Survival outcomes of children with relapsed or refractory myeloid leukemia associated with Down syndrome

被引:8
|
作者
Raghuram, Nikhil [1 ]
Hasegawa, Daisuke [2 ]
Nakashima, Kentaro [3 ]
Rahman, Syaza [4 ]
Antoniou, Evangelia [5 ]
Skajaa, Torjus [6 ]
Merli, Pietro [7 ]
Verma, Anupam [8 ,9 ]
Rabin, Karen R. [10 ]
Aftandilian, Catherine [11 ]
Kotecha, Rishi S. [12 ,13 ,14 ]
Cheuk, Daniel [15 ,16 ]
Jahnukainen, Kirsi [17 ,18 ]
Kolenova, Alexandra [19 ]
Balwierz, Walentyna [20 ]
Norton, Alice [21 ]
O'Brien, Maureen [22 ]
Cellot, Sonia [23 ]
Chopek, Ashley [24 ]
Arad-Cohen, Nira [25 ]
Goemans, Bianca [26 ]
Rojas-Vasquez, Marta [27 ]
Ariffin, Hany [4 ]
Bartram, Jack [6 ]
Kolb, E. Anders [28 ]
Locatelli, Franco [7 ]
Klusmann, Jan-Henning [29 ]
Hasle, Henrik [30 ]
Mcguire, Bryan [1 ]
Hasnain, Afia [31 ]
Sung, Lillian [1 ]
Hitzler, Johann [1 ,32 ,33 ]
机构
[1] Univ Toronto, Hosp Sick Children, Div Hematol Oncol, Toronto, ON, Canada
[2] St Lukes Int Hosp, Dept Pediat, 9-1 Akashi Cho,Chuo Ku, Tokyo 1048560, Japan
[3] Kyushu Univ, Grad Sch Med Sci, Dept Pediat, Fukuoka, Japan
[4] Univ Malaya, Div Paediat Haematol Oncol & BM Transplantat, Med Ctr, Kuala Lumpur, Malaysia
[5] Univ Duisburg Essen, Univ Childrens Hosp Essen, Dept Pediat Hematol & Oncol, Essen, Germany
[6] Great Ormond St Hosp Sick Children, Dept Haematol, London, England
[7] Sapienza Univ Rome, Osped Pediatr Bambino Gesu, Ist Ricovero & Cura Carattere Sci IRCCS, Dept Pediat Hematol Oncol, Rome, Italy
[8] Univ Utah, Primary Childrens Hosp, Dept Pediat, Div Hematol Oncol, Salt Lake City, UT USA
[9] NCI, NIH, Ctr Canc Res, Pediat Oncol Branch, Bethesda, MD USA
[10] Baylor Coll Med, Texas Childrens Canc Ctr, Pediat Hematol Oncol, Houston, TX USA
[11] Stanford Univ, Dept Pediat, Div Hematol Oncol Stem Cell Transplantat & Regener, Sch Med, Stanford, CA USA
[12] Perth Childrens Hosp, Dept Clin Haematol Oncol Blood & Marrow Transplant, Perth, WA, Australia
[13] Univ WA, Telethon Kids Inst, Telethon Kids Canc Ctr, Leukaemia Translat Res Lab, Perth, WA, Australia
[14] Curtin Univ, Curtin Med Sch, Perth, WA, Australia
[15] Univ Hong Kong, Dept Paediat & Adolescent Med, Hong Kong, Peoples R China
[16] Hong Kong Childrens Hosp, Hong Kong, Peoples R China
[17] Univ Helsinki, Childrens Hosp, Helsinki, Finland
[18] Helsinki Univ Hosp, Helsinki, Finland
[19] Comenius Univ, Childrens Hosp Limbova 1, Bratislava, Slovakia
[20] Jagiellonian Univ, Med Coll, Inst Pediat, Dept Pediat Oncol & Hematol, Krakow, Poland
[21] Birmingham Womens & Childrens NHS Fdn Trust, Dept Haematol, Birmingham, England
[22] Univ Cincinnati, Coll Med, Dept Pediat, Cincinnati, OH USA
[23] Ste Justine Hosp, Dept Pediat, Div Hematol, Montreal, PQ, Canada
[24] Univ Manitoba, Pediat Blood & Marrow Transplant Program, Canc Care Manitoba, Winnipeg, MB, Canada
[25] Ruth Rappaport Childrens Hosp, Pediat Hematol Oncol Dept, Rambam Hlth Care Campus, Haifa, Israel
[26] Princess Maxima Ctr Pediat Oncol, Utrecht, Netherlands
[27] Univ Alberta, Dept Pediat Hematol Oncol, Stollery Childrens Hosp, Edmonton, AB, Canada
[28] Alfred I duPont Hosp Children, Nemours Ctr Canc & Blood Disorders, Wilmington, DE USA
[29] Goethe Univ Frankfurt, Frankfurt, Germany
[30] Aarhus Univ Hosp, Dept Paediat & Adolescent Med, Aarhus, Denmark
[31] Hosp Sick Children, Div Genome Diagnost, Dept Paediat Lab Med, Toronto, ON, Canada
[32] Hosp Sick Children, Res Inst, Program Dev & Stem Cell Biol, Toronto, ON, Canada
[33] Hosp Sick Children, Div Haematol Oncol, 555 Univ Ave, Toronto, ON M5G 1X8, Canada
关键词
ACUTE MEGAKARYOBLASTIC LEUKEMIA; TRANSIENT LEUKEMIA; MUTATIONS; TRIAL; GATA1; CHEMOTHERAPY; REDUCTION; THERAPY; RISK;
D O I
10.1182/bloodadvances.2022009381
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
Children with Down syndrome (DS) are at a significantly higher risk of developing acute myeloid leukemia, also termed myeloid leukemia associated with DS (ML-DS). In contrast to the highly favorable prognosis of primary ML-DS, the limited data that are available for children who relapse or who have refractory ML-DS (r/r ML-DS) suggest a dismal prognosis. There are few clinical trials and no standardized treatment approach for this population. We conducted a retrospective analysis of international study groups and pediatric oncology centers and identified 62 patients who received treatment with curative intent for r/r ML-DS between year 2000 to 2021. Median time from diagnosis to relapse was 6.8 (range, 1.1-45.5) months. Three-year event-free survival (EFS) and overall survival (OS) were 20.9 +/- 5.3% and 22.1 +/- 5.4%, respectively. Survival was associated with receipt of hematopoietic stem cell transplantation (HSCT) (hazard ratio [HR], 0.28), duration of first complete remission (CR1) (HR, 0.31 for > 12 months) and attainment of remission after relapse (HR, 4.03). Patients who achieved complete remission (CR) before HSCT, had an improved OS and EFS of 56.0 +/- 11.8% and 50.5 +/- 11.9%, respectively compared to those who underwent HSCT without CR (3-year OS and EFS of 10.0 +/- 9.5%). Treatment failure after HSCT was predominantly because of disease recurrence (52%) followed by treatment-related mortality (10%). The prognosis of r/r ML-DS remains dismal even in the current treatment period and serve as a reference point for current prognostication and future interventional studies. Clinical trials aimed at improving the survival of patients with r/r ML-DS are needed.
引用
收藏
页码:6532 / 6539
页数:8
相关论文
共 50 条
  • [21] Tisagenlecleucel in pediatric and young adult patients with Down syndrome-associated relapsed/refractory acute lymphoblastic leukemia
    Laetsch, Theodore W.
    Maude, Shannon L.
    Balduzzi, Adriana
    Rives, Susana
    Bittencourt, Henrique
    Boyer, Michael W.
    Buechner, Jochen
    De Moerloose, Barbara
    Qayed, Muna
    Phillips, Christine L.
    Pulsipher, Michael A.
    Hiramatsu, Hidefumi
    Tiwari, Ranjan
    Grupp, Stephan A.
    LEUKEMIA, 2022, 36 (06) : 1508 - 1515
  • [22] Myeloid Leukemia of Down Syndrome
    Kosmidou, Aikaterini
    Tragiannidis, Athanasios
    Gavriilaki, Eleni
    CANCERS, 2023, 15 (13)
  • [23] Treating acute myeloid leukemia among children with down syndrome
    Kapoor, Rajan
    Mohan, Karthik Ram
    Roy, Shuvendu
    Pramanik, Suman Kumar
    Khera, Sanjeev
    Simalti, A. K.
    INDIAN JOURNAL OF MEDICAL AND PAEDIATRIC ONCOLOGY, 2020, 41 (06) : 841 - 845
  • [24] Mechanisms of Progression of Myeloid Preleukemia to Transformed Myeloid Leukemia in Children with Down Syndrome
    Labuhn, Maurice
    Perkins, Kelly
    Matzk, Soeren
    Varghese, Leila
    Garnett, Catherine
    Papaemmanuil, Elli
    Metzner, Marlen
    Kennedy, Alison
    Amstislayskiy, Vyacheslav
    Risch, Thomas
    Bhayadia, Raj
    Samulowski, David
    Hernandez, David Cruz
    Stoilova, Bilyana
    Lotchkova, Valentina
    Oppermann, Udo
    Scheer, Carina
    Yoshida, Kenichi
    Schwarzer, Adrian
    Taub, Jeffrey
    Crispino, John D.
    Weiss, Mitchell J.
    Hayashi, Asuhide
    Taga, Takashi
    Ito, Etsuro
    Ogawa, Seishi
    Reinhardt, Dirk
    Yaspo, Marie-Laure
    Campbell, Peter J.
    Roberts, Irene
    Constantinescu, Stefan
    Vyas, Paresh
    Heckl, Dirk
    Klusmann, Jan-Henning
    CANCER CELL, 2019, 36 (02) : 123 - +
  • [25] Real-world clinical outcomes with enasidenib in relapsed or refractory acute myeloid leukemia
    Klink, Andrew J.
    Gajra, Ajeet
    Knoth, Russell L.
    Marshall, Landon
    Hou, Ying
    McBride, Ali
    Copher, Ronda
    LEUKEMIA RESEARCH, 2022, 122
  • [26] Salvage Therapy Outcomes in a Historical Cohort of Patients with Relapsed or Refractory Acute Myeloid Leukemia
    Ravandi, Farhad
    Pierce, Sherry A.
    Garcia-Manero, Guillermo
    Kadia, Tapan M.
    Jabbour, Elias J.
    Borthakur, Gautam
    DiNardo, Courtney D.
    Daver, Naval G.
    Short, Nicholas J.
    Alvarado, Yesid
    Cortes, Jorge E.
    Kim, Christopher
    Kelsh, Michael A.
    Katz, Aaron Jacob
    Williams, Richard
    Yang, Zhao
    Mehta, Bhakti
    Kantarjian, Hagop M.
    BLOOD, 2018, 132
  • [27] Salvage Therapy Outcomes in a Historical Cohort of Patients With Relapsed or Refractory Acute Myeloid Leukemia
    Ravandi, Farhad
    Pierce, Sherry
    Garcia-Manero, Guillermo
    Kadia, Tapan
    Jabbour, Elias
    Borthakur, Gautam
    DiNardo, Courtney D.
    Daver, Naval
    Short, Nicholas J.
    Alvarado, Yesid
    Cortes, Jorge
    Kim, Christopher
    Kelsh, Michael
    Katz, Aaron
    Williams, Richard
    Yang, Zhao
    Mehta, Bhakti
    Kantarjian, Hagop
    CLINICAL LYMPHOMA MYELOMA & LEUKEMIA, 2020, 20 (11): : E871 - E882
  • [28] Outcomes of Patients with Relapsed or Refractory Acute Myeloid Leukemia Receiving Hypomethylating Agent and Venetoclax
    Asghari, Hannah
    Lee, Dasom
    Deutsch, Yehuda E.
    Chan, Onyee
    Al Ali, Najla
    Boisclair, Stephanie
    Brahim, Amanda
    Padron, Eric
    Kuykendall, Andrew T.
    List, Alan F.
    Fernandez, Hugo F.
    Lancet, Jeffrey E.
    Sallman, David A.
    Komrokji, Rami S.
    Sweet, Kendra L.
    Talati, Chetasi
    BLOOD, 2019, 134
  • [29] Need for new thinking: Treatment of relapsed leukemia in children with Down syndrome
    Rabin, Karen
    Izraeli, Shai
    Hijiya, Nobuko
    Hitzler, Johann
    PEDIATRIC BLOOD & CANCER, 2019, 66 (06)
  • [30] A Refractory Case of Myeloid Leukemia of Down Syndrome Showing Atypical Clinical Course
    Takasugi, Nao
    Kato, Shota
    Hougetsu, Keita
    Hidaka, Moe
    Sekiguchi, Masahiro
    Kubota, Yasuo
    Watanabe, Kentarou
    Oka, Akira
    Takita, Junko
    Hiwatari, Mitsuteru
    PEDIATRIC BLOOD & CANCER, 2019, 66 : S18 - S18