A Study - Transplant Treatment Severe Aplastic Anemia - Journal of American Society of Hemotology

Severe aplastic anemia: a prospective study of the effect of early marrow transplantation on acute mortality


BM Camitta, ED Thomas, DG Nathan, G Santos, EC Gordon-Smith, RP Gale, JM Rappeport and R Storb

A prospective randomized trial of therapy for severe aplastic anemia was designed to compare early bone marrow transplantation with conventional treatments. All patients with a sibling matched at the major histocompatibility region were transplanted. Transplantation was performed with 17-100 (median 33) days of original diagnosis. Conventional treatments included transfusion support with or without androgens. Twenty-four of 36 patients intered on the transplant arm are alive after 4-20 (median 9) mo with full marrow reconstitution. Only two are limited by chronic graft-versus-host disease. In contrast only 12 of 31 conventionally treated patients are alive. Six of these survivors have improved, five incompletely. The 19 nontransplant deaths have occurred within 1-11 (median 3) mo of diagnosis. Compared to nontransplant regimens, early transplantation more effectively restores normal marrow function and decreases the acute mortality of severe marrow aplasia (p = 0.006). Pending longer follow-up, early marrow transplantation appears to be the most effective available treatment for severe aplastic anemia.

SUMMARY
http://bloodjournal.hematologylibrary.org/cgi/reprint/48/1/63 (2007 article – the key to successful treatment according to the study is early transplant (within 17-100 days or original diagnosis)). Further reading evidences that siblings were matched for all participants in the study, an excellent citation. Patients were given prednisone (10 mg sq m/day) to detect underlying leukemia. Cyclophosphamide was used alone for conditioning for engraftment. ATG or ATS and procarbazine (PCB) were given. After transplant, methotrexate was given to ameliorate potential graft-versus-host disease.


Study included 17 females transplanted who had idiopathic etiology (severe aplastic anemia from an unknown cause). Diagnosis was made within 17 weeks. Initial, PMN range was 0-1500 (avg 200), 1-23 platelets (avg. 6k), .1-1.4 retriculocytes (avg. .1%), and 40-99 nonmyeloid marrow cells (avg. 85%).

26 patients received CY alone, 10 CY plus ATG and PCB in preparation for engraftment.

Immunosuppression began within 12-97 days after diagnosis (avg. 33 days). Delays in treatments were due to INCOMPATIBLE DONORS, WAITING FOR AN AVAILABLE BED, DIAGNOSIS, INFORMED CONSENT (OF PATIENT AND FAMILY).

Initial complication was sepsis (https://www.google.com/health/ref/Sepsis).