Autologous haematopoietic stem cell transplantation: a viable treatment option for CIDP.

Abstract

OBJECTIVE:

Only 70-80% of patients with chronic inflammatory demyelinating polyneuropathy (CIDP) respond satisfactorily to the established first-line immunomodulatory treatments. Autologous haematopoietic stem cell transplantation (AHSCT) has been performed as a last treatment resort in a few therapy-refractory cases with CIDP. We describe the results of AHSCT in 11 consecutive Swedish patients with therapy-refractory CIDP with a median follow-up time of 28 months.

METHOD:

Case data were gathered retrospectively for AHSCT treatments in 11 patients with CIDP refractory to the first-line immunomodulatory treatments, intravenous high-dose immunoglobulin, corticosteroids and plasma exchange and to one or more second-line treatments used in 10 of the 11 patients.

RESULTS:

The median Inflammatory Neuropathy Cause and Treatment (INCAT) score within 1 month prior to AHSCT was 6 and the Rankin score 4. Total INCAT and Rankin scores improved significantly within 2-6 months after AHSCT and continued to do so at last follow-up. The motor action potential amplitudes (CMAP) improved already within 4 months (median) after AHSCT. Three of the 11 patients relapsed during the follow-up period, requiring retransplantation with AHSCT in one. Eight of the 11 patients maintained drug-free remission upon last follow-up. AHSCT was safe but on the short term associated with a risk of cytomegalovirus (CMV) and Epstein-Barr virus reactivation, CMV disease, haemorrhagic cystitis and pancreatitis.

CONCLUSIONS:

Our results though hampered by the limited number of patients and the lack of a control group suggest AHSCT to be efficacious in therapy-refractory CIDP, with a manageable complication profile. Confirmation of these results is necessary through randomised controlled trials.

• Authors: R Press, H Askmark, A Svenningsson, O Andersen, H W Axelson, U Strömberg, A Wahlin, C Isaksson, J E J Johansson, H Hägglund
• Journal: Journal of neurology, neurosurgery, and psychiatry (J Neurol Neurosurg Psychiatry) Vol. 85 Issue 6 Pg. 618-24 (Jun 2014) ISSN: 1468-330X [Electronic] England
• PMID: 24262917 (Publication Type: Journal Article)

Chemical References

• Adrenal Cortex Hormones
• Immunoglobulins, Intravenous

Topics

• Adolescent
• Adrenal Cortex Hormones (administration & dosage)
• Adult
• Aged
• Cystitis (diagnosis)
• Cytomegalovirus Infections (diagnosis)
• Epstein-Barr Virus Infections (diagnosis)
• Female
• Hematopoietic Stem Cell Transplantation (methods)
• Humans
• Immunoglobulins, Intravenous (administration & dosage)
• Male
• Middle Aged
• Pancreatitis (diagnosis)
• Plasma Exchange
• Polyradiculoneuropathy, Chronic Inflammatory Demyelinating (physiopathology, surgery)
• Recurrence
• Reoperation
• Retrospective Studies
• Sweden
• Transplantation Conditioning
• Transplantation, Autologous
• Treatment Outcome