Autologous Hemopoietic Stem Cell Transplantation for Paraneoplastic Cerebellar Degeneration
Victor Guerra-Fernandez1, Jose M Cabrera-Maqueda2, Mar Guasp2, Elianet Fonseca5, Juan Francisco Luchoro3, Raquel Ruiz-Garcia3, Yolanda Blanco Morgado2, Carmen Martinez4, Francesc Fernandez-Aviles4, Maria Suarez-Lledo4, Francesc Graus5, Montserrat Rovira4, Albert Saiz5, Eugenia Martinez-Hernandez2
1Department of Neurology, Hospital Clínic de Barcelona, University of Barcelona, Spain., 2Neuroimmunology Unit, Department of Neurology, Hospital Clínic de Barcelona, University of Barcelona, Spain., 3Department of Immunology, Hospital Clínic de Barcelona, Universitat de Barcelona, Spain, 4Hematopoietic Stem Cell Transplantation Unit, Hematology Department, Institute of Cancer and Hematological Diseases (ICAMS), IDIBAPS, Hospital Clinic of Barcelona, Barcelona, Spain, Hospital Clinic de Barcelona, 5Neuroimmunology Program, Institut d’Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS) – Caixa Research Institute, Hospital Clínic de Barcelona, Barcelona, Spain, Hospital Clínic Barcelona - IDIBAPS
Objective:
To describe the outcomes of two patients with paraneoplastic cerebellar degeneration (PCD) treated with autologous hematopoietic stem cell transplantation (AHSCT).
Background:
PCD is an immune-mediated disease induced by the underlying cancer. The antibodies most commonly identified are anti-Yo/PCA1 in gynecologic/breast cancer and anti-Tr/DNER in Hodgkin’s lymphoma. Most patients with anti-Yo/PCA1 do not respond to conventional treatments, and although those with anti-Tr/DNER have better outcomes, good functional recovery is exceptional. AHCST has shown high efficacy in some refractory immune-mediated diseases but has not yet been tried in paraneoplastic neurological diseases.
Design/Methods:
The protocol included mobilization of stem cells with cyclophosphamide and filgastrim, followed by plasma exchange and non-myeloablative regimen with cyclophosphamide, antithymocite globulin and rituximab. We assessed the clinical features and treatment response after AHSCT.
Results:
Patient 1: A 38-year-old woman, treated for ovarian cancer with surgery and chemotherapy, developed rapidly progressive cerebellar ataxia two years later. Yo/PCA1 antibodies were detected in serum and CSF, 6 months after onset of the ataxia. A whole-body PET-CT-scan resulted negative. Despite steroids and rituximab, she continued deteriorating (bilateral support to walk). AHSCT was performed at 8 months. She progressively improved (+9 months: unaided walk), and anti-Yo/PAC1 became negative (+15 months). She has remained stable for 30 months.
Patient 2: A 48-year-old male developed PCD and anti-Tr/DNER were detected in serum and CSF two months later. Despite steroids and immunoglobulins, neurological symptoms worsened (unilateral support). Axillary lymphadenopathies were detected and he was diagnosed with Hodgkin’s lymphoma, receiving chemotherapy and radiotherapy with complete metabolic response. AHSCT was performed at 7 months. He gradually improved and anti-Tr/DNER became negative (+8 months). He has remained independent for instrumental activities of daily living for 24 months.
Conclusions:
In our experience AHSCT was a safe, effective and long-lasting therapy. It may be considered as an early therapeutic option for patients with non-devastating PCD.
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