A Brief Analysis Of The Therapeutic Efficacy And Safety In Autologous Peripheral Blood Stem Cells Transplantation For Treating Amyotrophic Lateral Sclerosis

Background and Objective: Amyotrophic lateral sclerosis is a progressive neurodegenerative disease affecting both upper and lower motor neurons of unknown actiology,characterized by progressive muscular weakness and atrophy,bulbar paralysis and pyramidal sign.This research aims to make a brief analysis of therapeutic efficacy and safety in autologous peripheral blood stem cells transplantation for treating amyotrophic lateral sclerosis.Materials and Methods::Collecting the clinical date of 22 patients in the First Affiliated Hospital of Dalian Medical University from June 2010 to December 2013 who have been diagnosed ALS according according to the revised El Escorial and neurophysiological criteria.22 ALS patients were enrolled in this study.These patients were randonmly assigned in a double-blind manner to receive either G-CSF(12 patients) or placebo(10 patients).Recombinant human granulocyte colony-stimulating factor Injection was injected subcutaneously at a dose of 5μg /kg/day for four consecutive days.Patients on placebo were injected similarly with equal normal saline solution.We chosen the operational procedures of the Spectra Auto PBSC for collecting autologous peripheral blood stem cells.These stem cells were injected into the subarachnoid space.This treatment was repeated every three months for a year.The index of evaluating efficacy including the revised ALS functional rating scale(ALSFRS-R) score,a forced vital capacity(FVC),manual muscle strength(MMT),compound muscle action potential(CMAP) amplitudes,distal motor latencies(DML), F-wave persistence,motor nerve conduction velocity(MCV),neurophysiological index(NI),McGill single item quality of life score(QoL).These outcome measures were analyzed by SPSS 21.0 to evaluate the therapeutic efficacy and safety in autologous peripheral blood stem cells transplantation for treating amyotrophic lateral sclerosis.Results: This study showed that G-CSF administration in ALS patients caused successful mobilization of autologous bone marrow cells.During the first six months,patients on G-CSF had a functional decrease of only 0.75 per month,while patients on placebo had a functional deterioration of 1.08 per month.It showed that the decreased rate of decline slowed down in ALSFRS-R of patients on G-CSF.However,this difference was not statistically significant(P>0.05).The same situation also occurred in the measure of the McGill quality of life score.During the six months, patients on G-CSF had a functional decrease of only 0.6 per month, patients on placebo had a functional deterioration of 1.5 per month, the difference was not statistically significant(P>0.05)。Compared with the placebo, patients on G-CSF showed lower decline of the decreased rate in FVC and the MMT score,but the differences between them were not statistically significant(P>0.05).During the three months and the six months after transplantation,only the compound muscle action potential(CMAP) amplitudes(increased) after six months transplantation showed statistically significant,other nerve electrophysiological data,including distal motor latencies(DML), F-wave persistence, motor nerve conduction velocity(MCV),neurophysiological index(NI),were not statistically significant(P>0.05).Conclusion: 1. The treatment was safe with almost no side-effects.The major adverse reactions were not observed in the treatment and follow-up.Some patients appeared mild adverse reactions,such as headache,lumbago and so on.After symptomatic treatment,these symptoms relieved or disappeared.2.G-CSF administration in ALS patients caused successful mobilization of bone marrow.3.The revised ALS functional rating scale(ALSFRS-R) score,a forced vital capacity(FVC),manual muscle strength(MMT) and McGill single item quality of life score(QoL) in the patients on G-CSF both showed lower decline of the decreased rate than them in the patients on placebo,but these differences between them were not statistically significant(P > 0.05). Compared with the patients on placebo,although the electrophysiological data related to the ALS,including compound muscle action potential(CMAP) amplitudes,distal motor latencies(DML), F-wave persistence,motor nerve conduction velocity(MCV),neurophysiological index(NI) both showed better in the patients on G-CSF,only the compound muscle action potential(CMAP) amplitudes(increased) during the six months showed statistically significant(P<0.05),This could be due to the small sample size or, an inadequate mobilization or, the unknown etiology and pathological mechanisms in ALS either directly or indirectly.4.Expanding the sample size and strengthening long-term follow-up may improve the accuracy of estimation of therapeutic efficacy in autologous peripheral blood stem cells transplantation for treating amyotrophic lateral sclerosis.