Unrelated CD3/CD19-Depleted Peripheral Stem Cell Transplantation for Hurler Syndrome

REFERENCES

• Muenzer J, Wraith JE, Clarke LA. Mucopolysaccharidosis I: management and treatment guidelines. Pediatrics. 2009;123:19–29.
   PubMed Web of Science ®Google Scholar
• De Ru MH, Boelens JJ, Das AM, Enzyme replacement therapy and/or hematopoietic stem cell transplantation at diagnosis in patients with mucopolysaccharidosis type I: results of a European consensus procedure. Orphanet J Rare Dis. 2011;6:55.
   Google Scholar
• D'Aco K, Underhill L, Rangachari L, Diagnosis and treatment trends in mucopolysaccharidosis I: findings from the MPS I Registry. Eur J Pediatr. 2012;171:911–919.
   PubMedGoogle Scholar
• Wraith JE, Beck M, Lane R, Enzyme replacement therapy in patients who have mucopolysaccharidosis I and are younger than 5 years: results of a multinational study of recombinant human α-1-iduronidase (laronidase). Pediatrics. 2007;120:37–46.
   PubMed Web of Science ®Google Scholar
• Hobbs J, Hugh-Jones K, Barrett A, Reversal of clinical features of Hurler's disease and biochemical improvement after treatment by bone marrow transplantation. Lancet. 1981;2:709–712.
   PubMed Web of Science ®Google Scholar
• Boelens JJ, Rocha V, Aldenhoven M, Risk factor analysis of outcomes after unrelated cord blood transplantation in patients with Hurler syndrome. Biol Blood Marrow Transplant. 2009;15:618–625.
   PubMed Web of Science ®Google Scholar
• Gordon PR, Leimig T, Mueller I, A large-scale method for T-cell depletion: towards graft engineering of mobilized peripheral blood stem cells. Bone Marrow Transplant. 2002;30:69–74.
   PubMed Web of Science ®Google Scholar
• Schumm M, Lang P, Taylor G, Isolation of highly purified autologous and allogeneic peripheral CD34+ cells using the CliniMACS device. J Hematother. 1999;8:209–218.
   PubMedGoogle Scholar
• Prasad VK, Kurtzberg J. Cord blood and bone marrow transplantation in inherited metabolic diseases: scientific basis, current status and future directions. Br J Haematol. 2009;148:356–372.
   Google Scholar
• Prasad VK, Kurtzberg J. Transplant outcomes in mucopolysaccharidoses. Semin Hematol. 2010;47:59–69.
   PubMed Web of Science ®Google Scholar
• Boelens JJ, Aldenhoven, M, Purtill D, On behalf of all participating centers from Eurocord, Inborn Errors Working Party of the EBMT, Duke University and the Centre for International Blood and Marrow Research. Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning. Blood. 2013;121:3981–3987.
   PubMed Web of Science ®Google Scholar
• Grigull L, Beilken A, Schrappe M, Transplantation of allogeneic CD34-selected stem cells after fludarabine-based conditioning regimen for children with mucopolysaccharidosis 1H (M. Hurler). Bone Marrow Transplant. 2005;35:265–269.
   PubMed Web of Science ®Google Scholar
• Sauer M, Meissner B, Fuchs D, Allogeneic blood SCT for children with Hurler's syndrome: results from the German multicenter approach MPS-HCT 2005. Bone Marrow Transplant. 2009;43:375–381.
   PubMed Web of Science ®Google Scholar
• Danylesko I, Shimoni A, Nagler A. Treosulfan-based conditioning before hematopoietic SCT: more than a BU look-alike. Bone Marrow Transplant. 2012;47:5–14.
   PubMed Web of Science ®Google Scholar
• Greystoke B, Bonanomi S, Carr TF, Treosulfan-containing regimens achieve high rates of engraftment associated with low transplant morbidity and mortality in children with non-malignant disease and significant co-morbidities. Br J Haematol. 2008;142:257–262.
   PubMed Web of Science ®Google Scholar
• Peters C, Balthazor M, Shapiro EG, Outcome of unrelated donor bone marrow transplantation in 40 children with Hurler syndrome. Blood. 1996;87:4894–4902.
   PubMed Web of Science ®Google Scholar
• Boelens JJ, Wynn RF, O'Meara A, Outcomes of hematopoietic stem cell transplantation for Hurler's syndrome in Europe: a risk factor analysis for graft failure. Bone Marrow Transplant. 2007;40:225–233.
   PubMed Web of Science ®Google Scholar
• Staba SL, Escolar ML, Poe M, Cord-blood transplants from unrelated donors in patients with Hurler's syndrome. N Engl J Med. 2004;350:1960–1969.
   PubMed Web of Science ®Google Scholar
• Prasad VK, Mendizabal A, Parikh SH, Unrelated donor umbilical cord blood transplantation for inherited metabolic disorders in 159 pediatric patients from a single center: influence of cellular composition of the graft on transplantation outcomes. Blood. 2008;112:2979–2989.
   PubMed Web of Science ®Google Scholar
• Gaipa G, Dassi M, Perseghin P, Allogeneic bone marrow stem cell transplantation following CD34+ immunomagnetic enrichment in patients with inherited metabolic storage diseases. Bone Marrow Transplant. 2003;31:857–860.
   Google Scholar
• Bader P, Willasch A, Jarisch A, Transplantation CD3/CD19 depletierter Stammzellen. Monatsschr Kinderheilkd. 2010;158:216–222.
   Google Scholar
• Bader P, Soerensen J, Jarisch A, Rapid immune recovery and low TRM in haploidentical stem cell transplantation in children and adolescence using CD3/CD19 depleted stem cells. Best Pract Res Clin Haematol. 2011;24:331–337.
   PubMed Web of Science ®Google Scholar
• Bethge WA, Haegele M, Faul C, Haploidentical allogeneic hematopoietic cell transplantation in adults with reduced intensity conditioning and CD3/CD19 depletion: fast engraftment and low toxicity. Exp Hematol. 2006;34:1746–1752.
   PubMed Web of Science ®Google Scholar
• Handgretinger R. Negative depletion of CD3+ and TcRαβ+ T cells. Curr Opin Hematol. 2012;19:434–439.
   PubMed Web of Science ®Google Scholar