Outcomes of hematopoietic stem cell transplantation in 813 pediatric patients with Fanconi anemia

被引:4
作者
Lum, Su Han [1 ,2 ]
Eikema, Dirk-Jan [3 ]
Piepenbroek, Brian [4 ]
Wynn, Robert F. [5 ]
Samarasinghe, Sujith [6 ]
Dalissier, Arnaud [7 ]
Kalwak, Krysztof [8 ]
Ayas, Mouhab [9 ]
Hamladji, Rose-Marie [10 ]
Yesilipek, Akif [11 ]
Dalle, Jean-Hugues [12 ]
Uckan-Cetinkaya, Duygu [13 ]
Bierings, Marc [14 ]
Kupesiz, Alphan [15 ]
Halahleh, Khalid [16 ]
Skorobogatova, Elena [17 ]
Ozturk, Gulyuz [18 ]
Faraci, Maura [19 ]
Renard, Cecile [20 ]
Evans, Pamela [21 ]
Corbacioglu, Selim [22 ]
Locatelli, Franco [23 ]
Dufour, Carlo [19 ]
Risitano, Antonio [24 ]
de Latour, Regis Peffault [25 ]
机构
[1] Newcastle Univ, Translat & Clin Res Inst, Newcastle Upon Tyne, England
[2] Great North Childrens Hosp, Clin Resource Bldg,Floor 4,Block 2,Queen Victoria, Newcastle Upon Tyne NE1 4LP, England
[3] EBMT Stat Unit, Leiden, Netherlands
[4] EBMT Leiden Study Unit, Leiden, Netherlands
[5] Royal Manchester Childrens Hosp, Manchester, England
[6] Great Ormond St Hosp Sick Children, London, England
[7] EBMT Paris Study Unit, Paris, France
[8] Wroclaw Med Univ, Dept Pediat Hematol Oncol & BMT, Wroclaw, Poland
[9] King Faisal Specialist Hosp & Res Ctr, Riyadh, Saudi Arabia
[10] Ctr Pierre & Marie Curie, Algiers, Algeria
[11] Med Pk Antalya Hosp, Antalya, Turkiye
[12] Univ Paris Cite, Hop Robert Debre, GHU AP HP Nord, Paris, France
[13] Hacettepe Univ Childrens Hosp, BMT Unit, Ankara, Turkiye
[14] Univ Hosp Children, Princess Maxima Ctr, Utrecht, Netherlands
[15] Akdeniz Univ Med Sch, Antalya, Turkiye
[16] King Hussein Canc Ctr, Amman, Jordan
[17] Russian Childrens Res Hosp, Moscow, Russia
[18] Acibadem Saglik Hizmetleri & Ticaret, Istanbul, Turkiye
[19] G Gaslini IRCCS Inst, Dept Hematol Oncol, Hematol Unit, Genoa, Italy
[20] Hosp Civils Lyon, Inst Hematol & Oncol Pediat, Lyon, France
[21] Childrens Hlth Ireland Crumlin, Dublin, Ireland
[22] Univ Regensburg, Dept Pediat Hematol Oncol & Stem Cell Transplantat, Regensburg, Germany
[23] Univ Cattolica Sacro Cuore, IRCCS Osped Pediatr Bambino Gesu, Rome, Italy
[24] AORN S Giuseppe Moscati, Avellino, Italy
[25] Univ Paris Cite, Hop St Louis, French Reference Ctr Aplast Anemia & PNH, BMT Unit, Paris, France
关键词
BONE-MARROW-TRANSPLANTATION; T-CELL; CONDITIONING REGIMEN; EUROPEAN GROUP; ACUTE-LEUKEMIA; CHILDREN; CYCLOPHOSPHAMIDE; FLUDARABINE; BLOOD; IRRADIATION;
D O I
10.1182/blood.2023022751
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
Allogeneic hematopoietic stem cell transplantation (HSCT) is the only established curative option for Fanconi anemia (FA)-associated - associated bone marrow failure (BMF)/aplastic anemia (AA) and acute myeloid leukemia (AML)/myelodysplastic syndrome (MDS). We performed a retrospective multicenter study on 813 children with FA undergoing fi rst HSCT between 2010 and 2018. Median duration of follow-up was 3.7 years. Median age at transplant was 8.8 years (IQR, 6.5-18.1). Five-year overall survival (OS), event-free survival (EFS), and graft-versus-host disease (GVHD)-free, relapse-free survival (GRFS) were 83% (95% confi dence interval [CI], 80-86), 78% (95% CI, 75-81), and 70% (95% CI, 67-74), respectively. OS was comparable between matched family donor (MFD; n = 441, 88%) and matched unrelated donor (MUD; n = 162, 86%) and was superior to that of mismatched family donor (MMFD) or mismatched unrelated donor (MMUD; n = 144, 72%) and haploidentical donor (HID; n = 66, 70%; P < .001). In multivariable analysis, a transplant indication of AML/MDS (vs AA/BMF), use of MMFD/MMUD and HID (vs MFD), and fl udarabine-cyclophosphamide (FluCy) plus other conditioning (vs FluCy) independently predicted inferior OS, whereas alemtuzumab vs antithymocyte globulin was associated with better OS. Age >= 10 years was associated with worse EFS and GRFS. Cumulative incidences (CINs) of primary and secondary graft failure were 2% and 3% respectively. CINs of grade 3 to 4 acute GVHD and chronic GVHD were 12% and 8% respectively. The 5-year CIN of secondary malignancy was 2%. These data suggest that HSCT should be offered to patients with FA with AA/BMF at a younger age in the presence of a well-matched donor.
引用
收藏
页码:1329 / 1342
页数:14
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