Autologous stem cell transplantation – a therapeutic option in childhood onset chronic inflammatory demyelinating polyneuroradiculopathy?

U Gruber-Sedlmayr; M Brunner-Krainz; M Müller; A Schwerin-Nagel; A Kortschak; S Prager-Puntigam; W Schwinger; MG Seidel; E Sorantin; C Mache

doi:10.1055/s-0033-1337787

Autologous stem cell transplantation – a therapeutic option in childhood onset chronic inflammatory demyelinating polyneuroradiculopathy?

U Gruber-Sedlmayr ¹, M Brunner-Krainz ¹, M Müller ¹, A Schwerin-Nagel ¹, A Kortschak ¹, S Prager-Puntigam ¹, W Schwinger ¹, MG Seidel ¹, E Sorantin ², C Mache ¹

¹Universitätsklinik für Kinder- und Jugendheilkunde Graz, Graz, Austria
²Uni-Klinik für Radiologie, Department Kinderradiologie, Graz, Austria

Abstract

Introduction: Childhood onset chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is a rare immune-mediated disorder presenting as mainly motor neuropathy with relapsing remitting or progressive course. Childhood CIDP prognosis is mostly favorable, but severe neurological deficits or a course without sustained remission are possible. In the past 10 years, many patients with severe autoimmune diseases underwent a hematopoietic stem cell transplantation (HSCT) with durable remissions in approximately 30%. But there are only some patients with CIDP described, who were treated with HSCT.

Case report: At the age of nearly 10 years, a boy presented with insidious gait problems. CSF protein was augmented and intravenous immunoglobulin (IVIG) was given two times, but he lost ambulation over the next 6 months. Now therapy with corticoids, plasma exchanges (PE) and IVIG was started and the patient improved, but suffered from hydrocephalus malresorptivus and a shunt was implanted. To spare corticoids, cyclosporine was added and the patient was stable for years under low dose corticoid and cyclosporine. Because of nephrotoxicity a change to rituximab and mycophenolate mofetil was tried without benefit. After relapse and nearly loss of ambulation, the therapy was changed to azathioprine, steroids, PE and IVIG. After 14 years of course of the disease, the patient decided to be treated with an autologous stem cell transplantation.

Conclusion: Although prognosis of CIDP in children and juvenile patients is mostly favorable, some patients are resistant to immunomodulatory therapies or suffer from side effects. For these patients, autologous stem cell transplantation might be an option.