Hematopoietic Stem Cell Transplantation for Mucopolysaccharidoses: Past, Present, and Future.
Publication/Presentation Date
7-1-2019
Abstract
Allogenic hematopoietic stem cell transplantation (HSCT) has proven to be a viable treatment option for a selected group of patients with mucopolysaccharidoses (MPS), including those with MPS types 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 by a multidisciplinary team, including a transplantation physician. Treatment recommendations for MPS are based on multiple biological, sociological, and financial factors, including type of MPS, clinical severity, prognosis, present clinical signs and symptoms (disease stage), age at onset, rate of progression, family factors and expectations, financial burden, feasibility, availability, risks and benefits of available therapies such as HSCT, enzyme replacement therapy (ERT), surgical interventions, and other supportive care. International collaboration and data review are critical to evaluating the therapeutic efficacy and adverse effects of HSCT for MPS. Collaborative efforts to assess HSCT for MPS have been ongoing since the first attempt at HSCT in a patient with MPS reported in 1981. The accumulation of data since then has made it possible to identify early outcomes (ie, transplantation 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 transplantation-related mortality. Assessment of long-term outcomes has considered a variety of factors, including type of MPS, type of graft, age at transplantation, and stage of disease progression, among others. Studies on long-term outcomes are considered a key factor in the use of HSCT in patients with MPS. These studies have shown the effects and limitations of HSCT on improving disease manifestations and quality of life. In this review, we summarize the efficacy, side effects, risks, and cost of HSCT for each type of MPS.
Volume
25
Issue
7
First Page
226
Last Page
226
ISSN
1523-6536
Published In/Presented At
Taylor, M., Khan, S., Stapleton, M., Wang, J., Chen, J., Wynn, R., Yabe, H., Chinen, Y., Boelens, J. J., Mason, R. W., Kubaski, F., Horovitz, D. D. G., Barth, A. L., Serafini, M., Bernardo, M. E., Kobayashi, H., Orii, K. E., Suzuki, Y., Orii, T., & Tomatsu, S. (2019). Hematopoietic Stem Cell Transplantation for Mucopolysaccharidoses: Past, Present, and Future. Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 25(7), e226–e246. https://doi.org/10.1016/j.bbmt.2019.02.012
Disciplines
Medicine and Health Sciences
PubMedID
30772512
Department(s)
Fellows and Residents
Document Type
Article