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Objective:To preliminarily assess the clinical value of sirolimus combined with prednisone in the treatment of Kaposiform hemangioendothelioma (KHE) complicated by Kasabach-Merritt phenomenon (KMP) .Methods:A retrospective study was conducted. General clinical data and relevant data on efficacy and adverse reactions were collected from 36 patients with KHE complicated by KMP, who received oral sirolimus combined with prednisone in Department of Pediatric Surgery, West China Hospital, Sichuan University from January 2011 to January 2018.Results:Among the 36 patients with KHE complicated by KMP, the male-to-female ratio was 1∶0.8; their average age was 15.0 months (range, 4.0 - 60.0 months) , and the average age of onset was 6.3 months (range, 0.8 - 48.0 months) ; 32 (88.9%) patients were diagnosed with mixed KHE, and 4 (11.1%) with deep KHE; the tumor size ranged from 2.5 cm × 4.0 cm to 20.0 cm × 24.0 cm. During the combined treatment, the average duration of prednisone administration was 6.4 weeks (range, 5.0 - 9.0 weeks) , and that of sirolimus administration was 19.3 months (range, 13.0 - 27.0 months) . After 1-5 weeks of combined treatment, platelet counts and fibrinogen levels of the 36 patients gradually returned to normal. After short-term prednisone combined with long-term sirolimus treatment, the average disease severity score decreased from 4.0 before treatment to 2.4 at 6 months and 1.6 at 12 months. After 12 months of the combined treatment, tumors mostly regressed in 32 (88.9%) patients, partially regressed in 3 (8.3%) , showed no obvious change in 1 (2.8%) . During the treatment, common adverse reactions included gastrointestinal discomfort and oral ulcers, no patient discontinued the treatment due to severe adverse reactions, and no drug toxicity-related death occurred.Conclusion:Sirolimus combined with prednisone is effective and safe for the treatment of KHE complicated by KMP.
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Objective:To investigate differentially expressed long noncoding RNAs (lncRNAs) and mRNAs between proliferating and involuting infantile hemangioma (IH) .Methods:Eight IH specimens were surgically resected from 4 children with proliferating IH and 4 with involuting IH in Department of Pediatric Surgery, West China Hospital, Sichuan University from January to March in 2019. Differentially expressed lncRNAs and mRNAs between proliferating and involuting IH (a P value < 0.05 and a fold change ≥ 2.0) were screened by microarray analysis, and verified by real-time fluorescence-based quantitative PCR (qRT-PCR) . Bioinformatics analysis was further performed, including GO and KEGG pathway analyses of differentially expressed mRNAs, construction of a lncRNA-mRNA co-expression network, and prediction of cis-acting target genes of differentially expressed lncRNAs. Results:A total of 405 differentially expressed lncRNAs and 772 differentially expressed mRNAs were identified between the proliferating and involuting IH specimens by using microarray technology. Of them, 108 lncRNAs and 107 mRNAs were downregulated, 297 lncRNAs and 665 mRNAs were upregulated in the proliferating IH specimens. Four lncRNAs (n335248, ENST00000450864, n333319, and n335185) and 4 mRNAs (EDNRA, IFI6, HK2, and ITGA1) were verified by qRT-PCR, and the results were consistent with those of microarray analysis. GO and KEGG enrichment analyses showed that differentially expressed mRNAs were mainly involved in the biological processes blood coagulation, axon guidance, angiogenesis and cell adhesion. Besides, differentially expressed mRNAs were mostly enriched in the metabolic pathways focal cell adhesion, regulation of actin cytoskeleton, leukocyte transendothelial migration and phosphatidylinositol 3-kinase/serine-threonine protein kinase and other signaling pathways. In addition, a lncRNA-mRNA co-expression network was constructed with 23 mRNAs and 58 lncRNAs with the degree ≥ 15.Conclusion:Lots of differentially expressed lncRNAs and mRNAs were identified between proliferating and involuting IH tissues, and these lncRNAs may play important roles in the development of IH by regulating corresponding target mRNAs.
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Objective@#To investigate the clinical symptoms, treatment intervention and prognosis of Kaposiform lymphangiomatosis(KLA).@*Methods@#Medical information and clinical characteristics data of 8 KLA patients who were admitted to Department of Pediatric Surgery of West China Hospital of Sichuan University from January 2016 to February 2019 were retrospectively reviewed and analyzed. There were 5 males and 3 females with age of 5.8 years old (from 8 months to 29 years old).@*Results@#The lesions in all patients were diffusely distributed. In all 8 patients, the lung and mediastinum were involved with different degrees. Three cases had lesions involving pelvic and abdominal organs. Three cases had lesions involving bones. One case simultaneously involved pelvic and abdominal organs, and 1 case was involved laryngeal and neck. The clinical characteristics were mainly respiratory symptoms. In the laboratory tests, 6 patients had different degrees of thrombocytopenia (minimum 3 × 109/L), and 4 patients had severe fibrinogen reduction (minimum 0.42 g/L). Three patients had prolonged activated partial thromboplastin time (up to 64.2 seconds) and 3 patients had prolonged prothrombin time (up to 18.6 seconds). After surgery (including thoracotomy, chest tube, pericardiocentesis, splenectomy) and empiric medicine therapy (vincristine, sirolimus and corticosteroid), the symptoms improved in 1 case, 2 cases died of complications, 2 cases were stable and 3 cases progressed up to February 2019.@*Conclusions@#KLA is a rare disease that should be differentiated from other types of vascular diseases. Currently, there is no consensus treatment guidelines exist. Accurate diagnosis in KLA can be a challenge. The situation in patients with KLA is prone to rapid deterioration and progress. Future research efforts should seek to develop target-specific drugs for KLA.