Hematopoietic stem cell transplantation in patients with Hurler syndrome – myeloablative conditioning efficiency

Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning.

We report transplantation outcomes of 258 children with Hurler syndrome (HS) after a myeloablative conditioning regimen from 1995 to 2007. Median age at transplant was 16.7 months and median follow-up was 57 months. The cumulative incidence of neutrophil recovery at day 60 was 91%, acute graft-versus-host disease (GVHD) (grade II-IV) at day 100 was 25%, and chronic GVHD and 5 years was 16%. Ove...

Pediatric Hematopoietic Stem Cell Transplantation

The introduction and evolution of hematopoietic stem cell transplantation (HSCT) could be traced back to 1950s, to the studies on interactions among irradiation, covering spleen and bone marrow from it and injection of bone marrow cells. Today, HSCT is considered a well-established, effective and promising means of therapy for various malignant and non-malignant medical conditions, both in chil...

Pemphigus vulgaris treated with allogeneic hematopoietic stem cell transplantation following non-myeloablative conditioning.

OBJECTIVES Successful treatment of severe, refractory Pemphigus vulgaris (PV) is reported. METHODS Reduced intensity, non-myeloablative conditioning was employed, followed by allogeneic hematopoietic stem cell transplantation (HSCT) from a fully matched sibling. RESULTS Treatment of refractory PV with myeloablation and subsequent allogenic HSCT has been previously reported, and sustained re...

Allogeneic hematopoietic stem cell transplantation after immunosuppressive but non-myeloablative conditioning: "miniallografts" are no small matter.

Hematopoietic Stem Cell Transplantation for Thalassemia

Thalassemia is an autosomal recessive disorder associated with defective synthesis of the α- or β-chain of hemoglobin. For β-thalassemia major patients, therapeutic options are either monthly red cell transfusions and chelation therapy or allogeneic stem cell transplant. Stem cell transplant is the only curative approach and success is inversely correlated with the degree of iron overload and h...