4.5 Article

Reduced-intensity conditioning is effective for hematopoietic stem cell transplantation in young pediatric patients with Diamond-Blackfan anemia

Journal

BONE MARROW TRANSPLANTATION
Volume 56, Issue 5, Pages 1013-1020

Publisher

SPRINGERNATURE
DOI: 10.1038/s41409-020-01056-1

Keywords

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Funding

  1. Ministry of Health, Labor and Welfare of Japan (Research on Measures for Intractable Diseases) [19FC1005]
  2. Ministry of Education, Culture, Sports, Science and Technology of Japan [KAKENHI: 17K10093, 18K07868]
  3. Japan Agency for Medical Research and Development (AMED) [JP19cm0106501, 15H05909]
  4. Japan Society for the Promotion of Science (JSPS) [JP19H05656]
  5. Ministry of Education, Culture, Sports, Science and Technology [hp160219]
  6. Grants-in-Aid for Scientific Research [18K07868] Funding Source: KAKEN

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This study retrospectively compared the outcomes of DBA patients undergoing HSCT with MAC or RIC conditioning regimens. All 27 patients who underwent HSCT successfully engrafted, with a 3-year overall survival rate of 95.2% and a failure-free survival rate of 88.4%, showing no significant differences between MAC and RIC regimens.
Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative therapy for the hematologic manifestations of Diamond-Blackfan anemia (DBA). However, data regarding the optimal conditioning regimen for DBA patients are limited. We retrospectively compared the outcomes of DBA patients who underwent HSCT using either myeloablative conditioning (MAC) or reduced-intensity conditioning (RIC) regimens. The patients belonged to a cohort treated at our hospitals between 2000 and 2018. HSCT was performed in 27 of 165 patients (16.4%). The median age at the time of HSCT was 3.6 years. Stem cell sources included bone marrow for 25 patients (HLA-matched sibling donors,n = 5; HLA-mismatched related donors,n = 2; HLA-matched/mismatched unrelated donors,n = 18) or cord blood for 2 patients. MAC or RIC regimens were used in 12 and 15 patients, respectively. Engraftment was successful in all 27 patients who underwent HSCT. Three patients who underwent HSCT using MAC regimens developed sinusoidal obstruction syndrome. The 3-year overall survival (OS) and failure-free survival rates (FFS) post-transplantations were 95.2% and 88.4%, respectively, with no significant differences between MAC and RIC regimens. Our data suggest that HSCTs using RIC regimens are effective and obtain engraftment with excellent OS and FFS for young DBA patients.

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