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Long‐term survival of beta thalassemia major patients treated with hematopoietic stem cell transplantation compared with survival with conventional treatment
Author(s) -
Caocci Giovanni,
Orofino Maria Grazia,
Vacca Adriana,
Piroddi Antonio,
Piras Eugenia,
Addari Maria Carmen,
Caria Rossella,
Pilia Maria Paola,
Origa Raffaella,
Moi Paolo,
La Nasa Giorgio
Publication year - 2017
Publication title -
american journal of hematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.456
H-Index - 105
eISSN - 1096-8652
pISSN - 0361-8609
DOI - 10.1002/ajh.24898
Subject(s) - medicine , treosulfan , busulfan , thalassemia , hematopoietic stem cell transplantation , cohort , transplantation , surgery , graft versus host disease , cumulative incidence , gastroenterology
Allogeneic hematopoietic stem cell transplantation (HSCT) in thalassemia remains a challenge. We reported a single‐centre case‐control study of a large cohort of 516 children and adult patients treated with HSCT or blood transfusion support and iron chelation therapy; 258 patients (median age 12, range 1‐45) underwent sibling (67%) or unrelated (33%) HSCT; 97 patients were adults (age ≥ 16 years). The median follow‐up after HSCT was 11 years (range 1‐30). The conditioning regimen was busulfan (80.6%) or treosulfan‐based (19.4%). A cohort of 258 age‐sex matched conventionally treated (CT) patients was randomly selected. In transplanted patients the 30‐year overall survival (OS) and thalassemia‐free survival (TFS) were 82.6 ± 2.7% and 77.8 ± 2.9%, compared to the OS of 85.3 ± 2.7% in CT patients ( P = NS); The incidence of grade II‐IV acute and chronic graft versus host disease (GvHD) was 23.6% and 12.9% respectively. The probability of rejection was 6.9%. Transplant‐related mortality (TRM) (13.8%) was similar to the probability of dying of cardiovascular events in CT patients (12.2%). High‐risk Pesaro score (class 3) was associated with lower OS (OR = 1.99, 95% C.I.=1.31‐3.03) and TFS (OR = 1.54, 95% C.I.=1.12‐2.12). In adult patients, the 23‐years OS and TFS after HSCT were 70 ± 5% and 67.3 ± 5%, compared to 71.2 ± 5% of OS in CT ( P = NS). Finally, treosulfan was associated with lower risk of acute GvHD ( P = .004; OR = 0.28, 95% C.I.=0.12‐0.67). In conclusion, the 30‐year survival rate of ex‐thalassemia patients after HSCT was similar to that expected in CT thalassemia patients, with the vast majority of HSCT survivors cured from thalassemia.