Rare diseases Xagena

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Chronic inflammatory demyelinating polyneuropathy: autologous haematopoietic stem cell transplantation, a viable treatment option

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 chronic inflammatory demyelinating polyneuropathy.

The results of AHSCT in 11 consecutive Swedish patients with therapy-refractory chronic inflammatory demyelinating polyneuropathy with a median follow-up time of 28 months, were described.

Case data were gathered retrospectively for AHSCT treatments in 11 patients with chronic inflammatory demyelinating polyneuropathy 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.

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.

In conclusion, the results though hampered by the limited number of patients and the lack of a control group suggest AHSCT to be efficacious in therapy-refractory chronic inflammatory demyelinating polyneuropathy, with a manageable complication profile.
Confirmation of these results is necessary through randomised controlled trials. ( Xagena )

Press R et al, J Neurol Neurosurg Psychiatry 2013 ; Epub ahead of print