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Non‐myeloablative matched sibling stem cell transplantation with the optional reinforced stem cell infusion for patients with hemoglobinopathies
Author(s) -
Shin SeungHwan,
Park SungSoo,
Park Silvia,
Jeon YoungWoo,
Yoon JaeHo,
Yahng SeungAh,
Cho ByungSik,
Kim YooJin,
Lee Seok,
Kim HeeJe,
Min ChangKi,
Cho SeokGoo,
Kim DongWook,
Lee JongWook,
Eom KiSeong
Publication year - 2020
Publication title -
european journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.904
H-Index - 84
eISSN - 1600-0609
pISSN - 0902-4441
DOI - 10.1111/ejh.13455
Subject(s) - medicine , alemtuzumab , thalassemia , transplantation , stem cell , immunosuppression , hematopoietic stem cell transplantation , gastroenterology , surgery , genetics , biology
Background The NIH protocol for non‐myeloablative (NMA) conditioning allogeneic stem cell transplantation (alloSCT) with alemtuzumab and low‐dose total body irradiation corrected the abnormal sickle cell disease (SCD) phenotype without the risk of graft‐versus‐host disease. However, alloSCT using NMA conditioning had been rarely applied to β‐thalassemia major (β‐TM) patients. Methods To avoid prolonged immunosuppression, we developed a two‐stage strategy. Mixed donor chimerism was initially achieved using the protocol developed by the NIH protocol. Thereafter, we facilitated donor chimerism using the optional reinforced stem cell (SC) infusion in cases requiring protracted immunosuppression or experiencing impending graft failure. Results In this study, β‐TM (n = 9) and SCD (n = 4) patients were equally effectively treated with eradicating the abnormal hemoglobin phenotype. Five patients, including four β‐TM, achieved stable mixed chimerism without receiving optional reinforced SC infusion. All patients that received optional reinforced infusion achieved complete (n = 4) or mixed chimerism (n = 1). The overall survival rate and event‐free survival at 4 years were 91.7% (95% CI; 53.9‐98.8) in both groups, with a thalassemia‐free survival rate in β‐TM patients of 87.5% (95% CI; 38.7‐98.1). Conclusion This study is the first to report successful NMA conditioning alloSCT to achieve stable mixed chimerism correcting the abnormal hemoglobin phenotype in adult β‐TM patients.