HLA-haploidentical stem cell transplantation in children with inherited bone marrow failure syndromes: A retrospective analysis on behalf of EBMT severe aplastic Anemia and pediatric diseases working parties

被引：3

作者：

Giardino, Stefano ^{[1
,28
]}

Eikema, Dirk-Jan ^{[2
]}

Piepenbroek, Brian ^{[3
]}

Algeri, Mattia ^{[4
,5
]}

Ayas, Mouhab ^{[6
]}

Faraci, Maura ^{[1
]}

Tbakhi, Abdelghani ^{[7
]}

Zecca, Marco ^{[8
]}

Essa, Mohammed ^{[9
]}

Neven, Benedicte ^{[10
]}

Bertrand, Yves ^{[11
]}

Kharya, Gaurav ^{[12
]}

Bykova, Tatiana ^{[13
]}

Lawson, Sarah ^{[14
]}

Petrini, Mario ^{[15
]}

Mohseny, Alexander ^{[16
]}

Rialland, Fanny ^{[17
]}

James, Beki ^{[18
]}

Colita, Anca ^{[19
]}

Fahd, Mony ^{[20
]}

Cesaro, Simone ^{[21
]}

Schulz, Ansgar ^{[22
]}

Kleinschmidt, Katharina ^{[23
]}

Kalwak, Krzysztof ^{[24
]}

Corbacioglu, Selim ^{[23
]}

Dufour, Carlo ^{[25
]}

Risitano, Antonio ^{[26
]}

de Latour, Regis Peffault ^{[27
]}

机构：

[1] IRCCS Ist Giannina Gaslini, Dept Pediat Hematol & Oncol, Hematopoiet Stem Cell Transplantat Unit, Genoa, Italy

[2] European Soc Blood & Marrow Transplantat EBMT, Stat Unit & Data Off, Leiden, Netherlands

[3] EBMT Leiden Study Unit, Leiden, Netherlands

[4] IRRCS Bambino Gesu Childrens Hosp, Dept Pediat Haematol & Oncol & Stem & Gene Therapy, Rome, Italy

[5] Magna Graecia Univ Catanzaro, Dept Hlth Sci, Catanzaro, Italy

[6] King Faisal Specialist Hosp & Res Ctr, Dept Pediat Hematol Oncol, Riyadh, Saudi Arabia

[7] King Hussein Canc Ctr, Amman, Jordan

[8] Fdn IRCCS Policlin San Matteo, Pediat Hematol Oncol, Pavia, Italy

[9] King Saud bin Abdulaziz Univ Hlth Sci, King Abdullah Specialist Childrens Hosp, King Abdullah Int Med Res Ctr, Minist Natl Guard Hlth Affairs, Riyadh, Saudi Arabia

[10] Necker Children Hosp, AP HP, Pediat Immune Hematol Unit, Paris, France

[11] Hosp Civil Lyon, Inst Hemato Oncol Pediat, Paris, France

[12] Indraprastha Apollo Hosp, Ctr Bone Marrow Transplant & Cellular Therapy, New Delhi, India

[13] Pavlov Univ, RM Gorbacheva Res Inst, St Petersburg, Russia

[14] Birmingham Childrens Hosp, Dept Haematol, Birmingham, England

[15] Univ Pisa, Santa Chiara Univ Hosp, Unit Hematol, Pisa, Italy

[16] Leiden Univ, Willem Alexander Childrens Hosp, Pediat SCT Program, Med Ctr, Leiden, Netherlands

[17] Hop Mere Enfant, Serv Chirurg Pediat, Nantes, France

[18] Leeds Childrens Hosp, Leeds Gen Infirm, Leeds, England

[19] Carol Davila Univ Med & Pharm, Fundeni Clin Inst, Bucharest, Romania

[20] GHU APHP Nord Univ Paris Cite, Hematol & Immunol Pediat Dept, Paris, France

[21] Azienda Osped Univ Integrata Verona, Dept Mother & Child, Pediat Hematol Oncol, Verona, Italy

[22] Univ Hosp Ulm, Dept Pediat Med, Eythstr 24, Ulm, Germany

[23] Univ Childrens Hosp Regensburg, Dept Pediat Hematol Oncol & Stem Cell Transplantat, Regensburg, Germany

[24] Wroclaw Med Univ, Suprareg Ctr Pediat Oncol Cape Hope, Wroclaw, Poland

[25] IRCCS Ist Giannina Gaslini, Dept Pediat Hematol & Oncol, Hematol Unit, Genoa, Italy

[26] Azienda Osped Rilievo Nazl San Giuseppe Moscati AO, Hematol & Hematopoiet Transplant Unit, Avellino, Italy

[27] Hop St Louis, AP HP, Bone Marrow Transplant Unit, Paris, France

[28] Ist Giannina Gaslini, Hematopoiet Stem Cell Transplantat Unit, Lgo Gaslini 5, I-16147 Genoa, GE, Italy

来源：

CURRENT SCIENCE | 2024年 / 126卷 / 08期

关键词：

VERSUS-HOST-DISEASE; POSTTRANSPLANT CYCLOPHOSPHAMIDE; FANCONI-ANEMIA; GRAFT FAILURE; ALPHA-BETA; B-CELLS; DEPLETION; BLOOD; ADOLESCENTS; OUTCOMES;

D O I：

10.1002/ajh.27293

中图分类号：

O [数理科学和化学]; P [天文学、地球科学]; Q [生物科学]; N [自然科学总论];

学科分类号：

07 ; 0710 ; 09 ;

摘要：

Haploidentical stem cell transplantation (haplo-SCT) represents the main alternative for children with inherited bone marrow failure syndrome (I-BMF) lacking a matched donor. This retrospective study, conducted on behalf of the EBMT SAAWP and PDWP, aims to report the current outcomes of haplo-SCT in I-BMFs, comparing the different in vivo and ex vivo T-cell depletion approaches. One hundred and sixty-two I-BMF patients who underwent haplo-SCT (median age 7.4 years) have been registered. Fanconi Anemia was the most represented diagnosis (70.1%). Based on different T-cell depletion (TCD) approaches, four categories were identified: (1) TCR alpha beta(+)/CD19+-depletion (43.8%); (2) T-repleted with post-transplant Cyclophosphamide (PTCy, 34.0%); (3) In-vivo T-depletion with ATG/alemtuzumab (14.8%); (4) CD34(+) positive selection (7.4%). The cumulative incidences (CI) of neutrophil and platelet engraftment were 84% and 76% respectively, while that of primary and secondary graft failure was 10% and 8% respectively. The 100-day CI of acute GvHD grade III-IV(95% CI) was 13%, while the 24-month CI of extensive chronic GvHD was 4%. After a median follow-up of 43.4 months, the 2-year overall survival(OS) and GvHD/Rejection-free Survival (GRFS) probabilities are 67% and 53%, respectively. The TCR CD3(+)alpha beta(+)/CD19(+) depletion group showed a significantly lower incidence of both acute and chronic GvHD and higher OS (79%; p0.013) and GRFS (71%; p < .001), while no significant differences in outcomes have been observed by different diagnosis and conditioning regimens. This large retrospective study supports the safety and feasibility of haplo-SCT in I-BMF patients. TCR alpha beta(+)/CD19(+ )depletion offers higher chances of patients' survival, with a significantly lower risk of severe a- and c-GvHD in I-BMFs compared to other platforms.

引用

页码：1066 / 1076

页数：4

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