Safety and feasibility of long term administration of recombinant human granulocyte-colony stimulating factor in patients with amyotrophic lateral sclerosis

Grassinger, Jochen and Khomenko, Andrei and Hart, Christina and Baldaranov, Dobri and Johannesen, Siw W. and Mueller, Gunnar and Schelker, Roland and Schulte-Mattler, Wilhelm and Andreesen, Reinhard and Bogdahn, Ulrich (2014) Safety and feasibility of long term administration of recombinant human granulocyte-colony stimulating factor in patients with amyotrophic lateral sclerosis. CYTOKINE, 67 (1). pp. 21-28. ISSN 1043-4666, 1096-0023

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Abstract

Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neuronal disease resulting in a loss of the upper and lower motor neurons and subsequent death within three to four years after diagnosis. Mouse models and preliminary human exposure data suggest that the treatment with granulocyte-colony stimulating factor (G-CSF) has neuro-protective effects and may delay ALS progression. As data on long-term administration of G-CSF in patients with normal bone marrow (BM) function are scarce, we initiated a compassionate use program including 6 ALS patients with monthly G-CSF treatment cycles. Here we demonstrate that G-CSF injection was safe and feasible throughout our observation period up to three years. Significant decrease of mobilization efficiency occurred in one patient and a loss of immature erythroid progenitors was observed in all six patients. These data imply that follow-up studies analyzing BM function during long-term G-CSF stimulation are required. (C) 2014 Elsevier Ltd. All rights reserved.

Item Type: Article
Uncontrolled Keywords: HEMATOPOIETIC PROGENITOR CELLS; G-CSF; PERIPHERAL-BLOOD; BONE-MARROW; STEM-CELLS; HEALTHY DONORS; MESSENGER-RNA; MOUSE MODEL; MOBILIZATION; LEUKEMIA; Amyotrophic lateral sclerosis; ALS; Granulocyte-colony stimulating factor; G-CSF
Subjects: 600 Technology > 610 Medical sciences Medicine
Divisions: Medicine > Lehrstuhl für Innere Medizin II
Medicine > Lehrstuhl für Neurologie
Depositing User: Dr. Gernot Deinzer
Date Deposited: 13 Nov 2019 09:59
Last Modified: 13 Nov 2019 09:59
URI: https://pred.uni-regensburg.de/id/eprint/10255

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