TY - JOUR
T1 - Hematopoietic Stem Cell Transplantation for Mucopolysaccharidoses
T2 - Past, Present, and Future
AU - Taylor, Madeleine
AU - Khan, Shaukat
AU - Stapleton, M.
AU - Wang, Jianmin
AU - Chen, Jing
AU - Wynn, Robert
AU - Yabe, Hiromasa
AU - Chinen, Yasutsugu
AU - Boelens, Jaap Jan
AU - Mason, Robert W.
AU - Kubaski, Francyne
AU - Horovitz, Dafne D.G.
AU - Barth, Anneliese L.
AU - Serafini, M.
AU - Bernardo, Maria Ester
AU - Kobayashi, Hironori
AU - Orii, Kenji E.
AU - Suzuki, Yasuyuki
AU - Orii, T.
AU - Tomatsu, Shunji
N1 - Publisher Copyright:
© 2019 American Society for Blood and Marrow Transplantation
PY - 2019/7
Y1 - 2019/7
N2 - 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.
AB - 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.
KW - Allogenic hematopoietic stem cell transplantation
KW - Enzyme replacement therapy
KW - Limitations
KW - Mucopolysaccharidoses
KW - Outcomes
UR - https://www.scopus.com/pages/publications/85063505811
U2 - 10.1016/j.bbmt.2019.02.012
DO - 10.1016/j.bbmt.2019.02.012
M3 - Review article
C2 - 30772512
AN - SCOPUS:85063505811
SN - 1083-8791
VL - 25
SP - e226-e246
JO - Biology of Blood and Marrow Transplantation
JF - Biology of Blood and Marrow Transplantation
IS - 7
ER -