Autologous Hematopoietic Stem Cell Transplantation for Paraneoplastic Cerebellar Degeneration

The aim of this study was to describe 2 patients with paraneoplastic cerebellar degeneration (PCD) treated with autologous hematopoietic stem cell transplantation (AHSCT).

Off-label AHSCT was performed at Hospital Clinic Barcelona, including stem cell mobilization (cyclophosphamide, filgrastim), plasma exchange, and a nonmyeloablative regimen (cyclophosphamide, antithymocyte globulin, rituximab [RTX]).

A 38-year-old woman developed anti-Yo-associated PCD 17 months after treatment of a gynecologic cancer (without evidence of tumor recurrence). Despite treatment with steroids and RTX, she became unable to walk. AHSCT resulted in progressive improvement, no longer needing assistance to walk 9 months after AHSCT. Tumor recurrence, without neurologic worsening, was identified 16 months after AHSCT, and chemotherapy was restarted. At the last follow-up (46 months), she continues on chemotherapy without neurologic deterioration. A 48-year-old man developed PCD associated with Tr/delta/notch-like epidermal growth factor-related receptor antibodies. Axillary lymph node biopsy demonstrated Hodgkin lymphoma. Chemoradiation resulted in complete tumor response, but cerebellar ataxia worsened despite treatment with steroids and IV immunoglobulins. Ten months after cerebellar symptom onset, he underwent AHSCT resulting in neurologic improvement. At the last follow-up, 39 months after AHSCT, he remains independent in activities of daily living.

In our experience, AHSCT is worth to be considered in patients with progressive PCD refractory to conventional oncological and immunotherapy treatments.

This is a single observational study without controls and provides Class IV evidence.