A phase I/IIa clinical trial of autologous hematopoietic stem cell transplantation in amyotrophic lateral sclerosis.

Autor: Lunetta C; NEMO Clinical Center Milano, Fondazione Serena Onlus, Piazza Ospedale Maggiore 3, 20162, Milan, Italy. christian.lunetta@centrocliniconemo.it.; Neurorehabilitation Department of Milano Institute, Istituti Clinici Scientifici Maugeri IRCCS, Milan, Italy. christian.lunetta@centrocliniconemo.it., Lizio A; NEMO Clinical Center Milano, Fondazione Serena Onlus, Piazza Ospedale Maggiore 3, 20162, Milan, Italy., Cabona C; Department of Neuroscience, IRCCS Ospedale Policlinico San Martino, Genova, Italy., Gerardi F; NEMO Clinical Center Milano, Fondazione Serena Onlus, Piazza Ospedale Maggiore 3, 20162, Milan, Italy., Sansone VA; NEMO Clinical Center Milano, Fondazione Serena Onlus, Piazza Ospedale Maggiore 3, 20162, Milan, Italy.; Neurorehabilitation Unit, University of Milan, Milan, Italy., Corbo M; Department of Neurorehabilitation Sciences, Casa Cura Policlinico (CCP), Milan, Italy., Scialò C; Department of Quantitative Biomedicine, University of Zurich, Zurich, Switzerland., Angelucci E; Hematology and Cell Therapy, IRCCS Ospedale Policlinico San Martino, Genoa, Italy., Gualandi F; Hematology and Cell Therapy, IRCCS Ospedale Policlinico San Martino, Genoa, Italy., Marenco P; Division of Haematology, ASST GOM Niguarda, Milan, Italy., Grillo G; Division of Haematology, ASST GOM Niguarda, Milan, Italy., Cairoli R; Division of Haematology, ASST GOM Niguarda, Milan, Italy., Cesana C; Department of Laboratory Medicine, ASST GOM Niguarda, Milan, Italy., Saccardi R; Cell Therapy and Transfusion Medicine Unit, Careggi University Hospital, Florence, Italy., Melazzini MG; Istituti Clinici Scientifici Maugeri, Pavia, Italy., Mancardi G; Istituti Clinici Scientifici Maugeri, Pavia, Italy.; Department of Neurosciences, Rehabilitation, Ophtalmology, Genetics, Maternal and Child Health, University of Genoa, Genoa, Italy., Caponnetto C; Department of Neurosciences, Rehabilitation, Ophtalmology, Genetics, Maternal and Child Health, University of Genoa, Genoa, Italy.
Jazyk: angličtina
Zdroj: Journal of neurology [J Neurol] 2022 Oct; Vol. 269 (10), pp. 5337-5346. Date of Electronic Publication: 2022 May 21.
DOI: 10.1007/s00415-022-11185-w
Abstrakt: Objective: To verify the safety and potential effect on ALS progression of a low-intensity immunosuppressive regimen followed by autologous hematopoietic stem cell transplantation (aHSCT) in amyotrophic lateral sclerosis (ALS) patients.
Methods: ALS eligible patients underwent a set of clinical and laboratory evaluations at T-4 (screening), T-1 (pre-treatment visit), and for the 12 consecutive months after treatment (T3, T6, T9, T12). We evaluated the tolerability of the procedure, its efficacy on clinical course and quality of life (QoL).
Results: Eight of the 11 ALS patients enrolled received the established immunoablative protocol. The procedure was well tolerated and side effects were those expected. One patient died 4 months after the conditioning regimen and another patient underwent tracheotomy just before T3 for a sudden respiratory failure, but he is still alive 4 years after the procedure without being ventilated any more. A third patient died 10 months after conditioning. In the other cases, there was no statistical difference in all functional measures and QoL pre- and post-treatment; however, a transitory slopes' reduction of ALSFRS-R and seated SVC% after the conditioning procedures was reported. Moreover, although not statistically significant, trends of reduction of CD4 + and increment of CD8 + were found.
Conclusions: aHSCT was overall well tolerated, but it was not followed by any significant modification in disease progression. Considering the negative results of this small trial, further studies aimed to evaluate the possible efficacy of the aHSCT using a higher-intensity regimen should be carefully and with caution evaluated.
(© 2022. The Author(s), under exclusive licence to Springer-Verlag GmbH Germany.)
Databáze: MEDLINE
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