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Alternative donor HSCT in refractory acquired aplastic anemia – Prevention of graft rejection and graft versus host disease by immunoablative conditioning and graft manipulation
Author(s) -
Urban Christian,
Benesch Martin,
Sovinz Petra,
Sipurzynski Sabine,
Lackner Herwig,
Müller Ewa,
Schwinger Wolfgang
Publication year - 2012
Publication title -
pediatric transplantation
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.457
H-Index - 69
eISSN - 1399-3046
pISSN - 1397-3142
DOI - 10.1111/j.1399-3046.2012.01692.x
Subject(s) - medicine , aplastic anemia , graft versus host disease , graft vs host reaction , refractory (planetary science) , graft rejection , disease , surgery , transplantation , immunology , bone marrow transplantation , bone marrow , physics , astrobiology
Urban C, Benesch M, Sovinz P, Sipurzynski S, Lackner H, Müller E, Schwinger W. Alternative donor HSCT in refractory acquired aplastic anemia – Prevention of graft rejection and graft versus host disease by immunoablative conditioning and graft manipulation. Abstract: Early alternative donor HSCT is a potentially curative therapeutic option for patients with AAA not responding to IST. Seven patients (median age at diagnosis, 11 yr) with refractory AAA without a MSD underwent HSCT from matched unrelated (n = 6) or haploidentical (n = 1) donors. Conditioning regimens included CY (n = 7), muromonab‐CD3/ATG (n = 7), TT (n = 6), FLU (n = 5), and TLI (n = 2). Grafts were either CD34 purified and/or CD3/19 depleted and contained a median of 10.17 × 10 6 /kg CD34 and 5.5 × 10 4 /kg CD3 cells. All patients engrafted rapidly. Median time to leukocyte engraftment was 10 days. With a median follow‐up of 26 (range, 11–153) months, six patients are alive and well with complete donor hematopoiesis. One heavily pretreated patient developed GVHD grade III and died from progressive renal failure (resulting from microangiopathic hemolytic anemia) and disseminated aspergillosis. Early alternative donor HSCT can help to avoid complications from prolonged IST and presumably improve survival of patients with refractory AAA. Administration of high doses of CD34 purified and/or CD3/19 depleted stem cells following novel immunoablative conditioning may prevent graft rejection and GVHD. However, a long interval from diagnosis to HSCT seems to be associated with poor outcome.