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• W2028573456 abstract "AlloSCT from a HLA matched sibling donor is the preferred treatment in children with acquired SAA in children. Alternative donor HSCT gives inferior results, and has been reserved for patients lacking matched family donors and unresponsive to medical therapy. However, given the poor outcome of medical therapy in subsets of patients with SAA, the advent less toxic fludarabine (FLU)-based non-myeloablative (NMA) regimens (Chan et al, BMT, 2001), and our recent experience with UCBT (Styczynski/Cairo et al, BMT, 2004), we investigated a risk-adapted AlloSCT approach to 17 consecutive children with SAA with matched related or unrelated donors between 01/2001 and 08/2006. There were 11 males and 6 females with median age of 11 years (range 4-16). Patients with fewer than 10 transfusions underwent NMA AlloSCT with FLU 180 mg/m2, cyclophosphamide (CY) 60 mg/kg, and ATG 8 mg/kg (n=8) or FLU 180 mg/m2, busulfan (BU) 12.8 mg/kg, and alemtuzumab 54 mg/m2(n=2). The remainder underwent myeloablative (MA) AlloSCT with FLU 180 mg/m2, CY 200mg/kg and ATG 8mg/kg (n=6) or CY 200 mg/kg and ATG 8 mg/kg (n=1). Stem cell sources were BM (n=12) from 6/6- (n=9) or 5/6- (n=2) matched family donors or 9/10-matched unrelated donor (MUD) (n=1), or UCB (n=5), either 6/6- (n=1), 5/6- (n=2), or 4/6-matched (n=2). All patients received tacrolimus and mycophenolate mofetil as GVHD prophylaxis as we previously described (Osunkwo/Cairo et al, BBMT, 2004). The median time to myeloid and platelet engraftment were 14 (9-30) and 40.5 (12-161) days, respectively. 15 pts (88.2%) engrafted and 2 (11.8%) had secondary graft failure. Acute GVHD grade II-IV was observed in 35.3% of pts, whereas only 5.9 % developed chronic GVHD. 1 patient developed PTLD. 6 pts (35.3 %) died, multiorgan failure (n=3), extensive AGVHD (n=1), fungal infection (n=1) and thrombotic microangiopathy (n=1). The probability of overall survival (OS) for all patients was 60.9% (CI 95: 36.4-86.5). We did not observe differences in OS according to donor type (related vs. unrelated), intensity of conditioning regimen (MA vs. NMA), or CMV status of donor and recipient. However, we found a significant difference in OS by degree of match (6/6 or 9/10 vs. <6/6) 77.8 % (CI 95: 50.6-100) vs. 33.3 % (CI 95: 0-71.1) (P<0.05). In conclusion, risk-adapted AlloSCT is feasible in children with newly diagnosed acquired SAA and further investigation of this approach is warranted." @default.
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• W2028573456 date "2007-02-01" @default.
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• W2028573456 title "164: Risk adapted allogeneic stem cell transplantation (AlloSCT) for acquired severe aplastic anemia (SAA) in pediatric recipients" @default.
• W2028573456 doi "https://doi.org/10.1016/j.bbmt.2006.12.168" @default.
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