title: 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.
  
  
  METHODS
  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.affiliation: Department of Neurology, Clinical Neuroscience, Karolinska Institutet, Karolinska University Hospital Huddinge, , Stockholm, Huddinge, Sweden.
authors.forename: R
authors.initials: R
authors.lastname: Press
authors.forename: H
authors.initials: H
authors.lastname: Askmark
authors.forename: A
authors.initials: A
authors.lastname: Svenningsson
authors.forename: O
authors.initials: O
authors.lastname: Andersen
authors.forename: H W
authors.initials: HW
authors.lastname: Axelson
authors.forename: U
authors.initials: U
authors.lastname: Strömberg
authors.forename: A
authors.initials: A
authors.lastname: Wahlin
authors.forename: C
authors.initials: C
authors.lastname: Isaksson
authors.forename: J E J
authors.initials: JE
authors.lastname: Johansson
authors.forename: H
authors.initials: H
authors.lastname: Hägglund
mesh_terms: D000293:Adolescent; D000305:Adrenal Cortex Hormones / Q000008:administration & dosage; D000328:Adult; D000368:Aged; D003556:Cystitis / Q000175:diagnosis; D003586:Cytomegalovirus Infections / Q000175:diagnosis; D020031:Epstein-Barr Virus Infections / Q000175:diagnosis; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods; D006801:Humans; D016756:Immunoglobulins, Intravenous / Q000008:administration & dosage; D008297:Male; D008875:Middle Aged; D010195:Pancreatitis / Q000175:diagnosis; D010951:Plasma Exchange; D020277:Polyradiculoneuropathy, Chronic Inflammatory Demyelinating / Q000503:physiopathology / Q000601:surgery*; D012008:Recurrence; D012086:Reoperation; D012189:Retrospective Studies; D013548:Sweden; D019172:Transplantation Conditioning; D014182:Transplantation, Autologous; D016896:Treatment Outcome
chemical_list: D000305:Adrenal Cortex Hormones; D016756:Immunoglobulins, Intravenous
keywords: Neuroimmunology; Neuromuscular; NeuropathY; Neurophysiology