[The application of extracorporeal membrane oxygenation in children in China].

Objective: To investigate the application of extracorporeal membrane oxygenation (ECMO) in pediatrics in China as well as the outcomes. Methods: Data was conducted by questionnaire to investigate the use of ECMO in children under the age of 18 in China by June 30, 2017. All patients were divided into two age groups: pediatric patients (29 d-18 y) and neonates (1-28 d); Also by the causes of ECMO treating including cardiac, respiratory and extracorporeal cardiopulmonary resuscitation (ECPR). The form included the numbers of ECMO cases, weaned and discharged cases, according to the different ages and causes. In addition, the departments that routinely participate in ECMO management were acquired. Results: Totally 43 tertiary hospitals were enrolled, of which 30 have implemented ECMO for the children patients (comprising pediatrics and neonates), including 14 general hospitals, 5 cardiothoracic specialty hospitals and 11 children's or women and children's hospitals. ECMO for pediatrics and neonates was firstly carried out at mainland China in 2004. To the deadline of investigation, 800 patients were supported with ECMO, among which 658 were pediatrics, much more than 142 of neonates. As to pediatrics, 453 were supported with ECMO for cardiac indications with 287 (63.4%) weaned off and 215 (47.5%) survived to discharge; for respiratory causes, 79 cases were registered with 47 (59.5%) weaned off and 36 (45.6%) discharged; for ECPR, 126 were enrolled with 62 (49.2%) successfully weaned off and 48 (38.1%) discharged. In contrast, neonatal patients undergoing cardiac ECMO contained 79 cases, with 39 (49.4%) weaned off and 26 (32.9%) discharged; due to respiratory causes, 40 neonates were included, with 26 (65.0%) weaned off and 21 (52.5%) discharged; 23 neonatal patients consisted of ECPR cause and 10 (43.5%) of them successfully weaned off, but only 6 (26.1%) finally survived. Among the 30 hospitals conducted ECMO for pediatrics and neonates, the average number of departments for ECMO management is 4.03±1.87. Conclusions: Although ECMO used for children in mainland China is relatively late, a certain number of cases have been accumulated, and there is still a gap compared with the international standard. Meanwhile, each hospital has preliminarily built up its own ECMO team.

[Targeted sequencing analysis of hyper-eosinophilic syndrome and chronic eosinophilic leukemia].

Objective: Analysis of the molecular characteristics of eosinophilia. Methods: Targeting sequence to 24 patients with chronic eosinophilic leukemia (CEL) with rearrangement of PDGFRA, PDGFRB, or FGFR1 and 62 patients with hyper-eosinophilic syndrome (HES). Mutation annotation and analysis of amino acid mutation using authoritative databases to speculate on possible pathogenic mutation. Results: Thirty-seven kinds of clonal variant were detected from 17 patients with CEL, no recurrent mutation site and hot spot region were found. No pathogenic mutation was detected in 19 patients with PDGFRA rearrangement, but pathogenic mutations of ASXL1, RUNX1 and NRAS were detected from 2 patients with FGFR1 rearrangement who progressed to acute myeloid leukemia and 1 patient with PDGFRB rearrangement who progressed to T lymphoblastic lymphoma, respectively. One hundred and two kinds of clonal abnormalities were detected in 49 patients with HES. The main hot spot mutation regions included: CEBPA Exon1, TET2 Exon3, ASXL1 Exon12, IDH1 Y208C, and FGFR3 L164V. CRRLF2 P224L and PDGFRB R370C point mutations were detected separately in 2 patients with HES who treated with imatinib monotherapy and achieved hematologic remission. Conclusion: The pathogenesis of CEL with PDGFRA, PDGFRB or FGFR1 rearrangement is usually single, and the progression of the disease may involve other driver mutation. A variety of genes with hot mutation regions may be involved in the pathogenesis of HES, and some mutation sites are sensitive to tyrosine kinase inhibitors.

[Clinical characteristics and long- term therapeutic effects of 60 patients with idiopathic hypereosinophilic syndrome in a single center].

Objective: To analyze the long term outcome of patients with hypereosinophilic syndrome(HES). Methods: The clinical characteristics and efficacy of 60 newly diagnosed HES patients who received corticosteroids(CS)monotherapy were retrospectively analyzed. The survival and death causes of patients were obtained by follow- up. Results: Of all 60 HES patients, 45 were male and 15 female. The median age was 38(11-80)years old. The most frequent organ involvement of HES occurred in cutaneous(55.0%), gastrointestinal(40.0%), pulmonary(35.0%), cardiac(13.3%), vascular(10.0%)and neuromuscular system(10.0%). Single organ involvement was observed in 45.0% of the patients, two or at least three organ involvements were observed in 36.7% and 18.3%, respectively. The median daily dose of prednisone equivalent was 30(15-60)mg. The total response rate(CR plus PR)was 88.3%, and the rate was elevated to 93.3% after receiving alterative or combined treatment regimens. Thirty- eight patients with response to treatment received corticosteroid(CS)as mono(33 cases)or combined(5 cases)maintenance treatment with a median duration of 51(5-92)months; the median maintenance daily dose of prednisone equivalent was 5(1.25-40)mg. Twenty patients experienced cessation of CS. The main causes of patients' withdrawal were poor compliance after CR or ineffective treatment. The 5-year overall survival was(90.0±4.3)%, and the main cause of mortality was cardiac dysfunction. Conclusion: CS was highly effective on HES with manageable side effects. Most patients who have not obtained satisfactory effect could improve response via combination therapy. Cardiac dysfunction was the most common cause of mortality.

[Prognostic value of JAK2, MPL and CALR mutations in Chinese patients with primary myelofibrosis].

OBJECTIVE: To evaluate the prognostic value of JAK2, MPL and CALR mutations in Chinese patients with primary myelofibrosis (PMF). METHODS: Four hundred and two Chinese patients with PMF were retrospectively analyzed. The Kaplan-Meier method, the Log-rank test, the likelihood ratio test and the Cox proportional hazards regression model were used to evaluate the prognostic scoring system. RESULTS: This cohort of patients included 209 males and 193 females with a median age of 55 years (range: 15- 89). JAK2V617F mutations were detected in 189 subjects (47.0% ), MPLW515 mutations in 13 (3.2%) and CALR mutations in 81 (20.1%) [There were 30 (37.0%) type-1, 48 (59.3%) type-2 and 3 (3.7%) less common CALR mutations], respectively. 119 subjects (29.6%) had no detectable mutation in JAK2, MPL or CALR. Univariate analysis indicated that patients with CALR type-2 mutations or no detectable mutations had inferior survival compared to those with JAK2, MPL or CALR type- 1 or other less common CALR mutations (the median survival was 74vs 168 months, respectively [HR 2.990 (95% CI 1.935-4.619),P<0.001]. Therefore, patients were categorized into the high-risk with CALR type- 2 mutations or no detectable driver mutations and the low- risk without aforementioned mutations status. The DIPSS-Chinese molecular prognostic model was proposed by adopting mutation categories and DIPSS-Chinese risk group. The median survival of patients classified in low risk (132 subjects, 32.8% ), intermediate- 1 risk (143 subjects, 35.6%), intermediate- 2 risk (106 subjects, 26.4%) and high risk (21 subjects, 5.2%) were not reached, 156 (95% CI 117- 194), 60 (95% CI 28- 91) and 22 (95% CI 10- 33) months, respectively, and there was a statistically significant difference in overall survival among the four risk groups (P<0.001). There was significantly higher predictive power for survival according to the DIPSS-Chinese molecular prognostic model compared with the DIPSS-Chinese model (P=0.005, -2 log-likelihood ratios of 855.6 and 869.7, respectively). CONCLUSION: The impact of the CALR type- 2 mutations or no detectable driver mutation on survival was independent of current prognostic scoring systems. The DIPSS- Chinese molecular prognostic model based on the molecular features of Chinese patients was proposed and worked well for prognostic indication.