Hematopoietic stem cell transplantation for mucopolysaccharidoses; past, present, and future.

Hematopoietic stem cell transplantation for mucopolysaccharidoses; past, present, and future.

Biol Blood Marrow Transplant. 2019 Feb 14;:

Authors: Taylor M, Khan S, Stapleton M, Wang J, Chen J, Wynn R, Yabe H, Chinen Y, Boelens JJ, Mason RW, Kubaski F, Horovitz DDG, Barth AL, Serafini M, Bernardo ME, Kobayashi H, Orii KE, Suzuki Y, Orii T, Tomatsu S

Abstract
Allogenic hematopoietic stem cell transplantation (HSCT) has been shown to be a treatment option for a selected group of patients with mucopolysaccharidoses (MPS) (MPS I, II, IVA, VI, and VII). Early diagnosis and timely referral to an expert in MPS are critical, followed by a complete examination and evaluation with a multidisciplinary team, including a transplant physician. Treatment recommendations for MPS are based on multiple factors such as biological, sociological, and financial effects. These include type of MPS, clinical severity, prognosis, present clinical signs and symptoms (disease stage), age at onset, the rate of progression, family significances and expectations, financial burdens, feasibility, availability, risks and benefits with available therapies such as HSCT, enzyme replacement therapy (ERT), surgical interventions, and other supportive care. To evaluate therapeutic efficacy and adverse effects of HSCT for MPS, international collaboration and data review are critical. Since the first attempt of HSCT in a patient with MPS in 1981, collaborative efforts to assess HSCT for MPS have been made continuously. Accumulation of data has made it possible to identify early outcomes (transplant outcomes) and long-term disease-specific outcomes resulting from HSCT. The recent identification of predictive factors and the development of innovative regimens have significantly improved the outcomes of both engraftment failure and transplant mortality. Assessment of long-term outcomes has also been described under consideration of a variety of factors: type of MPS, graft-type, age at transplantation, stage of disease progression, etc. Studies on the long-term outcomes are considered a key achievement for the use of HSCT in MPS communities. These studies have shown the effects and limitations of HSCT to improve disease manifestations and quality of life. In this review, we summarize the efficacy, side effects, risks, and the cost of HSCT for each type of MPS.

PMID: 30772512 [PubMed - as supplied by publisher]

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