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Objective:To investigate clinical efficacy and safety of venetoclax (VEN)-based regimens in the treatment of acute myeloid leukemia (AML).Methods:The clinical data of 41 AML patients treated with venetoclax-based regimens from January 2021 to December 2021 in Ruijin Hospital North of Shanghai Jiao Tong University School of Medicine were retrospectively analyzed. The treatment regimens included VEN+demethylating drugs ± gene mutation inhibitors or VEN+chemotherapy with a median number of 2 courses (1- 5 courses).Results:The median age of all patients was 60 years (18-73 years), and there were 24 males and 17 females. After 1 course of VEN-based therapy, 22 (53.7%) patients achieved complete remission (CR) or morphological complete remission without complete blood count recovery (CRi), including 5 patients achieving minimal residual disease (MRD) negative. After 2 courses of treatment, of 17 patients available for efficacy evaluation, 7 patients achieved MRD negative. Among 20 relapsed/refractory AML patients, 9 cases achieved CR/CRi after 1 course of treatment, of which 1 patient had MRD negative. Among 21 patients initially treated and re-treated, 13 cases achieved CR/CRi and 1 case achieved partial remission after 1 course of treatment, of which 4 cases had MRD negative.Conclusions:VEN-based treatment regimens for AML have a high remission rate and tolerable adverse effects.
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Objective To analyze the therapeutic effect and tolerability of decitabine monotherapy or combined with arsenic trioxide for the treatment of patients with myelodysplastic syndromes (MDS). Methods Clinical characteristics of 32 patients with primary MDS in North Hospital of Ruijin Hospital Affiliated to Medical College of Shanghai Jiaotong University from January 2014 to April 2017 were retrospectively analyzed. The clinical data of these patients were collected, and the patients were followed up. Decitabine combined with arsenic trioxide was used in 23 cases, decitabine (20 mg·m-·2d-1) and arsenic trioxide (0.16 mg/kg) were administrated from day 1 to day 5 and was repeated every 4-6 weeks. For the remaining 9 cases, only decitabine was applied, decitabine(20 mg·m-2·d-1) was administrated from day 1 to day 5 and was repeated every 4-6 weeks. The clinicopathological characteristics and the effect of genetic mutations on the efficacy of treatment were investigated. Results Of the 32 patients with primary MDS, 18 were male and 14 were female. The patients were 17-72 years old with a median age of 56 years old. Genetic analysis revealed 10 cases with TP53 mutations, 8 cases with TET2 mutations, 4 cases with U2AF1 mutations, 3 cases with RUNX1 mutations, 3 cases with ASXL1 mutations, 2 cases with NRAS mutations, 2 cases with DNMT3A mutations and 1 case with JAK2 V617 mutation. The follow﹣up time was 2-23 months with a median follow﹣up time of 8 months. A total of 21 cases (65.6%) attained treatment response. Among them, there were 10 cases (31.3%) with complete remission (CR), 5 cases (15.6%) with bone marrow complete remission (MCR), and 6 cases (18.7%) with hematological improvement. There was no significant difference in the efficiency and CR rate between the combination group and the monotherapy group (P＝0.441, P＝0.681). Ten cases were found to have TP53 mutations, of which 7 cases had CR. Multivariate analysis demonstrated that TP53 mutation was an independent risk factor for CR (P＝ 0.037). All patients developed myelosuppression after treatment, of which 16 cases developed pulmonary infection. Conclusions Decitabine combined with arsenic trioxide in the treatment of MDS is effective and well tolerated. The therapeutic effects of decitabine monotherapy or decitabine combined with arsenic trioxide for treatment of patients with TP53 mutations are better than the average levels.
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Objective To observe any differences in motor cortex excitability between Parkinson's disease (PD)patients and patients with multiple system atrophy (MSA) and to explore whether motor evoked potentials (MEPs) can be used as an electrophysiological indicator for differentiating the 2 diseases.Methods Thirty-four PD patients, 22 MSA patients and 15 age- and sex-matched healthy control subjeets were included in this study. Relaxed motor thresholds (RMTs), central motor conduction time (CMCTs) and MEP amplitudes (AMPs) were recorded in all three groups. The relationships of RMT, CMCT and AMP with the severity of the disease were observed.Results Average RMT in the PD group was significantly lower than that in the MSA and control groups. Average RMT in the MSA group was also significantly lower than that in the control group. There was no significant difference among the three groups with regard to CMCT. AMP in the PD group was significantly higher on average than in the MSA and control groups, but there was no significant difference between the MSA and control groups. RMT decreased and CMCT shortened progressively with the severity of the disease in the PD group, but not in the MSA group.Conclusions There were differences in motor cortex excitability between PD patients and MSA patients. MEP RMTs and CMCTs may be valuable for identifying PD and MSAc but the clinical significance of the amplitude differences remains to be further explored.
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Objective To evaluate the changes in and the regularity of brainstem evoked potentials (BA-EPs) in Parkiuson's disease (PD) as an objective criterion for early diagnosis and assessment. Methods Thirty-five healthy SD rats were divided into two groups at random. Twenty-two rats were in the experimental group and 13 in the control group. The rats were injected with 8 μg of 6-OHDA solution in the right substantia nigra pars compacta (SNc) and the right ventral tegmentum area (VTA) to create a PD model. The BAEPs of the rats in the experimental group were recorded in a quiet shielded room before the 6-OHDA injection, and one week and two weeks after injec-tion. The control group rats were injected with saline (Ns) and their BAEPs were recorded at the corresponding times. One week and two weeks later, the model rats were injected with apomorphine (APO) and their rotating cycles were counted. Results The Ⅱ , Ⅳ, andV PLs and the Ⅲ-Ⅴ IPLs on the fight ears of the experimental group were prolonged significantly compared with the control group one week after APO injection. There was no significant differ-ence in the BAEPs of the left ears after the first week. After two weeks, the Ⅱ , Ⅳ, and Ⅴ PLs and the Ⅲ-Ⅴ, and Ⅰ-Ⅴ IPLs of the right ears in the experimental group were prolonged significantly compared with the controls and the Ⅳ, and Ⅴ PLs and the Ⅲ -Ⅴ , and Ⅰ-Ⅴ IPLs on their left ears were prolonged significantly. Conclusion In the early course of a PD model in rats, their BAEPs show abnormal changes, which indicates that BAEP could be an ob-jective criterion for early diagnosis and assessment of PD. BAEP may serve as one index of damage in PD. The Ⅲ-Ⅴ PL and Ⅰ-Ⅴ iPL are sensitive indices of PD.
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Objective To investigate the effects of low frequency repetitive transcranial magnetic stimulation (rTMS) on motor function and excitability of motor cortex in Parkinson's disease (PD) patients and to study the mechanism of PD from the electrophysiology. Methods Twenty-eight patients with PD received 1 Hz rTMS therapy for 15 d. Thirty normal volunteers were enrolled as controls. Unified Parkinson's Disease Rating Scale (UPDRS) and motor evoked potential (MEP) were adopted as assessment indicators. The excitability of motor cortex was assessed by rest motor threshold (RMT), central motor conduction time (CMCT) and the amplitude of MEP. Results The initial RMTs and CMCTs of PD patients were significantly lower than those of the controls, but MEP amplitudes were not significantly different. After rTMS treatment, motor function of PD patients improved, RMTs increased and CMCTs prolonged. Conclusion In PD patients, motor function disorder and increased motor cortical excitability were observed. Low frequency rTMS may inhibit these changes to some extent.
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Objective To study the effects of repeated transcranial magnetic stimulation(rTMS)on Parkinson's plus syndrome(PPS).Methods Fifteen in-patients with PPS were studied between 2005 and 2008.The patients received 1 Hz rTMS at an intensity 30%over the threshold.The rTMS was applied on the hand representive area of the bilateral first motor cortex,50 stimulations on each side,5 arrays,for 5 min,once daily for 15 d.Hamilton's depression scale(HAMD),Hamilton's anxiety scale(HAMA),the unified Parkinson's disease rating scale(UPDRS,which can be subdivided into UPDRS Ⅰ,UPDRS Ⅱ and UPDRS Ⅲ),an activities of daily living scale(ADL),the mini-mental state examination(MMSE)and motor evoked potential(MEP)were assessed before and immediately after 15 d of rTMS treatment. Results Average HAMD,HAMA,UPDRS,UPDRS Ⅱ and UPDRS Ⅲ scores all decreased,and ADL scores increased significantly after treatment,while UPDRSⅠand MMSE scores were unchanged before and after treatment.No significant changes in resting motor threshold or central motor conduction time of the MEP were observed after rTMS treatment. Conclusion Clinical symptoms of PPS patients improved after rTMS treatment and side effects were few.Depression,anxiety,motor function and ability in the activities of daily living improved greatly.Repeated transcranial magnetic stimulation is a potential treatment for PPS patients.There may be no correlation between the effective mechanism of rTMS and cortex excitation.