was established in 1972 at the Medical College of Wisconsin. The overarching goal of the registry is to study trends in transplantations and to advance the understanding and application of allogeneic hematopoietic cell transplantation for malignant and non-malignant diseases. Included in this dataset are children, adolescents and young adults with severe sickle cell disease who received an allogeneic hematopoietic cell transplant in the United States and provided written informed consent for research.

Background

Hematopoietic cell transplant for sickle cell disease is curative. Offering this treatment for patients with severe disease is challenging as only about 20-25% of patients expected to benefit have an HLA-matched sibling. Consequently, several transplantations have utilized an HLA-matched or mismatched unrelated adult donor and HLA-mismatched relative. Transplantation strategies have also evolved over time that has included transplant conditioning regimens of varying intensity, grafts other than bone marrow and novel approaches to overcome the donor-recipient histocompatibility barrier and limit graft-versus-host disease. The data that is available for sickle cell disease transplants have been utilized to report on outcomes after transplantation and compare outcomes after transplantation of grafts HLA-matched related, HLA-mismatched related, HLA-matched and HLA-mismatched unrelated donors. Collectively, these data have advanced our knowledge and understanding of hematopoietic cell transplant for this disease. These data can also serve as “contemporaneous controls” for comparison with other more recent curative treatments like gene therapy and gene editing.

Subjects

Included are patients aged < 1 – 58 years who were transplanted for sickle cell disease in the United States from 106 transplant centers. 63% underwent HLA-matched sibling, 16%, HLA-mismatched related donor, 21% HLA-matched or mismatched unrelated donor transplant.

This dataset includes 1,518 patients with Transplant Essential Data and a subset (699 of 1,518 patients) with Comprehensive Data.

Design

The CIBMTR is a working group of transplant centers who submit data on standardized reporting forms for consecutive transplantations at each center. Data are collected prospectively and at defined time-intervals on standardized data collection forms that are reported by the center. Patients are followed longitudinally until death or lost to follow-up. There are 2 levels of data collection: 1) patient-, disease- and transplant-characteristics on consecutive transplants at each participating transplant center is reported on Transplant Essential Data collection form at the following intervals – pre-transplant, 100 days, 6 months, 12 months and thereafter annually until lost to follow-up or death and 2) a subset of patients are selected randomly at registration (pre-transplant) for Comprehensive Report forms. The comprehensive forms are detailed and transplant centers are reimbursed for data reported on comprehensive report forms. On site audits and physician review of submitted data ensure accuracy of data submitted by each transplant center. A complete listing of data collection forms are available at https://www.cibmtr.org/DataManagement/DataCollectionForms/pages/index.aspx.

Publications


  1. *Brazauskas R, Scigliuolo GM, Wang HL, et al. Risk score to predict event-free survival after hematopoietic cell transplant for sickle cell disease. Blood. 2020; 136(5): 623-6

  2. Eapen M, Brazauskas R, Walters MC, et al. Effect of donor type and conditioning regimen intensity on allogeneic transplantation outcomes in patients with sickle cell disease: a retrospective multicentre, cohort study. Lancet Haematol. 2019; 6(11): e585-96

  3. Abraham A, Heish M, Eapen M, et al. Relationship between mixed donor-recipient chimerism and disease recurrence after hematopoietic cell transplantation for sickle cell disease. Biol Blood Marrow Transplant. 2017; 23(12): 2178-83

  4. *Gluckman E, Cappelli B, Bernaudin F, et al. Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation. Blood. 2017; 129(11): 1548-56

  5. Shenoy S, Eapen M, Panepinto JA, et al. A trial of unrelated donor bone marrow transplantation for children with severe sickle cell disease. Blood. 2016; 128(21): 2562-7

  6. Kamani N, Walters MC, Carter S, et al. Unrelated donor cord blood transplantation for children with severe sickle cell disease: results of one cohort from the phase II study from the Blood and Marrow Transplant Clinical Trails Network (BMT CTN). Biol Blood Marrow Transplant. 2012; 18(8): 1265-72

  7. Panepinto JA, Walters MC, Carreras J et al. Matched-related donor transplantation for sickle cell disease: report from the Center for International Blood and Marrow Transplant Research. B J Haematol. 2007; 137(5): 479-85


*Includes data from collaborators outside the United States that are not available in this dataset.

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