1.Application of autologous peripheral blood stem cell transplantation in children with malignanttumor
SQ TANG ; DS HUANG ; JW WANG ; XJ WEI ; CR RAN ; Y PENG ; SG LU ; JZ ZHANG
Chinese Medical Journal 2001;114(10):1098-1101
Objective To investigate if low dose total body irradiation (TBI, 6.0- 9.0 Gy) combined with intensified chemotherapy followed by autologous peripheral blood stem cell transplantation results in better survival in children with refractory leukemia or solid tumors. Methods Twenty-one children with malignant tumors were included in this study. There were 14 males and 7 females aged 3.5- 12 years. Underlying disease included high-risk acute lymphoblastic leukemia (ALL, CR1 in 3 children and CR2 in 5 children), acute myeloblastic leukemia (AML, 9 children), non Hodgkin' s lymphoma stage Ⅳ (2 children), and neuroblastoma stage Ⅳ (2 children). The peripheral hematopoietic stem cells were collected six to eleven months after complete response, mobilized with high dose chemotherapy alone or combined with GM-CSF or G-CSF. The conditioning regimen consisted of chemotherapy with two to three combinations of the following drugs: cyclophosphamide, arabinosylcytosine, McNU, etopside, and Idarubicin on the basis of TBI (6.0-9.0Gy). A mean of (1.8 ± 0.5) × 108/kg autologous mononuclear cells were transplanted. The patients were followed up after transplantation. Results Severe bone marrow suppression occurred in all patients around day + 7. Peripheral white blood cell count decreased to 0 in all patients at day + 4.8 ± 2.9, and platelet count decreased to less than 20× 109/L at day + 9.0 ± 2.6. Successful engraftment was achieved in 21 patients, but four died of infection at day + 17, + 20, + 31 and + 67, respectively. Recovery of white blood cell (WBC) to 10 × 109/L, absolute neutrophil count to 0.5 × 109/L, platelet count to 20 × 109/L occurred on 21 ± 12, 26± 13, and 27 ± 10 days, respectively. During the follow up period, three patients relapsed at + 5 months, + 1.5 years, and + 2 years 10 months, respectively. One patient died of intracranial hemorrhage at +8 months. Thirteen patients had event-free survival for 2 - 12 years, with a mean of 6.7±3.4 years. Conclusion Our preliminary data suggest that myeloablative therapy with low dose TBI (6.0 - 9.0 Gy) combined with intensified chemotherapy followed by autologous paripheral blood stem cell transplantation might be associated with favorable results in children with refractory leukemia or solid tumors.
2.Interferon beta-1b is effective and has a favourable safety profile in Chinese patients with relapsing forms of multiple sclerosis
ZX Li ; CZ Lu ; XH Zhang ; LY Cui ; XH Xu ; E Radue ; SG Chu ; LR Wang ; K Beckmann ; J Lampe ; C Pohl ; B Stemper ; R Sandbrink
Neurology Asia 2014;19(2):179-189
Background & Objective: No clinical study of any interferon beta therapy has yet been successfully conducted in Chinese multiple sclerosis patients, probably due to the low incidence of this disease in China. The primary objective of this study was to demonstrate that treating multiple sclerosis patients of Chinese origin with interferon beta-1b has a beneficial effect on disease course, as measured by the decrease of newly active lesions on magnetic resonance imaging. Methods: Chinese patients diagnosed with relapsing-remitting or secondary-progressive multiple sclerosis were enrolled in this multicenter, open label, single-arm study. Following a 3-month pre-treatment phase, patients were treated with 250 µg interferon beta-1b subcutaneously every other day for 6 months. Patients had regular assessments for treatment safety and efficacy of the treatment. Results: Thirty seven patients completed the trial. Significant decreases in the number of newly active lesions were observed in the 6-month treatment period compared with the pre-treatment period (median decrease 1.5 lesions, p<0.001). Most adverse events were mild and transient and no serious ones were observed. Conclusions: Treatment with interferon beta-1b significantly reduced the occurrence of new lesions and was well tolerated in this Chinese population. These findings support the use of interferon beta- 1b for treating Chinese MS patients.