Background Patients with homozygous beta-thalassemia, who havea good prognosis during treatment with conventional therapy,appear to have an especially high probability of hematologiccure with bone marrow transplantation, although the morbidityand mortality associated with such treatment are not established.
Methods The records of all patients with thalassemia who receivedbone marrow transplants from HLA-identical donors in Pesaro,Italy, were examined from October 1982 through May 1992. Detailedevaluation of the outcome was conducted in the 89 patients identifiedas being in class 1 according to the Pesaro classification,in which hepatomegaly, portal fibrosis, and the inadequacy ofiron chelation therapy are considered independent risk factors,and the patients are classified as being in class 1 if noneof these factors are present, class 2 if one or two of the factorsare present, and class 3 if all three factors are present. Sixty-fourof the patients had been prepared for transplantation with adrug regimen in current use that includes busulfan and cyclophosphamidefollowed by cyclosporine as prophylaxis against acute graft-versus-hostdisease (protocol 6).
Results There were seven deaths, all within 101 days of transplantation.Two of the 64 patients treated according to protocol 6 died.The probabilities of survival, rejection-free survival, deathfrom causes unrelated to rejection, and rejection were 0.92,0.85, 0.06, and 0.08, respectively, in the total group and 0.97,0.93, 0.03, and 0.04 in the 64 patients treated according toprotocol 6. Preliminary evidence suggests that there was usefulunloading of tissue iron deposits.
Conclusions The high probability of cure with little early orlate morbidity and mortality suggests that patients with class1 thalassemia who have HLA-identical donors available shouldbe treated by bone marrow transplantation. However, this wasnot a controlled trial, so we cannot directly compare the outcomewith that of conventional treatment.
Source Information
From the Divisione Ematologica e Centro Trapianto Midollo Osseo di Muraglia, Ospedale di Pesaro, Pesaro, Italy (G.L., M.G., P.P., E.A., D.B., C.G., M.A., F. Agostinelli, F. Albertini), and the Fred Hutchinson Cancer Research Center, Seattle (R.A.C.).
Address reprint requests to Mr. Clift at 2919 129th Ave. N.E., Seattle, WA 98104.
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