HLA Identical Siblings Are the Best Donors for Children with ALL

Christina Peters, Andre Schrauder, Martin Schrappe, Arend von Stackelberg, Peter Bader, Wolfram Ebell, Peter Lang, Bernhard Kremens, Karl-Walter Sykora, Ingo Mueller, Karoline Ehlert, Michael Albert (+11 others)
2014 Biology of Blood and Marrow Transplantation  
2 received myeloablative conditioning regimens (MAC). Patients received HSCT from MRDs (4) and MMUDs (2). 5/6 patients engrafted with the 1 st HSCT. One patient had primary engraftment failure and was rescued with a 2 nd HSCT from the same donor using MAC. 3/6 six patients developed GVHD (acute GVHD e 2, chronic extensive GVHD -1). 4/6 patients are alive (OS: 67%) with a median follow up of 1753 days (range: 92e5963). However, all 4 patients with TINF2 mutations suffered unusual multi-system
more » ... plications which developed late (4e5 years) after HSCT. The 2 patients with DKC1 and TERT mutation had no such atypical complications and are doing well at last follow up. Recent reports describe a more severe phenotype in patients with TINF2 mutations and the extra-hematopoietic manifestations that continue after transplantation suggest that HSCT can have only limited impact on the natural course of their disease and on their long-term outcome. These patients require close multi-disciplinary follow up after HSCT to ensure early detection of complications. Future improvements could focus on the use of less toxic RIC regimens, measurement of biomarkers to predict complications and perhaps novel therapies to correct the underlying telomere defect.
doi:10.1016/j.bbmt.2013.12.292 fatcat:n53qyazwifbqbbdyfcwdll7udm