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Sporadic late-onset nemaline myopathy with MGUS: long-term follow-up after melphalan and SCT.

AbstractOBJECTIVE:
Sporadic late-onset nemaline myopathy (SLONM) is a rare, late-onset myopathy that progresses subacutely. If associated with a monoclonal gammopathy of unknown significance (MGUS), the outcome is unfavorable: the majority of these patients die within 1 to 5 years of respiratory failure. This study aims to qualitatively assess the long-term treatment effect of high-dose melphalan (HDM) followed by autologous stem cell transplantation (SCT) in a series of 8 patients with SLONM-MGUS.
METHODS:
We performed a retrospective case series study (n = 8) on the long-term (1-8 years) treatment effect of HDM followed by autologous SCT (HDM-SCT) on survival, muscle strength, and functional capacities.
RESULTS:
Seven patients showed a lasting moderate-good clinical response, 2 of them after the second HDM-SCT. All of them had a complete, a very good partial, or a partial hematologic response. One patient showed no clinical or hematologic response and died.
CONCLUSIONS:
This case series shows the positive effect of HDM-SCT in this rare disorder. Factors that may portend an unfavorable outcome are a long disease course before the hematologic treatment and a poor hematologic response. Age at onset, level and type of M protein (κ vs λ), and severity of muscle weakness were not associated with a specific outcome.
CLASSIFICATION OF EVIDENCE:
This study provides Class IV evidence that for patients with SLONM-MGUS, HDM-SCT increases the probability of survival and functional improvement.
AuthorsNicol C Voermans, Olivier Benveniste, Monique C Minnema, Henk Lokhorst, Martin Lammens, Wouter Meersseman, Michel Delforge, Thierry Kuntzer, Jan Novy, Thomas Pabst, Françoise Bouhour, Norma Romero, Veronique Leblond, Peter van den Bergh, Marie-Christiane Vekemans, Baziel G van Engelen, Bruno Eymard
JournalNeurology (Neurology) Vol. 83 Issue 23 Pg. 2133-9 (Dec 02 2014) ISSN: 1526-632X [Electronic] United States
PMID25378674 (Publication Type: Journal Article)
Copyright© 2014 American Academy of Neurology.
Chemical References
  • Melphalan
Topics
  • Adult
  • Age of Onset
  • Female
  • Follow-Up Studies
  • Humans
  • Male
  • Melphalan (administration & dosage, adverse effects, therapeutic use)
  • Myopathies, Nemaline (complications, therapy)
  • Paraproteinemias (complications, therapy)
  • Retrospective Studies
  • Stem Cell Transplantation
  • Transplantation, Autologous (methods)
  • Treatment Outcome

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