1. ¹Department of Pediatric Hematology/Oncology, Hannover Medical School, Hannover, Germany
2. ²Department of Pediatric Hematology and Oncology, Friedrich-Schiller-University Jena, Jena, Germany
3. ³Department of Pediatric Hematology and Oncology, University Children's Hospital Hamburg-Eppendorf, Hamburg, Germany
4. ⁴Department of Pediatric Hematology and Oncology, Carl Gustav Carus University, Dresden; Germany
5. ⁵Department of Pediatric Neurology, Hannover Medical School, Hannover, Germany

Correspondence: Dr M Sauer, Children's Hospital—OE 6780, Hannover Medical University, Carl-Neuberg-Strasse 1, Hannover 30625, Germany. E-mail: sauer.martin@mh-hannover.de

Received 5 June 2008; Revised 7 July 2008; Accepted 13 July 2008; Published online 13 October 2008.

Abstract

Hurler's syndrome is an inborn error of mucopolysaccharide metabolism leading to premature death in childhood. Allogeneic hematopoietic SCT can achieve long-term survival by correcting the enzymatic deficiency. In an attempt to improve long-term engraftment and to reduce regimen-related toxicity (RRT), a prospective multicenter approach was initiated in Germany using a fludarabine-based radiation-free preparative regimen. Between 2001 and 2008, 12 children were enrolled. Median age at SCT was 14 months (range, 4–31 months). The conditioning regimen contained fludarabine, BU, melphalan and antithymocyte globulin. CD34 positively selected PBSC were used in 10 children with a matched unrelated donor. Median cell dose was 24.6 10⁶ CD34+ cells per kg (range 10.0–54.8). Two children with a matched sibling donor received non-manipulated BM. Donor lymphocyte infusions were given in 6/12 children for mixed hematopoietic chimerism. At a median follow-up of 29 months (range 2–85 months), all children engrafted and have either stabilized or improved neurological function. In total, 12/12 patients showed donor-derived engraftment with 9/12 having full and 3/12 having mixed hematopoiesis. One developed acute GVHD grade II. RRT grade II was observed in two patients.