Allogeneic hematopoietic stem cell transplantation (HSCT) appears safe for treatment of hepatitis-associated aplastic anemia, according to a study results reported in the International Journal of Hematology.

The study was a retrospective analysis of 37 adult patients who received their first allogeneic HSCT for hepatitis-associated aplastic anemia between 1985 and 2014. Patients were registered with the Japan Society for Hematopoietic Cell Transplantation database.

There were 32 patients alive at the time of study. The 5-year overall survival rate was 86.0% (95 CI, 69.4%-93.9%), and the 5-year failure-free survival rate was 75.0% (95% CI, 57.4%-86.2%). Median follow-up was at 54.1 months (range, 3.2-195.2).

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Two patients died early, and the remaining patients experienced neutrophil engraftment in a median of 17 days (range, 10-36) post-transplant. By 40 days post-transplant, engraftment had occurred with a cumulative incidence of 94.6% (95% CI, 75.2%-99.0%).

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Patients were a median age of 24 years (range, 16-61). There was a median of 6.0 months (range, 0.5-430.8) between hepatitis-associated aplastic anemia diagnosis and HSCT. Immunosuppressive therapy had previously been given to 14 patients for aplastic anemia.

Human leukocyte antigen-identical siblings were sources of bone marrow (19 patients) or peripheral blood stem cells (5 patients) for some patients, while unrelated donors provided bone marrow (11 patients) or cord blood (2 patients) for others.

Most conditioning approaches included either high-dose cyclophosphamide (12 patients) or fludarabine with low-dose cyclophosphamide (18 patients). The most common graft-versus-host-disease (GVHD) prophylaxis strategies used tacrolimus with methotrexate (15 patients) or cyclosporine with methotrexate (15 patients).

Causes of fatalities included hemorrhage (2 patients), bacterial infection (1 patient), idiopathic hyperammonemia (1 patient), and cardiomyopathy soon after high-dose cyclophosphamide (1 patient).

The authors reported no post-HSCT lymphoproliferative disorders or secondary cancers within this population. Acute GVHD at grades 1 to 2 affected 12 patients in total. Chronic GVHD was seen in 10 patients, with 3 treated for extensive chronic GVHD.

According to the authors, “our results suggested that allogeneic HSCT for hepatitis-associated aplastic anemia would provide favorable survival. Optimal transplant procedures should be explored by an accumulation of more patients managed with modern methods in future studies.”


1. Mori T, Onishi Y, Ozawa Y, et al. Outcome of allogeneic hematopoietic stem cell transplantation in adult patients with hepatitis-associated aplastic anemia [published online April 8, 2019]. Int J Hematol. doi:10.1007/s12185-019-02644-8