For nurses to correctly educate their patients and family members, and to aid nurses in anticipating patient's needs, an understanding of the potential different acute toxicities and supportive care between pediatric patients undergoing RIC- versus MAC-AlloHSCT is vital.
Allogenic hematopoietic cell transplantation (alloHCT) is currently the only curative treatment option for patients with sickle cell disease. Alemtuzumab is a monoclonal antibody directed against CD52 positive cells used in myeloablative conditioning regimens for alloHCT. Its use has been associated with development of autoimmune disease in adult patients with rheumatologic conditions. We report on three cases of new onset autoimmune thyroid disease after alloHCT treatment with alemtuzumab in pediatric patients with sickle cell disease.
(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.
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