Hematopoietic Cell Transplantation for Storage Diseases

Peters, Charles

doi:10.1002/9780470987070.ch107

Cited by 6 publications

(8 citation statements)

References 163 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…Allogeneic hematopoietic stem cell transplantation is the only possible cure or method of stopping the progression of the disease in most of these inherited disorders (5)(6)(7)(8). Our experience with this method, using HLA-identical siblings and matched unrelated donors, seems very encouraging as judged by a low TRM and low incidences of acute and chronic GVHD (Figs.…”

Section: Discussionmentioning

confidence: 98%

“…Patients with ALD should be transplanted in very early stage disease (7). More recently, it has been suggested that ASCT should be performed before symptoms appear (Peters and Shapiro, personal communication).…”

Section: Discussionmentioning

confidence: 99%

“…Allogeneic hematopoietic stem cell transplantation (ASCT) can correct genetic diseases in two ways: by replacing genetically deranged or absent blood cells, or by implanting bone marrow cells that will deliver a normal protein to the tissues of a defective host. Immunodeficiencies, hematologic defects, lysosomal storage disorders, and peroxisomal diseases are among the diseases that can be treated, cured, or rearrested by ASCT (5)(6)(7)(8). The first patients cured by ASCT had severe combined immunodeficiencies (9,10) and are still alive after 36 years.…”

mentioning

confidence: 99%

“…Histiocytic disorders cured by ASCT include familial hemophagocytic lymphohistiocytosis (FHL) and Langerhans' cell histiocytosis (20 -21). In patients with mucopolysaccharoidosis ASCT is given to those with Hurler's disease and Maroteaux-Lamy disease (6)(7)(8)22). A successful outcome from the treatment with ASCT has also been reported in the lipidosis Gaucher's disease and metachromatic leukodystrophy (MLD) (23,24).…”

mentioning

confidence: 99%

“…A successful outcome from the treatment with ASCT has also been reported in the lipidosis Gaucher's disease and metachromatic leukodystrophy (MLD) (23,24). Adrenoleukodystrophy (ALD), an X-linked, peroxisomal disorder, can be cured by the administration of ASCT early in its course (7,25).…”

mentioning

confidence: 99%

See 4 more Smart Citations

Allogeneic Hematopoietic Stem Cell Transplantation for Inherited Disorders: Experience in a Single Center

et al. 2006

View full text Add to dashboard Cite

show abstract

Section: Discussionmentioning

confidence: 98%

Section: Discussionmentioning

confidence: 99%

mentioning

confidence: 99%

mentioning

confidence: 99%

mentioning

confidence: 99%

See 3 more Smart Citations

Allogeneic Hematopoietic Stem Cell Transplantation for Inherited Disorders: Experience in a Single Center

et al. 2006

View full text Add to dashboard Cite

show abstract

Trends in haematopoietic cell transplantation for inborn errors of metabolism

Boelens

2006

J of Inher Metab Disea

100

View full text Add to dashboard Cite

For the last 25 years, haematopoietic cell transplantation (HCT) has been used as effective therapy for selected inborn errors of metabolism (IEMs), mainly lysosomal storage diseases and peroxisomal disorders. The main rational for HCT in IEMs is based on the provision of correcting enzymes by donor cells within and outside the blood compartment. The ultimate goal of HCT is to achieve a normal or near-normal life and normal neurodevelopment. HCT has been performed for more than 20 diseases. Only for Hurler syndrome, X-ALD and infantile Krabbe disease, are detailed studies available suggesting that HCT is indicated for carefully selected cases. Improvement of transplantation techniques and alternative therapies may change the recommended (contra-)indications for IEM. A recent example of emerging transplantation techniques is the fast availability of unrelated cord blood (UCB). UCB makes HCT feasible in patients with rapidly progressive neurological diseases. Because of the fast availability of UCB and therefore the ability to transplant shortly after diagnosis, there is no indication for patients in a moderate/good clinical condition to receive enzyme replacement therapy (ERT; in Hurler syndrome) prior to or during HCT and can ERT only be considered in patients with poor clinical condition. Mesenchymal stem cell infusions with HCT is an emerging technique, and might be interesting in halting the remaining defects after successful HCT. Improvement in HCT techniques and novel stem cell sources will significantly impact the safety and efficacy of this therapy as well as expand the list of candidate disorders. A good functioning worldwide registry would be necessary to measure the effects of the procedures performed in more detail.

show abstract