Jurnal Internasional Dilema pengobatan pada anak-anak asimptomatik dengan limfohistiositosis hemofagositik primer

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Jurnal Internasional Dilema pengobatan pada anak-anak asimptomatik dengan limfohistiositosis hemofagositik primer

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Asymptomatic carriers of pathogenic biological mutations in genes causing primary hemophagocytic lymphohistiocytosis (HLH) are at high risk of developing life-threatening HLH, which requires allogeneic hematopoietic stem (HSCT) transplants to be cured. There are no guidelines for managing this asymptomatic patient. We analyzed the results of the index case pairs (IC) and were later diagnosed as asymptomatic family members carrying the same genetic defect. We collect data from 22 HSCT centers throughout the world. Sixty-four children can be evaluated. IC is presented with HLH at an average age of 16 months. Seven of 32 ICs died during first-line therapy, and 2 were still alive after chemotherapy alone. Overall, 23/32 had HSCT, and 16 of them were still alive. At a median follow-up of 36 months from diagnosis, 18/32 ICs are alive. The average age of AC at diagnosis is 5 months. Ten of the 32 AC HLH activated transients were observed, and all undergoing HSCT: 6/10 were still alive and in complete remission (CR). 22/32 AC remained asymptomatic, and 6/22 did not receive treatment and was in CR at a 39-month follow-up average. Sixteen of 22 underwent preemptive HSCT: 15/16 were still alive and in CR. Eight years the probability of overall survival (pOS) in AC that did not activate HLH was significantly higher than in IC (95% vs 45%; P .02), and pOS in AC received HSCT before activation the disease was significantly higher than in AC receiving HSCT after activation of HLH (93% vs 64%; P [19459008=03)TheHSCTpreferenceinACprovedsafeandmustbeconsidered

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