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Treatment dilemmas in asymptomatic children with primary hemophagocytic lymphohistiocytosis

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Harvard

Lucchini, G, Marsh, R, Gilmour, K, Worth, A, Nademi, Z, Rao, A, Booth, C, Amrolia, P, Silva, J, Chiesa, R, Wynn, R, Lehmberg, K, Astigarraga, I, Güngör, T, Stary, J, Moshous, D, Ifversen, M, Zinn, D, Jordan, M, Kumar, A, Yasumi, T, Veys, P & Rao, K 2018, 'Treatment dilemmas in asymptomatic children with primary hemophagocytic lymphohistiocytosis' Blood, vol. 132, no. 19, pp. 2088-2096. https://doi.org/10.1182/blood-2018-01-827485

APA

Lucchini, G., Marsh, R., Gilmour, K., Worth, A., Nademi, Z., Rao, A., ... Rao, K. (2018). Treatment dilemmas in asymptomatic children with primary hemophagocytic lymphohistiocytosis. Blood, 132(19), 2088-2096. https://doi.org/10.1182/blood-2018-01-827485

CBE

Lucchini G, Marsh R, Gilmour K, Worth A, Nademi Z, Rao A, Booth C, Amrolia P, Silva J, Chiesa R, Wynn R, Lehmberg K, Astigarraga I, Güngör T, Stary J, Moshous D, Ifversen M, Zinn D, Jordan M, Kumar A, Yasumi T, Veys P, Rao K. 2018. Treatment dilemmas in asymptomatic children with primary hemophagocytic lymphohistiocytosis. Blood. 132(19):2088-2096. https://doi.org/10.1182/blood-2018-01-827485

MLA

Vancouver

Lucchini G, Marsh R, Gilmour K, Worth A, Nademi Z, Rao A et al. Treatment dilemmas in asymptomatic children with primary hemophagocytic lymphohistiocytosis. Blood. 2018 Nov 8;132(19):2088-2096. https://doi.org/10.1182/blood-2018-01-827485

Author

Lucchini, Giovanna ; Marsh, Rebecca ; Gilmour, Kimberly ; Worth, Austen ; Nademi, Zohreh ; Rao, Anupama ; Booth, Claire ; Amrolia, Persis ; Silva, Juliana ; Chiesa, Robert ; Wynn, Robert ; Lehmberg, Kai ; Astigarraga, Itziar ; Güngör, Tayfun ; Stary, Jan ; Moshous, Despina ; Ifversen, Marianne ; Zinn, Daniel ; Jordan, Michael ; Kumar, Ashish ; Yasumi, Takahiro ; Veys, Paul ; Rao, Kanchan. / Treatment dilemmas in asymptomatic children with primary hemophagocytic lymphohistiocytosis. In: Blood. 2018 ; Vol. 132, No. 19. pp. 2088-2096.

Bibtex

@article{517e1be7bef64a93bf20066f734a973a,
title = "Treatment dilemmas in asymptomatic children with primary hemophagocytic lymphohistiocytosis",
abstract = "Asymptomatic carriers (ACs) of pathogenic biallelic mutations in causative genes for primary hemophagocytic lymphohistiocytosis (HLH) are at high risk of developing life-threatening HLH, which requires allogeneic hematopoietic stem cell transplantation (HSCT) to be cured. There are no guidelines on the management of these asymptomatic patients. We analyzed the outcomes of pairs of index cases (ICs) and subsequently diagnosed asymptomatic family members carrying the same genetic defect. We collected data from 22 HSCT centers worldwide. Sixty-four children were evaluable. ICs presented with HLH at a median age of 16 months. Seven of 32 ICs died during first-line therapy, and 2 are alive after chemotherapy only. In all, 23/32 underwent HSCT, and 16 of them are alive. At a median follow-up of 36 months from diagnosis, 18/32 ICs are alive. Median age of ACs at diagnosis was 5 months. Ten of 32 ACs activated HLH while being observed, and all underwent HSCT: 6/10 are alive and in complete remission (CR). 22/32 ACs remained asymptomatic, and 6/22 have received no treatment and are in CR at a median follow-up of 39 months. Sixteen of 22 underwent preemptive HSCT: 15/16 are alive and in CR. Eight-year probability of overall survival (pOS) in ACs who did not have activated HLH was significantly higher than that in ICs (95{\%} vs 45{\%}; P = .02), and pOS in ACs receiving HSCT before disease activation was significantly higher than in ACs receiving HSCT after HLH activation (93{\%} vs 64{\%}; P = .03). Preemptive HSCT in ACs proved to be safe and should be considered.",
author = "Giovanna Lucchini and Rebecca Marsh and Kimberly Gilmour and Austen Worth and Zohreh Nademi and Anupama Rao and Claire Booth and Persis Amrolia and Juliana Silva and Robert Chiesa and Robert Wynn and Kai Lehmberg and Itziar Astigarraga and Tayfun G{\"u}ng{\"o}r and Jan Stary and Despina Moshous and Marianne Ifversen and Daniel Zinn and Michael Jordan and Ashish Kumar and Takahiro Yasumi and Paul Veys and Kanchan Rao",
note = "{\circledC} 2018 by The American Society of Hematology.",
year = "2018",
month = "11",
day = "8",
doi = "10.1182/blood-2018-01-827485",
language = "English",
volume = "132",
pages = "2088--2096",
journal = "Blood",
issn = "0006-4971",
publisher = "American Society of Hematology",
number = "19",

}

RIS

TY - JOUR

T1 - Treatment dilemmas in asymptomatic children with primary hemophagocytic lymphohistiocytosis

AU - Lucchini, Giovanna

AU - Marsh, Rebecca

AU - Gilmour, Kimberly

AU - Worth, Austen

AU - Nademi, Zohreh

AU - Rao, Anupama

AU - Booth, Claire

AU - Amrolia, Persis

AU - Silva, Juliana

AU - Chiesa, Robert

AU - Wynn, Robert

AU - Lehmberg, Kai

AU - Astigarraga, Itziar

AU - Güngör, Tayfun

AU - Stary, Jan

AU - Moshous, Despina

AU - Ifversen, Marianne

AU - Zinn, Daniel

AU - Jordan, Michael

AU - Kumar, Ashish

AU - Yasumi, Takahiro

AU - Veys, Paul

AU - Rao, Kanchan

N1 - © 2018 by The American Society of Hematology.

PY - 2018/11/8

Y1 - 2018/11/8

N2 - Asymptomatic carriers (ACs) of pathogenic biallelic mutations in causative genes for primary hemophagocytic lymphohistiocytosis (HLH) are at high risk of developing life-threatening HLH, which requires allogeneic hematopoietic stem cell transplantation (HSCT) to be cured. There are no guidelines on the management of these asymptomatic patients. We analyzed the outcomes of pairs of index cases (ICs) and subsequently diagnosed asymptomatic family members carrying the same genetic defect. We collected data from 22 HSCT centers worldwide. Sixty-four children were evaluable. ICs presented with HLH at a median age of 16 months. Seven of 32 ICs died during first-line therapy, and 2 are alive after chemotherapy only. In all, 23/32 underwent HSCT, and 16 of them are alive. At a median follow-up of 36 months from diagnosis, 18/32 ICs are alive. Median age of ACs at diagnosis was 5 months. Ten of 32 ACs activated HLH while being observed, and all underwent HSCT: 6/10 are alive and in complete remission (CR). 22/32 ACs remained asymptomatic, and 6/22 have received no treatment and are in CR at a median follow-up of 39 months. Sixteen of 22 underwent preemptive HSCT: 15/16 are alive and in CR. Eight-year probability of overall survival (pOS) in ACs who did not have activated HLH was significantly higher than that in ICs (95% vs 45%; P = .02), and pOS in ACs receiving HSCT before disease activation was significantly higher than in ACs receiving HSCT after HLH activation (93% vs 64%; P = .03). Preemptive HSCT in ACs proved to be safe and should be considered.

AB - Asymptomatic carriers (ACs) of pathogenic biallelic mutations in causative genes for primary hemophagocytic lymphohistiocytosis (HLH) are at high risk of developing life-threatening HLH, which requires allogeneic hematopoietic stem cell transplantation (HSCT) to be cured. There are no guidelines on the management of these asymptomatic patients. We analyzed the outcomes of pairs of index cases (ICs) and subsequently diagnosed asymptomatic family members carrying the same genetic defect. We collected data from 22 HSCT centers worldwide. Sixty-four children were evaluable. ICs presented with HLH at a median age of 16 months. Seven of 32 ICs died during first-line therapy, and 2 are alive after chemotherapy only. In all, 23/32 underwent HSCT, and 16 of them are alive. At a median follow-up of 36 months from diagnosis, 18/32 ICs are alive. Median age of ACs at diagnosis was 5 months. Ten of 32 ACs activated HLH while being observed, and all underwent HSCT: 6/10 are alive and in complete remission (CR). 22/32 ACs remained asymptomatic, and 6/22 have received no treatment and are in CR at a median follow-up of 39 months. Sixteen of 22 underwent preemptive HSCT: 15/16 are alive and in CR. Eight-year probability of overall survival (pOS) in ACs who did not have activated HLH was significantly higher than that in ICs (95% vs 45%; P = .02), and pOS in ACs receiving HSCT before disease activation was significantly higher than in ACs receiving HSCT after HLH activation (93% vs 64%; P = .03). Preemptive HSCT in ACs proved to be safe and should be considered.

U2 - 10.1182/blood-2018-01-827485

DO - 10.1182/blood-2018-01-827485

M3 - Journal article

VL - 132

SP - 2088

EP - 2096

JO - Blood

JF - Blood

SN - 0006-4971

IS - 19

ER -

ID: 56426839