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Research paper
Autologous haematopoietic stem cell transplantation: a viable treatment option for CIDP
  1. R Press1,
  2. H Askmark2,
  3. A Svenningsson3,
  4. O Andersen4,
  5. H W Axelson5,
  6. U Strömberg6,
  7. A Wahlin7,
  8. C Isaksson7,
  9. J-E J Johansson8,
  10. H Hägglund9
  1. 1Department of Neurology, Clinical Neuroscience, Karolinska Institutet, Karolinska University Hospital Huddinge, Stockholm, Huddinge, Sweden
  2. 2Department of Neuroscience, Neurology, Uppsala University Hospital, Uppsala, Sweden
  3. 3Department of Pharmacology and Clinical Neuroscience, Umeå University, Umeå, Sweden
  4. 4Institute of Neuroscience and Physiology, University of Gothenburg, Gothenburg, Sweden
  5. 5Department of Neuroscience, Clinical Neurophysiology, Uppsala University Hospital, Uppsala, Sweden
  6. 6Department of Medical Sciences, Uppsala University and Section of Hematology, Uppsala University Hospital, Uppsala, Sweden
  7. 7Department of Radiosciences, Umeå University, Umeå, Sweden
  8. 8Department of Hematology and Coagulation, Sahlgrenska University Hospital, Gothenburg, Sweden
  9. 9Department of Hematology, Karolinska Institutet, Karolinska University Hospital, Stockholm, Huddinge, Sweden
  1. Correspondence to Dr Rayomand Press, Department of Neurology, Karolinska University Hospital Huddinge, Stockholm, Huddinge SE-14186, Sweden; rayomand.press{at}ki.se

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.

  • NeuropathY
  • Neuromuscular
  • Neuroimmunology
  • Neurophysiology

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