Second hematopoietic SCT in patients with thalassemia recurrence following rejection of the first graft

Abstract

There is a substantial incidence of graft failure in patients with thalassemia after myeloablative conditioning regimens especially in class 3 patients in whom its incidence could be as high as 8-38.5%. Most patients with graft failure have recurrence of thalassemic marrow. Historically, results of second transplants for thalassemia were poor because of a high rejection rate and/or increased TRM. Sixteen patients with thalassemia recurrence following rejection of the first graft and with a median age of 9 years (range, 4-20) were given second transplants using BM (n=7) or PBSC (n=9) after preparation with a new treatment protocol. All but two patients received stem cells from the same donor. The median interval between two transplants was 28 months (range, 8-204). The sustained engraftment rate was high (94%) with only one patient having primary graft failure. The probability of overall survival, event-free survival, TRM and graft failure were 79, 79, 16 and 6%, respectively. There were three transplant-related deaths. Thirteen patients are alive with Lansky/Karnofsky score of 100. This intensified treatment protocol was well tolerated with no significant increase in toxicity. The excellent results obtained with this new preparative regimen allow us to recommend it for second transplantation for patients with thalassemia recurrence.

How I treat thalassemia

“…89 A few patients who failed the first transplantation underwent a second transplantation. Although the preliminary results are encouraging, 90 this approach requires more clinical data before it can be recommended.…”

HLA-matched sibling bone marrow transplantation for β-thalassemia major

“…5,36,37 However, in this series graft failure, which occurred in nearly 10% of cases, was fatal in 11 cases and required rescue by second transplantation to restore normal hematopoiesis. The reasons for this are unclear.…”

Recent advances in the management of Thalassaemia: A Review Update

“…In the alternating treatment group serum ferritin showed a significant reduction (P= 0.005) as compared to DFP group. DFO monotherapy versus DFP monotherapy, alternate or combined DFO-DFP regimes, have been evaluated in another long-term multicenter, randomized trial including 265 patients 48 . None of the patients on DFP alone or in combined treatment died, one death occurred with alternate treatment and 10 deaths with DFO treatment.…”

“…The small size or small number of stem cells in the UBC collection relative to the number required for engraftment are probably the main causes of failure of UCB transplantation; therefore, this procedure is being used mainly in pediatric patients 57 . The use of UCB from unrelated donors has resulted in only 77% survival and 65% event-free survival, respectively, in 36 thalassemia patients 58 .…”