本文選題：組織細(xì)胞增生癥 + 郎格漢斯細(xì)胞　； 參考：《中國循證兒科雜志》2017年02期

【摘要】：目的比較復(fù)旦大學(xué)附屬兒科醫(yī)院(我院)CHFU-LCH 2006方案(簡稱2006方案)和CHFU-LCH 2012方案(簡稱2012方案)治療郎格漢斯細(xì)胞組織細(xì)胞增生癥(LCH)患兒的療效和不良反應(yīng)。方法 2006年1月1日至2012年11月31日在我院接受2006方案治療的LCH初治患兒納入2006組,2012年12月1日至2015年12月31日在我院接受2012方案治療的LCH初治患兒納入2012組。兩組均經(jīng)病理確診LCH,排除治療6周內(nèi)自動(dòng)終止治療者。每組進(jìn)一步分為單系統(tǒng)LCH(SS-LCH)和多系統(tǒng)LCH(MS-LCH)亞組。所有患兒隨訪至2017年3月31日。治療有效為無活動(dòng)性病變或活動(dòng)性病變好轉(zhuǎn)。以Kaplan-Meier法計(jì)算5年預(yù)計(jì)總生存率(OS)和無病生存率(EFS)。不良反應(yīng)根據(jù)WHO急性和亞急性毒性反應(yīng)分級(jí)標(biāo)準(zhǔn)分為0~4級(jí)。比較兩組治療6和12周有效率,惡化、復(fù)發(fā)和死亡情況,5年預(yù)計(jì)OS、EFS和不良反應(yīng)發(fā)生情況。結(jié)果 96例患兒進(jìn)入2006組,男64例,女32例,中位年齡3.4歲,中位隨訪時(shí)間6.9年;86例患兒進(jìn)入2012組,男59例,女27例,中位年齡2.9歲,中位隨訪時(shí)間4.0年。兩組性別、診斷年齡、臨床分型和危險(xiǎn)器官受累(RO+)情況差異無統(tǒng)計(jì)學(xué)意義。(1)2006組和2012組比較,SS-LCH、MS-LCH亞組治療6、12周,有效率和復(fù)發(fā)率差異均無統(tǒng)計(jì)學(xué)意義。(2)2006組和2012組MS-LCH亞組分別有4例和5例退出方案,轉(zhuǎn)入其他挽救方案,分別有5例和4例死亡。(3)兩組MSLCH患兒共93例,其中2歲5年預(yù)計(jì)EFS和OS均明顯低于≥2歲患兒[EFS:(41.9±8.1)%vs(62.6±7.5)%,OS:(80.8±6.2)%vs(98.0±2.0)%],P均0.05;RO+患兒5年預(yù)計(jì)EFS和OS低于RO-患兒[EFS:(37.4±8.0)%vs(66.0±7.3)%,OS:(80.4±6.3)%vs(98.0±2.0)%],P均0.05;RO-患兒2歲和≥2歲5年預(yù)計(jì)EFS和OS差異無統(tǒng)計(jì)學(xué)意義;6周治療無效患兒5年預(yù)計(jì)EFS低于6周治療有效患兒[(33.1±7.9)%vs(70.8±7.2)%],P0.05。(4)2006組和2012組SS-LCH亞組5年預(yù)計(jì)EFS分別為(84.8±5.3)%和(86.7±5.6)%,5年預(yù)計(jì)OS均為100%;MS-LCH亞組5年預(yù)計(jì)EFS分別為(50.0±7.1)%和(53.2±10.0)%,5年預(yù)計(jì)OS分別為(90.0±4.1)%和(90.6±4.5)%;差異均無統(tǒng)計(jì)學(xué)意義。(5)2006組MS-LCH亞組化療相關(guān)3/4級(jí)不良反應(yīng)發(fā)生率(50.0%,25/50)高于2012組(23.3%,10/43),P=0.008 0。結(jié)論 CHFULCH 2012方案與2006方案療效未發(fā)現(xiàn)有差別,化療相關(guān)嚴(yán)重不良反應(yīng)較輕,MS-LCH的5年EFS仍不滿意。RO+和治療6周反應(yīng)情況是MS-LCH的重要預(yù)后影響因素。
[Abstract]:Objective to compare the efficacy and side effects of CHFU-LCH 2006 (2006) and CHFU-LCH 2012 (2012) in the treatment of Langerhans cell histiocytosis in children with Langerhans cell histiocytosis. Methods from January 1, 2006 to November 31, 2012, 2006 children with initial treatment received 2006 regimen in our hospital were enrolled in the study, and 2012 patients received 2012 regimen in our hospital from December 1, 2012 to December 31, 2015. LCHs were confirmed by pathology in both groups, excluding those who were automatically terminated within 6 weeks of treatment. Each group was further divided into single system LCHS-LCHS-LCH and multi-system LCHS-LCH subgroups. All children were followed up until March 31, 2017. The effective treatment was inactive lesions or improved active lesions. The overall and disease-free survival rates were calculated by Kaplan-Meier method. Adverse reactions were classified as 0 or 4 according to WHO classification criteria for acute and subacute toxic reactions. The efficacy, exacerbation, recurrence and death were compared between the two groups at 6 and 12 weeks. The incidence of EFS and adverse reactions were predicted for 5 years. Results 96 children were enrolled in group 2006 (64 males, 32 females, median age 3.4 years). The median follow-up time was 6.9 years. 86 patients (59 males and 27 females) were enrolled in the 2012 group. The median age was 2.9 years. The median follow-up time was 4.0 years. There was no significant difference in sex, diagnostic age, clinical type and risk organ involvement (RO) between the two groups. There was no significant difference between the two groups in the treatment of SS-LCHMS-LCH subgroup for 6 to 12 weeks. There was no significant difference in effective rate and recurrence rate. There were 4 cases in MS-LCH subgroup in 2006 and 5 cases in MS-LCH subgroup in 2012 group, and they were transferred to other rescue schemes, 5 cases and 4 cases died respectively. 93 cases of MSLCH children in two groups were treated with MS-LCH subgroup, and 93 cases with MS-LCH subgroup were treated with MS-LCH subgroup 2012. Both EFS and OS were significantly lower in 2 years old and 5 years old than those in children 鈮，

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