Non‐sibling hematopoietic stem cell transplantation using myeloablative conditioning regimen in children with Maroteaux‐Lamy syndrome: A brief report

Maroteaux‐Lamy syndrome is a rare inherited lysosomal storage disorder with a progressive course. HSCT is a curable option for treatment in these patients. The following report describes our experience in HSCT for three patients with Maroteaux‐Lamy syndrome using non‐sibling donors. All of the patie...

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Bibliographic Details
Published in:Pediatric transplantation 2017-08, Vol.21 (5), p.n/a
Main Authors: Behfar, Maryam, Dehghani, S. Sharareh, Rostami, Tahereh, Ghavamzadeh, Ardeshir, Hamidieh, Amir Ali
Format: Article
Language:English
Subjects:
Antilymphocyte serum
Blood & organ donations
Bone marrow
Bone marrow transplantation
Busulfan
Child
Child, Preschool
Children
Cord blood
Cyclophosphamide
Fatal Outcome
Female
Globulins
Graft rejection
Grafts
hematopoietic stem cell transplant
Hematopoietic Stem Cell Transplantation - methods
Humans
Intravenous administration
Male
Maroteaux-Lamy syndrome
Mucopolysaccharidosis VI - therapy
non‐sibling donor
Peripheral blood
Stem cell transplantation
Stem cells
Thymocytes
Transplantation
Transplantation Conditioning - methods
Transplants & implants
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Summary:Maroteaux‐Lamy syndrome is a rare inherited lysosomal storage disorder with a progressive course. HSCT is a curable option for treatment in these patients. The following report describes our experience in HSCT for three patients with Maroteaux‐Lamy syndrome using non‐sibling donors. All of the patients received the same myeloablative regimen consisting of intravenous busulfan, cyclophosphamide, and rabbit antithymocyte globulin. Patients underwent HSCT from haploidentical other‐related (n=1), full‐matched other‐related (n=1), and one‐locus‐mismatched unrelated donor. Stem cell sources included bone marrow (n=1), peripheral blood (n=1), and cord blood (n=1). Currently, two patients who received transplant from other‐related donors showed full engraftment and regression of the symptoms of the disease, while for the patient with unrelated cord blood donor, graft failure resulted in progression of the disease and death. The result of our study showed beneficial effects of HSCT even from heterozygote donor. Due to rarity of the disease, future multicenter studies are recommended to find the best treatment approaches based on the patients’ status.
ISSN:1397-3142
1399-3046
DOI:10.1111/petr.12981