Cancer Treatment Today

The inflammatory myopathies are a heterogeneous group of disorders characterized by muscle weakness and inflammation. Although no standard therapeutic guidelines exist, traditional treatment has included corticosteroids and a variety of second-line immunosuppressants. In severe refractory cases of myositis, autologous stem-cell transplantation (ASCT) may be a consideration. There are several published reports suggesting that it is a low toxicity and effective therapy. A 2014 review by Liu et al concluded: “Unfortunately, polymyositis/dermatomyositis (PM/DM) can
induce marked disability and mortality unless properly recognized, and timely and aggressive therapies
are given. In severe refractory cases of PM/DM, auto-hematopoietic stem-cell transplant (HSCT) may
be a salvage strategy.” There is still an ongoing clinical trial, Stem Cell Transplantation in Idiopathic Inflammatory Myopathy Diseases, NCT00278564.

However, repeating or serial autologous stem cell transplants have not been studied, to my knowledge.

Liu B, Shu S, Kenny TP, Chang C, Leung PS. Stem cell therapy in autoimmune rheumatic diseases: a
comprehensive review. Clin Rev Allergy Immunol. 2014 Oct;47(2):244-57. Alexander T, Bondanza A, Muraro PA, Greco R, Saccardi R, Daikeler T, Kazmi M, Hawkey C, Simoes BP,

Leblanc K, Fibbe WE, Moore J, Snarski E, Martin T, Hiepe F, Velardi A, Toubert A, Snowden JA, Farge D.
SCT for severe autoimmune diseases: Consensus guidelines of the European Society for Blood and
Marrow Transplantation for immune monitoring and biobanking. Bone Marrow Transplant. 2015.
Feb;50(2): 173-80.

Sureda A, Bader P, Cesaro S, Dreger P, Duarte RF, Dufour C, Falkenburg JH, Farge-Bancel D, Gennery A,
Kröger N, Lanza F, Marsh JC, Nagler A, Peters C, Velardi A, Mohty M, Madrigal A. Indications for alloand
auto-SCT for haematological diseases, solid tumours and immune disorders: current practice in
Europe, 2015. Bone Marrow Transplant. 2015 Aug;50(8):1037-56.
Alexander T, Bondanza A, Muraro PA, Greco R, Saccardi R, Daikeler T, Kazmi M, Hawkey C, Simoes BP,
Leblanc K, Fibbe WE, Moore J, Snarski E, Martin T, Hiepe F, Velardi A, Toubert A, Snowden JA, Farge D.
SCT for severe autoimmune diseases: consensus guidelines of the European Society for Blood and
Marrow Transplantation for immune monitoring and biobanking. Bone Marrow Transplant. 2015.
Feb;50(2):173-80. Jemima Albayda and Lisa Christopher-Stine, Novel approaches in the treatment of myositis and myopathies. Ther Adv Musculoskelet Dis. 2012 Oct; 4(5): 369–377