Literature DB >> 20618995

Effects of rituximab in two patients with dysferlin-deficient muscular dystrophy.

Alberto Lerario1, Filippo Cogiamanian, Chiara Marchesi, Marzia Belicchi, Nereo Bresolin, Laura Porretti, Yvan Torrente.   

Abstract

BACKGROUND: The administration of rituximab (RTX) in vivo results in B-cell depletion, but evidence for multiple mechanisms of action have been reported. Surprisingly, B cell depletion produced a response in patients with polymyositis, which is characterized as a T cell-mediated autoimmune disorder with biopsy findings similar to Miyoshi myopathy (MM). Indeed, in dysferlinopathies, there is evidence of immune system involvement including the presence of muscle inflammation and a down regulation of the complement inhibitory factor, CD55.
METHODS: Two patients were treated with four weekly infusions of RTX 375 mg/m2. To measure the improvement in muscle strength after treatment, the isometric hand grip maximal voluntary contraction (MVC) was measured by load cell four times during treatment, and again after one year. In order to assess the reproducibility of our grip assessment, we determined the hand MVC analysis in 16 healthy subjects. Moreover, we measured the number of B cells present in patients by flow cytometric analysis during the course of treatment.
RESULTS: The analysis of B cell number during the course of treatment showed that CD20- and CD19-positive cells were depleted to 0-0.01%. The decrease in B cells was followed by an improvement in the mobility of the pelvic and shoulder girdles as shown by the MRC%. The MVC values of both patients began at values lower than normal whereas during treatment patients had improved percentage of muscle strength. The strength peak in both patients coincided with the minimum B cell values. There were no severe adverse events associated with an infusion of RTX.
CONCLUSION: We consider the increase in muscle strength observed in both treated patients to be a consequence of their treatment with RTX. To our knowledge, these are the first cases of increased muscle strength in patients with MM. Furthermore, the results of this study indicate that B cell depletion with RTX may be useful in the treatment of patients affected by MM, suggesting a possible role for B cells in the pathophysiology of this muscle disorder.

Entities:  

Mesh:

Substances:

Year:  2010        PMID: 20618995      PMCID: PMC2912795          DOI: 10.1186/1471-2474-11-157

Source DB:  PubMed          Journal:  BMC Musculoskelet Disord        ISSN: 1471-2474            Impact factor:   2.362


  27 in total

Review 1.  B-lymphocyte depletion therapy in rheumatoid arthritis and other autoimmune disorders.

Authors:  J C W Edwards; M J Leandro; G Cambridge
Journal:  Biochem Soc Trans       Date:  2002-08       Impact factor: 5.407

Review 2.  B cell therapy for rheumatoid arthritis: the rituximab (anti-CD20) experience.

Authors:  T Shaw; J Quan; M C Totoritis
Journal:  Ann Rheum Dis       Date:  2003-11       Impact factor: 19.103

Review 3.  Overview of the clinical development of rituximab: first monoclonal antibody approved for the treatment of lymphoma.

Authors:  A J Grillo-López; C A White; C Varns; D Shen; A Wei; A McClure; B K Dallaire
Journal:  Semin Oncol       Date:  1999-10       Impact factor: 4.929

4.  Dysferlin mutations in Japanese Miyoshi myopathy: relationship to phenotype.

Authors:  T Takahashi; M Aoki; M Tateyama; E Kondo; T Mizuno; Y Onodera; R Takano; H Kawai; K Kamakura; H Mochizuki; M Shizuka-Ikeda; M Nakagawa; Y Yoshida; J Akanuma; K Hoshino; H Saito; M Nishizawa; S Kato; K Saito; T Miyachi; H Yamashita; M Kawai; T Matsumura; S Kuzuhara; T Ibi; K Sahashi; H Nakai; T Kohnosu; I Nonaka; K Arahata; R H Brown; H Saito; Y Itoyama
Journal:  Neurology       Date:  2003-06-10       Impact factor: 9.910

5.  Treatment of IgM antibody associated polyneuropathies using rituximab.

Authors:  A Pestronk; J Florence; T Miller; R Choksi; M T Al-Lozi; T D Levine
Journal:  J Neurol Neurosurg Psychiatry       Date:  2003-04       Impact factor: 10.154

6.  Efficacy of B-cell-targeted therapy with rituximab in patients with rheumatoid arthritis.

Authors:  Jonathan C W Edwards; Leszek Szczepanski; Jacek Szechinski; Anna Filipowicz-Sosnowska; Paul Emery; David R Close; Randall M Stevens; Tim Shaw
Journal:  N Engl J Med       Date:  2004-06-17       Impact factor: 91.245

7.  Rituximab therapy for multisystem autoimmune diseases in pediatric patients.

Authors:  Bryce A Binstadt; Alvaro M C Caldas; Stuart E Turvey; Kelley D Stone; Howard J Weinstein; Jean Jackson; Robert C Fuhlbrigge; Robert P Sundel
Journal:  J Pediatr       Date:  2003-11       Impact factor: 4.406

8.  First clinical use of ofatumumab, a novel fully human anti-CD20 monoclonal antibody in relapsed or refractory follicular lymphoma: results of a phase 1/2 trial.

Authors:  Anton Hagenbeek; Ole Gadeberg; Peter Johnson; Lars Møller Pedersen; Jan Walewski; Andrzej Hellmann; Brian K Link; Tadeusz Robak; Marek Wojtukiewicz; Michael Pfreundschuh; Michael Kneba; Andreas Engert; Pieter Sonneveld; Mimi Flensburg; Jørgen Petersen; Nedjad Losic; John Radford
Journal:  Blood       Date:  2008-04-04       Impact factor: 22.113

9.  Muscle pathology in dysferlin deficiency.

Authors:  M Fanin; C Angelini
Journal:  Neuropathol Appl Neurobiol       Date:  2002-12       Impact factor: 8.090

Review 10.  Rituximab: a review of its use in non-Hodgkin's lymphoma and chronic lymphocytic leukaemia.

Authors:  Greg L Plosker; David P Figgitt
Journal:  Drugs       Date:  2003       Impact factor: 9.546

View more
  16 in total

1.  Annexin A2 links poor myofiber repair with inflammation and adipogenic replacement of the injured muscle.

Authors:  Aurelia Defour; Sushma Medikayala; Jack H Van der Meulen; Marshall W Hogarth; Nicholas Holdreith; Apostolos Malatras; William Duddy; Jessica Boehler; Kanneboyina Nagaraju; Jyoti K Jaiswal
Journal:  Hum Mol Genet       Date:  2017-06-01       Impact factor: 6.150

Review 2.  Translational research and therapeutic perspectives in dysferlinopathies.

Authors:  Florian Barthélémy; Nicolas Wein; Martin Krahn; Nicolas Lévy; Marc Bartoli
Journal:  Mol Med       Date:  2011-05-06       Impact factor: 6.354

3.  The effects of MyD88 deficiency on disease phenotype in dysferlin-deficient A/J mice: role of endogenous TLR ligands.

Authors:  Kitipong Uaesoontrachoon; Hee-Jae Cha; Beryl Ampong; Arpana Sali; Jack Vandermeulen; Benjamin Wei; Brittany Creeden; Tony Huynh; James Quinn; Kathleen Tatem; Sree Rayavarapu; Eric P Hoffman; Kanneboyina Nagaraju
Journal:  J Pathol       Date:  2013-10       Impact factor: 7.996

4.  A dual acting compound releasing nitric oxide (NO) and ibuprofen, NCX 320, shows significant therapeutic effects in a mouse model of muscular dystrophy.

Authors:  Clara Sciorati; Daniela Miglietta; Roberta Buono; Viviana Pisa; Dario Cattaneo; Emanuele Azzoni; Silvia Brunelli; Emilio Clementi
Journal:  Pharmacol Res       Date:  2011-05-12       Impact factor: 7.658

5.  Adaptive Immune Response Impairs the Efficacy of Autologous Transplantation of Engineered Stem Cells in Dystrophic Dogs.

Authors:  Clementina Sitzia; Andrea Farini; Luciana Jardim; Paola Razini; Marzia Belicchi; Letizia Cassinelli; Chiara Villa; Silvia Erratico; Daniele Parolini; Pamela Bella; Joao Carlos da Silva Bizario; Luis Garcia; Marcelo Dias-Baruffi; Mirella Meregalli; Yvan Torrente
Journal:  Mol Ther       Date:  2016-08-10       Impact factor: 11.454

6.  1α,25(OH)(2)-Vitamin D3 increases dysferlin expression in vitro and in a human clinical trial.

Authors:  Noemi De Luna; Jordi Díaz-Manera; Carmen Paradas; Cristina Iturriaga; Ricardo Rojas-García; Josefa Araque; Mireia Genebriera; Ignasi Gich; Isabel Illa; Eduard Gallardo
Journal:  Mol Ther       Date:  2012-08-21       Impact factor: 11.454

7.  Muscle membrane repair and inflammatory attack in dysferlinopathy.

Authors:  Renzhi Han
Journal:  Skelet Muscle       Date:  2011-03-01       Impact factor: 4.912

8.  Dysferlinopathy course and sportive activity: clues for possible treatment.

Authors:  C Angelini; E Peterle; A Gaiani; L Bortolussi; C Borsato
Journal:  Acta Myol       Date:  2011-10

Review 9.  Influence of immune responses in gene/stem cell therapies for muscular dystrophies.

Authors:  Andrea Farini; Clementina Sitzia; Silvia Erratico; Mirella Meregalli; Yvan Torrente
Journal:  Biomed Res Int       Date:  2014-05-19       Impact factor: 3.411

Review 10.  Limb-girdle muscular dystrophies: where next after six decades from the first proposal (Review).

Authors:  Omar A Mahmood; Xin Mei Jiang
Journal:  Mol Med Rep       Date:  2014-03-13       Impact factor: 2.952

View more

北京卡尤迪生物科技股份有限公司 © 2022-2023.