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Long-term outcomes positive after HSCT with cyclophosphamide plus antithymocyte globulin

Issue: June 25, 2012

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The use of cyclophosphamide plus antithymocyte globulin conditioning is an effective conditioning regimen for patients with severe aplastic anemia who undergo hematopoietic stem cell transplantation.

However, despite the low treatment-related mortality of this regimen, survivors experienced long-term complications, including vascular necrosis and endocrine dysfunction, leading the researchers to conclude that the exploration of new conditioning regimens is warranted.

“At present long-term survival is now an expected outcome after hematopoietic stem cell transplantation (HSCT) for severe aplastic anemia. Therefore, improving long-term health conditions must be a priority for further research in this field,” the researchers wrote.

In the study, the researchers analyzed 61 patients with acquired severe aplastic anemia between 1991 and 2010. All patients had to have received first HSCT from a HLA-matched sibling. They also had to have been given a conditioning regimen of cyclophosphamide plus antithymocyte globulin, and graft-versus-host disease (GVHD) prophylaxis with cyclosporine and antithymocyte globulin.

The mean age of patients was 21 years, and 41 of 61 patients were adults. The cumulative incidence of acute grade-2/grade-3 GVHD was 16% at 3 months and 23% at 72 months. No one experienced grade-4 GVHD. Eighteen patients developed chronic GVHD, equaling a cumulative incidence of 32% at 72 months.

After a median of 73 months, the estimated 6-year survival was 87.5%. Seven patients died during the course of the study.

At 72 months, 10 patients developed avascular necrosis and 12 patients developed endocrine dysfunction.

Researchers also examined the frequency of second malignancy, the original purpose for using the cyclophosphamide plus antithymocyte globulin regimen. Only one patient was found to have a malignancy, which was Hodgkin’s lymphoma.

References:

• Konopacki J. Haematologica. 2012;97:710-716.