Abstract | OBJECTIVE: 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.
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Authors | Nicol 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 |
Journal | Neurology
(Neurology)
Vol. 83
Issue 23
Pg. 2133-9
(Dec 02 2014)
ISSN: 1526-632X [Electronic] United States |
PMID | 25378674
(Publication Type: Journal Article)
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Copyright | © 2014 American Academy of Neurology. |
Chemical References |
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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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