Outcome of 27 patients with Hurler's syndrome transplanted from either related or unrelated haematopoietic stem cell sources

Souillet, G; Guffon, Nathalie; Maire, I.; Pujol, M; Taylor, Pra; Sevin, F.; Bleyzac, Nathalie; Mulier, C; Durin, A.; Kébaïli, Kamila; Garrigue, Claire; Bertrand, Yves; Froissart, Roseline; Dorche, C.; Gebuhrer, L.; Garin, C.; Berard, Jason A.; Guibaud, P

doi:10.1038/sj.bmt.1704105

Cited by 201 publications

(198 citation statements)

References 28 publications

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“…Insufficient enzyme delivery to poorly vascularized tissues is thought to account for musculoskeletal deterioration after transplantation, similar to that of untreated children (Weisstein et al 2004;Aldenhoven 2008). Although HSCT may improve ventricular hypertrophy and ventricular function, cardiac valve disease persists , and hearing impairment, persistent corneal clouding, and noninfectious pulmonary complications (e.g., diffuse alveolar hemorrhage, idiopathic pneumonia syndrome) after transplantation are also common (Souillet et al 2003;Kharbanda et al 2006). Therefore, these patients require close follow-up consisting of at least annual assessment by a multidisciplinary team with experience treating children with MPS I (Muenzer et al 2009).…”

Section: Discussionmentioning

confidence: 99%

“…Since enzyme replacement therapy (ERT) is not capable of crossing the blood-brain barrier, HSC transplantation is the treatment of choice for Hurler syndrome. Clinical outcomes include reversal of organomegaly, preservation of neurocognitive development, and improved hearing, vision, and cardiopulmonary function in most transplanted patients (Guffon et al 1998;Whitley et al 1993;Shapiro et al 1995;Peters et al 1996;Vellodi et al 1997;Peters et al 1998;Souillet et al 2003;Aldenhoven et al 2008;Malm et al 2008). However, outcomes are highly variable, and manifestations in certain organ systems continue to progress.…”

Section: Introductionmentioning

confidence: 99%

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Long-Term Functional Outcomes of Children with Hurler Syndrome Treated with Unrelated Umbilical Cord Blood Transplantation

et al. 2014

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Objectives: Hurler syndrome is characterized by progressive multisystem deterioration leading to early death in childhood. This prospective study evaluated the longterm outcomes of patients with Hurler syndrome who underwent umbilical cord blood transplantation from unrelated donors.Study design: Only patients with Hurler syndrome who underwent umbilical cord blood transplantation between December 1995 and March 2006 (n ¼ 25) and who were followed for at least 5 years (n ¼ 19) were included in the analysis. The patients were longitudinally evaluated by a multidisciplinary team of specialists following a standardized protocol.Results: Median age at transplantation was 15.9 months (range 2.1-35), and patients were followed up until a median age of 10

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Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Long-Term Functional Outcomes of Children with Hurler Syndrome Treated with Unrelated Umbilical Cord Blood Transplantation

et al. 2014

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“…Remarkably low GVHD rates were reported in spite of the fact that the median age at transplantation was 25 months. 13 Boelens et al 14 retrospectively analyzed data of 146 MPS IH patients who were retrieved from the European Blood and Marrow Transplantation database. This very thorough report retrospectively identified T-cell depletion of the graft and reduced-intensity regimens with an increased rate of graft failure.…”

Section: Discussionmentioning

confidence: 99%

Allogeneic blood SCT for children with Hurler's syndrome: results from the German multicenter approach MPS-HCT 2005

Sauer

Meissner

Fuchs

et al. 2008

Bone Marrow Transplant

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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 fludarabinebased 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 Â 10 6 CD34 þ 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 Xgrade II. RRT Xgrade II was observed in two patients.

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“…27 The feasibility of standard BU þ CY regimen has been demonstrated in transplantations in the case of, for example, mucopolysaccharidosis type 1H (Hurler). 28 …”

Section: Additional Indicationsmentioning

confidence: 99%

Current European practice in pediatric myeloablative conditioning

Vettenranta

2008

Bone Marrow Transplant

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Myeloablative conditioning continues to be employed in hematopoietic stem cell transplantation among patients with pediatric transplant indications. Fractionated TBI (fTBI) remains, with its considerable anti-leukemic potential, the cornerstone of conditioning in the most common of pediatric indications, ALL in its first, second or subsequent remission despite its well-established longterm sequelae. The feasibility of chemotherapy-only regimens has been established and these regimens widely employed in other pediatric indications, for example, in ALL below the age of 2 years, AML, myelodysplasias or severe aplastic anemia. Conditioning regimens are being modified with data accumulating on the role of, for example, pre-transplant residual disease, advanced HLAtyping or haploidentical transplantations in the pediatric setting.

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Outcome of 27 patients with Hurler's syndrome transplanted from either related or unrelated haematopoietic stem cell sources

Cited by 201 publications

References 28 publications

Long-Term Functional Outcomes of Children with Hurler Syndrome Treated with Unrelated Umbilical Cord Blood Transplantation

Long-Term Functional Outcomes of Children with Hurler Syndrome Treated with Unrelated Umbilical Cord Blood Transplantation

Allogeneic blood SCT for children with Hurler's syndrome: results from the German multicenter approach MPS-HCT 2005

Current European practice in pediatric myeloablative conditioning

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