Tovorafenib has been approved by the U.S.Food and Drug Administration(FDA)for the treatment of patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma(LGGs)harboring the serine threonine kinae v-RAF murine sarcoma viral oncogene homologue B1(BRAF)fusion or rearrangement,or BRAV600E mutation.Tovorafenib is an oral,greater brain-penetrant,selective,type Ⅱ RAF inhibitor which has potent activity against both oncogenic BRAF fusions and BRAFV600E mutations.Most tumors have been showed some degree of shrinkage.The mechanism of action,pharmacodynamics,pharmacokinetics,clinical study and safety were introduced.

Tovorafenib has been approved by the U.S.Food and Drug Administration(FDA)for the treatment of patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma(LGGs)harboring the serine threonine kinae v-RAF murine sarcoma viral oncogene homologue B1(BRAF)fusion or rearrangement,or BRAV600E mutation.Tovorafenib is an oral,greater brain-penetrant,selective,type Ⅱ RAF inhibitor which has potent activity against both oncogenic BRAF fusions and BRAFV600E mutations.Most tumors have been showed some degree of shrinkage.The mechanism of action,pharmacodynamics,pharmacokinetics,clinical study and safety were introduced.

Objective:To investigate MRI texture features reflecting short-term medial femoral cartilage variations after anterior cruciate ligament (ACL) reconstruction.Methods:A retrospective case series analysis was conducted referring to the data of 20 patients who received ACL reconstruction at First Affiliated Hospital of Fujian Medical University from January 2017 to January 2020 including 17 males and 3 females, aged 23-43 years [(32.1±5.4)years]. The patients were evaluated before operation and at 1, 4, 12, 24, 48 weeks after operation: (1) the region of interest (ROI) in medial femoral cartilage was divided into ROI1 (meniscus anterior horn cartilage), ROI2 (meniscus body cartilage) and ROI3 (meniscus posterior horn cartilage) according to MRI image acquisition of the affected knee. The texture feature parameters were extracted for screening and analyzing to obtain the parameters reflecting sensitivity to cartilage variations; (2) the level of urinary C-telopeptide of type II collagen (uCTX-II) was measured by ELISA. At different time points, differences in texture feature parameters were compared to screen the parameters most sensitive to cartilage variations, and uCTX-II level were compared as well. Then, Pearson correlation analysis was used to determine the correlation between texture feature parameters most sensitive to cartilage variations and uCTX-II level.Results:After screening and analyzing, the horizontal direction of gray level non-uniformity (Horzl_GlevNonU) and Skewness of histogram (Skewness) of ROI3 were selected as sensitive texture feature parameters reflecting sensitivity to cartilage variations. There were significant difference in the Horzl_GlevNonU and Skewness of ROI3 before operation and at 1, 4, 12, 24, 48 weeks after operation (all P<0.05). Meanwhile, the Horzl_GlevNonU and Skewness of ROI3 were gradually increased over time, with a more significant tendency in the Horzl_GlevNonU. The Horzl_GlevNonU of ROI3 was similar at 4 weeks and 12 weeks after operation ( P>0.05), with significant differences at other time points (all P<0.05). The Skewness of ROI3 was significantly different at 1 week and 48 weeks after operation ( P<0.05), with insignificant differences at other time points (all P>0.05). The Horzl_GlevNonU of ROI3 was selected as the most sensitive texture feature parameter reflecting cartilage variations. Significant difference was reported in uCTX-II level before operation and at 1, 4, 12, 24, 48 weeks after operation (all P<0.05). After Pearson correlation analysis, the Horzl_GlevNonU of ROI3 was positively correlated with uCTX-II level before operation and at 1, 4, 12, 24, 48 weeks after operation ( r=0.554, 0.596, 0.550, 0.632, 0.756, 0.514, P<0.05 or 0.01). Conclusion:The Horzl_GlevNonU of ROI3 is the most sensitive texture feature parameter that reflects the early variations of medial femoral cartilage after ACL reconstruction and is positively correlated with uCTX-II level.

Objective: To evaluate the efficiency and safety of low intensity conditional regimen for children with Fanconi anemia (FA) receiving allogenic hematopoietic stem cells transplantation (allo-HSCT). Methods: Four patients diagnosed as Fanconi anemia were enrolled in this study. One patient received HLA-identical sibling donor hematopoietic stem cell transplantation, two patients underwent unrelated donor matched (UD) HSCT, and one patient received unrelated cord blood transplantation. The conditional regimen consisted of Busulfan with low dose of cyclophosphamide. Results: All 4 cases succeeded in allo-HSCT. The median time for neutrophils engraftment was 11(9-15) day, median time to platelets (PLT) engraftment was 12 (8-28) day. One case occurred with grade I of aGVHD, 1 case with hemorrhagic cystitis. No patient happened with hepatic veno-occlusive disease (VOD). Conclusion: Low intensity of conditional regimen is efficient and safe which should be recommended for FA patients with HSCT.
Busulfan ; Fanconi Anemia ; Graft vs Host Disease ; Hematopoietic Stem Cell Transplantation ; Humans ; Transplantation Conditioning

Objective To study the Mycoplasma pneumoniae infection in the clinical characteristics and treatment methods. Methods From June 2008 to December 2009,49 cases of Mycoplasma pneumoniae infection in patients with clinical data were retrospectively analyzed and summarized. Results 49 cases of children were diagnosed with acute upper respiratory tract infection in 13 cases(26.5% ) ,acute bronchitis 21 cases(42.9%) ,bronchial pneumonia, 15 cases( 30.6% ). Azithromycin alone treatment group,24 cases cured 22 cases ,2 cases improved, the cure rate of 91.7% ;erythromycin and azithromycin infusion + oral sequential treatment group,25 cases recovered,21cases improved,4 cases,the cure rate was 84%. Compared with the two groups,difference was not statistically significant(P ＞ 0.05). All patients were treated in our hospital with average of 13.8d,follow-up period without recurrence.Conclusion Mycoplasma pneumoniae infection in large differences in clinical manifestations, early diagnosis and treatment should be carried out. Azithromycin in the treatment effect was good, a short course of treatment could be used as treatment of Mycoplasma pneumoniae infection.