Hemotopoietic Stem Cell Transplant Examined for Inherited Blood Disorder

HealthDay News — Patients with thalassemia treated with hematopoietic stem cell transplantation (HSCT) have 30-year survival that is similar to that of conventionally treated (CT) patients, according to a study published online August 29 in the American Journal of Hematology.

Giovani Caocci, MD, from the University of Cagliari in Italy, and colleagues performed a single-center case-control study involving 258 patients who underwent sibling or unrelated HSCT (97 adults) and 258 age- and sex-matched CT patients. 

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The researchers found that in transplanted patients, the 30-year overall survival (OS) was 82.6±2.7% and thalassemia-free survival (TFS) was 77.8±2.9% compared with OS of 85.3±2.7% in CT patients (P=NS). Grade II-IV acute and chronic graft versus host disease incidence was 23.6 and 12.9%, respectively. There was a 6.9% probability of rejection. Transplant-related mortality was 13.8%, which was similar to the 12.2% cardiovascular event mortality in CT patients. High-risk Pesaro score correlated with lower OS and TFS (odds ratios, 1.99 and 1.54, respectively). For adult patients, the 23-year OS and TFS were 70±5% and 67.3±5% after HSCT compared with OS of 71.2±5% in CT (P=NS).

“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,” the authors write.

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