Abstract Details

Title Autologous Hemopoietic Stem Cell Transplantation for Paraneoplastic Cerebellar Degeneration

Topic Neuro-oncology

Presentation(s) P12 - Poster Session 12 (11:45 AM-12:45 PM)

Poster/Presentation
Number 6-003

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.

Authors/Disclosures
Jose Maria Cabrera Maqueda PRESENTER	Mr. Cabrera Maqueda has nothing to disclose.
Victor Guerra-Fernandez, MD	Mr. Guerra-Fernandez has nothing to disclose.
Mar Guasp, MD (Hospital Clínic Barcelona - IDIBAPS)	Dr. Guasp has nothing to disclose.
Elianet Fonseca, MD (HOSPITAL CLINICO DE BARCELONA)	Dr. Fonseca has nothing to disclose.
Juan Fran Luchoro Cerda	Mr. Luchoro Cerda has nothing to disclose.
Raquel Ruiz García, PhD	Dr. Ruiz García has nothing to disclose.
Yolanda Blanco Morgado, MD	Dr. Blanco Morgado has nothing to disclose.
Carmen Martínez, MD, PhD	Prof. Martínez has nothing to disclose.
Francesc F. Avilés, MD, PhD	Dr. Avilés has nothing to disclose.
María Suarez-Lledo, PhD	Mrs. Suarez-Lledo has nothing to disclose.
Francesc R. Graus, MD (IDIBAPS)	Dr. Graus has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for MedLink. Dr. Graus has received intellectual property interests from a discovery or technology relating to health care.
Montserrat Rovira, MD	Dr. Rovira has nothing to disclose.
Albert Saiz (Hospital Clinico De Barcelona)	Albert Saiz has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi. Albert Saiz has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Horizon Therapeutics. Albert Saiz has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Merck. Albert Saiz has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Janseen. Albert Saiz has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Roche. Albert Saiz has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for novartis. Albert Saiz has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Biogen.
Eugenia Martinez-Hernandez, MD (Hospital Clinic Barcelona)	Dr. Martinez-Hernandez has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Martinez-Hernandez has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB. The institution of Dr. Martinez-Hernandez has received research support from Instituto de Salud Carlos III. The institution of Dr. Martinez-Hernandez has received research support from Fundacio La Caixa. Dr. Martinez-Hernandez has received personal compensation in the range of $500-$4,999 for serving as a support for attending meetings and speaker honoraria with UCB. Dr. Martinez-Hernandez has received personal compensation in the range of $500-$4,999 for serving as a speaker honoraria and attending meetings with Alexion. Dr. Martinez-Hernandez has received personal compensation in the range of $500-$4,999 for serving as a support for attending meetings with CSL Behring.

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