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Johannesen, Siw Wollebæk ; Huie, J. Russell ; Budeus, Bettina ; Peters, Sebastian ; Wirth, Anna M. ; Iberl, Sabine ; Kammermaier, Tina ; Kobor, Ines ; Wirkert, Eva ; Küspert, Sabrina ; Tahedl, Marlene ; Grassinger, Jochen ; Pukrop, Tobias ; Schneider, Armin ; Aigner, Ludwig ; Schulte-Mattler, Wilhelm ; Schuierer, Gerhard ; Koch, Winfried ; Bruun, Tim-Henrik ; Ferguson, Adam R. ; Bogdahn, Ulrich

Modeling and Bioinformatics Identify Responders to G-CSF in Patients With Amyotrophic Lateral Sclerosis

Article

Johannesen, Siw Wollebæk, Huie, J. Russell, Budeus, Bettina, Peters, Sebastian, Wirth, Anna M., Iberl, Sabine, Kammermaier, Tina, Kobor, Ines, Wirkert, Eva, Küspert, Sabrina, Tahedl, Marlene, Grassinger, Jochen, Pukrop, Tobias, Schneider, Armin, Aigner, Ludwig, Schulte-Mattler, Wilhelm , Schuierer, Gerhard, Koch, Winfried, Bruun, Tim-Henrik, Ferguson, Adam R. and Bogdahn, Ulrich (2021) Modeling and Bioinformatics Identify Responders to G-CSF in Patients With Amyotrophic Lateral Sclerosis. Frontiers in Neurology 12 (616289), pp. 1-5.

DOI to cite this document: 10.5283/epub.45256


Abstract

Objective: Developing an integrative approach to early treatment response classification using survival modeling and bioinformatics with various biomarkers for early assessment of filgrastim (granulocyte colony stimulating factor) treatment effects in amyotrophic lateral sclerosis (ALS) patients. Filgrastim, a hematopoietic growth factor with excellent safety, routinely applied in oncology and ...

Objective: Developing an integrative approach to early treatment response classification using survival modeling and bioinformatics with various biomarkers for early assessment of filgrastim (granulocyte colony stimulating factor) treatment effects in amyotrophic lateral sclerosis (ALS) patients. Filgrastim, a hematopoietic growth factor with excellent safety, routinely applied in oncology and stem cell mobilization, had shown preliminary efficacy in ALS. Methods: We conducted individualized long-term filgrastim treatment in 36 ALS patients. The PRO-ACT database, with outcome data from 23 international clinical ALS trials, served as historical control and mathematical reference for survival modeling. Imaging data as well as cytokine and cellular data from stem cell analysis were processed as biomarkers in a non-linear principal component analysis (NLPCA) to identify individual response. Results: Cox proportional hazard and matched-pair analyses revealed a significant survival benefit for filgrastim-treated patients over PRO-ACT comparators. We generated a model for survival estimation based on patients in the PRO-ACT database and then applied the model to filgrastim-treated patients. Model-identified filgrastim responders displayed less functional decline and impressively longer survival than non-responders. Multimodal biomarkers were then analyzed by PCA in the context of model-defined treatment response, allowing identification of subsequent treatment response as early as within 3 months of therapy. Strong treatment response with a median survival of 3.8 years after start of therapy was associated with younger age, increased hematopoietic stem cell mobilization, less aggressive inflammatory cytokine plasma profiles, and preserved pattern of fractional anisotropy as determined by magnetic resonance diffusion tensor imaging (DTI-MRI). Conclusion: Long-term filgrastim is safe, is well-tolerated, and has significant positive effects on disease progression and survival in a small cohort of ALS patients. Developing and applying a model-based biomarker response classification allows use of multimodal biomarker patterns in full potential. This can identify strong individual treatment responders (here: filgrastim) at a very early stage of therapy and may pave the way to an effective individualized treatment option.



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Details

Item typeArticle
Journal or Publication TitleFrontiers in Neurology
PublisherFrontiers
Open Access TypeGold (with APC)
Place of PublicationLAUSANNE
Volume12
Number of Issue or Book Chapter616289
Page Rangepp. 1-5
Date18 March 2021
Date of publication18 Mar 2021 17:22
InstitutionsMedicine > Lehrstuhl für Innere Medizin III (Hämatologie und Internistische Onkologie)
Medicine > Lehrstuhl für Neurologie
Medicine > Lehrstuhl für Psychiatrie und Psychotherapie
Medicine > Zentrum für Neuroradiologie
Identification Number
ValueType
10.3389/fneur.2021.616289DOI
Keywords; amyotrophic lateral sclerosis; granulocyte-colony stimulating factor (G-CSF); filgrastim; biomarker; modeling; principal component analysis (PCA); cytokines; stem cell
Dewey Decimal Classification600 Technology > 610 Medical sciences Medicine
StatusPublished
RefereedYes, this version has been refereed
Created at the University of RegensburgYes
URN of the UB Regensburgurn:nbn:de:bvb:355-epub-452566
Item ID45256

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