TY - JOUR
T1 - High-dose cyclophosphamide without stem cell rescue in immune-mediated necrotizing myopathies
AU - Mecoli, Christopher A.
AU - Lahouti, Arash H.
AU - Brodsky, Robert A.
AU - Mammen, Andrew L.
AU - Christopher-Stine, Lisa
N1 - Funding Information:
From the Johns Hopkins University School of Medicine (C.A.M., A.H.L., R.A.B., A.L.M., L.C.-S.), Baltimore; and National Institute of Health (A.L.M.), Bethesda, MD. Funding information and disclosures are provided at the end of the article. Go to Neurology.org/nn for full disclosure forms. The Article Processing Charge was funded by the authors. This is an open access article distributed under the terms of the Creative Commons Attribution-NonCommercial-NoDerivatives License 4.0 (CC BY-NC-ND), which permits downloading and sharing the work provided it is properly cited. The work cannot be changed in any way or used commercially without permission from the journal.
Funding Information:
C.A. Mecoli and A.H. Lahouti report no disclosures. R.A. Brodsky served on the scientific advisory board for Alexion, Achillion, and Apel-lis; received research support from NIH, NHLBI, and AA/MDS Foundation; and is on the editorial board for Blood. A.L. Mammen served on the advisory board for aTYR Pharmaceuticals and Biogen; served on the scientific advisory board for Experimental Neurology and Arthritis and Rheumatism; holds patent for anti-AMGCR antibodies; and received research support from NIAMS/NIH and NIDDK/NIH. L. Christopher-Stine served on the scientific advisory board for Novar-tis, MedImmune, Walgreens/Option Care, Mallinckrodt, Idera Pharmaceuticals, Octapharma, and Corbus; received honoraria from Novartis, MedImmune, Walgreens/Option Care, Mallinckrodt, Idera Pharmaceuticals, and Octapharma; holds a patent for and receives royalties from detection of HMG-CoA reductase (HMGCR) antibodies in patient sera; consulted for NuFactor, Option Care, IgG America, Med-ProRx, CSL Behring, NHLBI; and participated in legal proceedings from McAloon and Friedman, P.C., Shaub, Ahmuty, Citrin, and Spratt, LLP, The Chartwell Law Offices, PK Law, and Bodie, Dolina, Hobbs, Friddell & Grenzer, P.C. Go to Neurology.org/nn for full disclosure forms.
Publisher Copyright:
© 2017 The Author(s). Published by Wolters Kluwer Health, Inc. on behalf of the American Academy of Neurology.
Copyright:
Copyright 2018 Elsevier B.V., All rights reserved.
PY - 2017
Y1 - 2017
N2 - To describe the experience managing treatment-refractory immune-mediated necrotizing myopathies (IMNM) with high-dose cyclophosphamide (HiCy) therapy. Methods: Five patients with severe refractory IMNM who were treated with HiCy without stem cell rescue were identified. Their medical records were reviewed to assess demographic, clinical, and histologic characteristics as well as response to therapy. Results: Three patients with anti-signal recognition particle (SRP) and 2 patients with anti-HMG-CoA reductase autoantibodies were included. The mean follow-up time after HiCy therapy was 37 ± 28 months. Two patients demonstrated substantial response, evidenced by improved muscle strength and decreased muscle enzymes after HiCy therapy; both of these patients were anti-SRP positive. Four patients experienced febrile neutropenia after HiCy therapy, one of which required a prolonged intensive care unit stay for infectious complications, from which they eventually recovered. Conclusions: These data suggest that HiCy therapy without stem cell rescue may be considered as an alternative for the treatment of refractory IMNM.
AB - To describe the experience managing treatment-refractory immune-mediated necrotizing myopathies (IMNM) with high-dose cyclophosphamide (HiCy) therapy. Methods: Five patients with severe refractory IMNM who were treated with HiCy without stem cell rescue were identified. Their medical records were reviewed to assess demographic, clinical, and histologic characteristics as well as response to therapy. Results: Three patients with anti-signal recognition particle (SRP) and 2 patients with anti-HMG-CoA reductase autoantibodies were included. The mean follow-up time after HiCy therapy was 37 ± 28 months. Two patients demonstrated substantial response, evidenced by improved muscle strength and decreased muscle enzymes after HiCy therapy; both of these patients were anti-SRP positive. Four patients experienced febrile neutropenia after HiCy therapy, one of which required a prolonged intensive care unit stay for infectious complications, from which they eventually recovered. Conclusions: These data suggest that HiCy therapy without stem cell rescue may be considered as an alternative for the treatment of refractory IMNM.
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U2 - 10.1212/NXI.0000000000000381
DO - 10.1212/NXI.0000000000000381
M3 - Article
C2 - 28975138
AN - SCOPUS:85029060261
VL - 4
JO - Neurology: Neuroimmunology and NeuroInflammation
JF - Neurology: Neuroimmunology and NeuroInflammation
SN - 2332-7812
IS - 5
M1 - 381
ER -