Polyneuropathy improvement following autologous stem cell transplantation for POEMS syndrome

Chafic Karam, Christopher J. Klein, Angela Dispenzieri, P. James B Dyck, Jay Mandrekar, Anita D'Souza, Michelle L. Mauermann

Research output: Contribution to journalArticle

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Abstract

Objective: To study the evolution of the neuropathy and long-term disability in a large cohort of patients with POEMS (polyneuropathy, organomegaly, endocrinopathy, M protein, and skin changes) syndrome following autologous stem cell transplantation (ASCT). Methods: Retrospective chart review documenting the clinical, electrophysiologic, and laboratory characteristics of patients with POEMS syndrome undergoing ASCT at Mayo Clinic, Rochester. Results: Sixty patients with a median follow-up time of 61 months were studied. All patients had peripheral polyneuropathy and demonstrated neurologic improvement after ASCT (apart from one patient who died early). Before ASCT, 27 patients (45%) required a wheelchair and 17 (29%) required a walker or foot brace. At the end of the follow-up period, no patient was using a wheelchair and 23 patients (38%) were using a foot brace. The median Neuropathy Impairment Score improved from 66 to 48 points at 12 months and to 30 points at most recent follow-up (p < 0.0001). Median Rankin Scale score improved from 3 to 1.5 (p < 0.0001). Vascular endothelial growth factor levels decreased from a median of 452 to 63.5 pg/mL (p < 0.0001). The ulnar compound motor action potential amplitude (median) improved from 4.3 to 7.6 mV (p < 0.0001) and ulnar compound motor action potential conduction velocity (median) improved from 34 to 51 m/s (p < 0.0001). Predicted forced vital capacity improved from 81% to 88% (p < 0.0001). Periengraftment syndrome occurred in 24 patients. Fourteen patients required additional chemotherapy and/or radiation following ASCT, but there was no clinical deterioration in the neuropathy in any of these patients. Six patients died: 1 due to POEMS, 1 due to failed engraftment, and 4 due to other malignancies (2 myelodysplastic syndrome, 1 lymphoma, 1 metastatic lung cancer). Conclusion: Patients with POEMS syndrome who undergo ASCT have a significant and meaningful improvement of their neuropathy by multiple measurements during both short and long-term follow-up, which corresponds to reduction in morbidity and disability (none are in wheelchair long-term). Periengraftment syndrome was common but manageable. Fatal complications, although rare, did occur, usually in association with other malignancies. Classification of evidence: This study provides Class IV evidence that for patients with POEMS syndrome, ASCT improves neuropathy-related function.

Original languageEnglish (US)
Pages (from-to)1981-1987
Number of pages7
JournalNeurology
Volume84
Issue number19
DOIs
StatePublished - May 12 2015
Externally publishedYes

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POEMS Syndrome
Polyneuropathies
Stem Cell Transplantation
Wheelchairs
Braces
Action Potentials
Foot
Median Neuropathy
Myelodysplastic Syndromes
Vital Capacity

ASJC Scopus subject areas

  • Clinical Neurology

Cite this

Karam, C., Klein, C. J., Dispenzieri, A., Dyck, P. J. B., Mandrekar, J., D'Souza, A., & Mauermann, M. L. (2015). Polyneuropathy improvement following autologous stem cell transplantation for POEMS syndrome. Neurology, 84(19), 1981-1987. https://doi.org/10.1212/WNL.0000000000001565

Polyneuropathy improvement following autologous stem cell transplantation for POEMS syndrome. / Karam, Chafic; Klein, Christopher J.; Dispenzieri, Angela; Dyck, P. James B; Mandrekar, Jay; D'Souza, Anita; Mauermann, Michelle L.

In: Neurology, Vol. 84, No. 19, 12.05.2015, p. 1981-1987.

Research output: Contribution to journalArticle

Karam, C, Klein, CJ, Dispenzieri, A, Dyck, PJB, Mandrekar, J, D'Souza, A & Mauermann, ML 2015, 'Polyneuropathy improvement following autologous stem cell transplantation for POEMS syndrome', Neurology, vol. 84, no. 19, pp. 1981-1987. https://doi.org/10.1212/WNL.0000000000001565
Karam, Chafic ; Klein, Christopher J. ; Dispenzieri, Angela ; Dyck, P. James B ; Mandrekar, Jay ; D'Souza, Anita ; Mauermann, Michelle L. / Polyneuropathy improvement following autologous stem cell transplantation for POEMS syndrome. In: Neurology. 2015 ; Vol. 84, No. 19. pp. 1981-1987.
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abstract = "Objective: To study the evolution of the neuropathy and long-term disability in a large cohort of patients with POEMS (polyneuropathy, organomegaly, endocrinopathy, M protein, and skin changes) syndrome following autologous stem cell transplantation (ASCT). Methods: Retrospective chart review documenting the clinical, electrophysiologic, and laboratory characteristics of patients with POEMS syndrome undergoing ASCT at Mayo Clinic, Rochester. Results: Sixty patients with a median follow-up time of 61 months were studied. All patients had peripheral polyneuropathy and demonstrated neurologic improvement after ASCT (apart from one patient who died early). Before ASCT, 27 patients (45{\%}) required a wheelchair and 17 (29{\%}) required a walker or foot brace. At the end of the follow-up period, no patient was using a wheelchair and 23 patients (38{\%}) were using a foot brace. The median Neuropathy Impairment Score improved from 66 to 48 points at 12 months and to 30 points at most recent follow-up (p < 0.0001). Median Rankin Scale score improved from 3 to 1.5 (p < 0.0001). Vascular endothelial growth factor levels decreased from a median of 452 to 63.5 pg/mL (p < 0.0001). The ulnar compound motor action potential amplitude (median) improved from 4.3 to 7.6 mV (p < 0.0001) and ulnar compound motor action potential conduction velocity (median) improved from 34 to 51 m/s (p < 0.0001). Predicted forced vital capacity improved from 81{\%} to 88{\%} (p < 0.0001). Periengraftment syndrome occurred in 24 patients. Fourteen patients required additional chemotherapy and/or radiation following ASCT, but there was no clinical deterioration in the neuropathy in any of these patients. Six patients died: 1 due to POEMS, 1 due to failed engraftment, and 4 due to other malignancies (2 myelodysplastic syndrome, 1 lymphoma, 1 metastatic lung cancer). Conclusion: Patients with POEMS syndrome who undergo ASCT have a significant and meaningful improvement of their neuropathy by multiple measurements during both short and long-term follow-up, which corresponds to reduction in morbidity and disability (none are in wheelchair long-term). Periengraftment syndrome was common but manageable. Fatal complications, although rare, did occur, usually in association with other malignancies. Classification of evidence: This study provides Class IV evidence that for patients with POEMS syndrome, ASCT improves neuropathy-related function.",
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