Comprehensive Analysis of Late Effects and Quality of Life Among Two-Year Survivors of Allogeneic Hematopoietic Cell Transplant (alloHCT) for Nonmalignant Diseases

Abstract

(10%) patients were found to have serum ferritin >1000 ug/dl post-transplant. Of these, a total of 16 (70%) displayed clinically significant iron overload (IOL) that was defined as excess iron leading to organ dysfunction with serum ferritin (SF) of at least 1000 ug/dl. The median age of 18 males and 05 females was 58 years (24-72). Primary diagnosis for AHT included, AML/MDS (n¼16), ALL (n¼2), SAA (n¼2) and others (n¼3). Patients received median of 12 (7-60) lifetime cumulative PRBCs transfusions. The median pre-transplant SF and that at IOL was 1718 ug/dl (850-4658) and 3712 ug/l (1461-45598) respectively. IOL developed at median of 7 months (2-27) post AHT. All but 3 patients had cGvHD (limited skin¼11, extensive ¼9). Liver dysfunctions (median AST ¼ 149 u/l, ALT ¼ 230 u/l and alkaline phosphatase ¼ 249 u/l) was thought to be potentially IOL related. Therapeutic intervention for IOL comprised of phlebotomy in all 23 patients with 2 receiving concurrent oral chelation. With a median of 9 (1-115) phlebotomies, SF <1000 ensued in 15 (2-30) months. Liver functions normalized at median of 22 (4-110) weeks. HFE analyses of both donor and recipients available in 19 patients had no impact on IOL occurrence of its response to treatment. Figure 1 demonstrates relationship of SF decline and influence on hematopoiesis. At 12 months post phlebotomy, Improvement in hemoglobin and platelets was incremental but modest with rise of 4.6% and 13% respectively. Phlebotomy was well tolerated. At the median follow-up of 72 months, 16/23 (70%) patients are alive with median survival of 58 months (19-153). Cause of death included fungal infections in 4 patients. Phlebotomy produces predictable and safe iron reduction in patients with post AHT IOL. We found modest but encouraging improvement in hematopoiesis that requires further testing.