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Abstract：
BACKGROUND: In pediatric multi-system high risk organs (RO +) Langerhans cell histiocytosis (LCH), failing 1st line treatment has the highest mortality. We aim to present the outcome of failure of 1st line whether due to disease progression (DP) at end of induction or reactivation (REA) after initial better status response.
METHODS: Sixty-seven RO + LCH patients with hemopoietic, hepatic or splenic involvement, treated between 2007 and 2019 were retrospectively analyzed. The median follow-up (IQR) is 6 years (4-8.8 y).They were subjected to 2 eras of treatment; one with salvage by 2-Cda based regimen (2-CdABR) and another without.
RESULTS: Of 67 patients, M/F 40/27, median age 1.74 y (0.2-10 y), 42 failed 1st line (62.7%). Of them DP n = 22 (52%) and REA n = 20 (48%). Of those with DP, 9/22 patients received 2-CdABR, where 5 survived in better status. While the remaining 13 did not receive 2-CdABR and all of them died. Otherwise, of those with REA, 12/20 reactivated on RO + mode. Of them, 8/12 received 2-CdABR, where only one survived in better status and the remaining 4 received vinblastine-based regimen,where 2 died and 2 were rescued. RO + 5-year overall survival (OS) was 65% (CI 95% 54 -78) while the event free survival (EFS) 36% (26.3-50.1). The OS of DP 27% (14-54) versus REA 67% (49-93) p 0.004. OS of DP with 2-CdABR 56% (31-97.7) versus 8% without (2-51), p < 0.001. While OS of REA with 2-CdABR 38% (13-100) versus 74% without (53-100) p 0.7.
CONCLUSIONS: Survival of RO + remains limited. Failure of 1st line in RO + due to DP carries worse prognosis in relation to REA. In DP those who were not salvaged by 2-CdABR, showed dismal outcome. This could not be shown when applied in REA.
METHODS: Sixty-seven RO + LCH patients with hemopoietic, hepatic or splenic involvement, treated between 2007 and 2019 were retrospectively analyzed. The median follow-up (IQR) is 6 years (4-8.8 y).They were subjected to 2 eras of treatment; one with salvage by 2-Cda based regimen (2-CdABR) and another without.
RESULTS: Of 67 patients, M/F 40/27, median age 1.74 y (0.2-10 y), 42 failed 1st line (62.7%). Of them DP n = 22 (52%) and REA n = 20 (48%). Of those with DP, 9/22 patients received 2-CdABR, where 5 survived in better status. While the remaining 13 did not receive 2-CdABR and all of them died. Otherwise, of those with REA, 12/20 reactivated on RO + mode. Of them, 8/12 received 2-CdABR, where only one survived in better status and the remaining 4 received vinblastine-based regimen,where 2 died and 2 were rescued. RO + 5-year overall survival (OS) was 65% (CI 95% 54 -78) while the event free survival (EFS) 36% (26.3-50.1). The OS of DP 27% (14-54) versus REA 67% (49-93) p 0.004. OS of DP with 2-CdABR 56% (31-97.7) versus 8% without (2-51), p < 0.001. While OS of REA with 2-CdABR 38% (13-100) versus 74% without (53-100) p 0.7.
CONCLUSIONS: Survival of RO + remains limited. Failure of 1st line in RO + due to DP carries worse prognosis in relation to REA. In DP those who were not salvaged by 2-CdABR, showed dismal outcome. This could not be shown when applied in REA.
摘要：
背景：在小儿多系统高风险器官（RO）朗格汉斯细胞组织细胞增生症（LCH）中，失败的一线治疗死亡率最高。我们的目标是在最初的更好状态反应后，无论是由于诱导结束时的疾病进展（DP）还是再激活（REA），都是一线失败的结果。
方法：67例RO+LCH患者，肝或脾受累，对2007年至2019年期间接受治疗的患者进行回顾性分析。中位随访时间（IQR）为6年（4-8.8年）。他们接受了2次治疗；一种通过基于2-Cda的方案（2-CdABR）进行抢救，另一种没有。
结果：在67例患者中，M/F40/27，中位年龄1.74y（0.2-10y），42条第一线失败（62.7%）。其中DPn=22(52%)，REAn=20(48%)。在那些有DP的人中，9/22患者接受2-CdABR，5以更好的状态存活。而其余13人没有接受2-CdABR，他们都死了。否则,那些有REA的人，12/20在RO+模式下重新激活。其中,8/12收到2-CdABR，其中只有一个以更好的状态存活，其余4人接受了基于长春碱的方案，其中2人死亡，2人获救。RO+5年总生存率（OS）为65%（CI95%54-78），无事件生存率（EFS）为36%（26.3-50.1）。DP27%(14-54)的OS对REA67%(49-93)的OSp0.004。具有2-CdABR的DP的OS为56％（31-97.7），与没有（2-51）的8％相比，p<0.001。而含2-CdABR的REA的OS为38%(13-100)，而不含(53-100)的为74%，p为0.7。
结论：RO+的存活率仍然有限。由于DP导致的RO一线失败与REA有关，预后较差。在DP中，那些没有被2-CdABR挽救的人，显示出令人沮丧的结果。这在REA中应用时无法显示。
方法：67例RO+LCH患者，肝或脾受累，对2007年至2019年期间接受治疗的患者进行回顾性分析。中位随访时间（IQR）为6年（4-8.8年）。他们接受了2次治疗；一种通过基于2-Cda的方案（2-CdABR）进行抢救，另一种没有。
结果：在67例患者中，M/F40/27，中位年龄1.74y（0.2-10y），42条第一线失败（62.7%）。其中DPn=22(52%)，REAn=20(48%)。在那些有DP的人中，9/22患者接受2-CdABR，5以更好的状态存活。而其余13人没有接受2-CdABR，他们都死了。否则,那些有REA的人，12/20在RO+模式下重新激活。其中,8/12收到2-CdABR，其中只有一个以更好的状态存活，其余4人接受了基于长春碱的方案，其中2人死亡，2人获救。RO+5年总生存率（OS）为65%（CI95%54-78），无事件生存率（EFS）为36%（26.3-50.1）。DP27%(14-54)的OS对REA67%(49-93)的OSp0.004。具有2-CdABR的DP的OS为56％（31-97.7），与没有（2-51）的8％相比，p<0.001。而含2-CdABR的REA的OS为38%(13-100)，而不含(53-100)的为74%，p为0.7。
结论：RO+的存活率仍然有限。由于DP导致的RO一线失败与REA有关，预后较差。在DP中，那些没有被2-CdABR挽救的人，显示出令人沮丧的结果。这在REA中应用时无法显示。
