Comparable outcomes after busulfan- or treosulfan-based conditioning for allo-HSCT in children with ALL: results of FORUM

被引：0

作者：

Kalwak, Krzysztof ^{[1
]}

Moser, Laura M. ^{[2
]}

Poetschger, Ulrike ^{[3
]}

Bader, Peter ^{[2
]}

Kleinschmidt, Katharina ^{[4
]}

Meisel, Roland ^{[5
]}

Dalle, Jean-Hugues ^{[6
]}

Yesilipek, Akif ^{[7
]}

Balduzzi, Adriana ^{[8
,9
]}

Krivan, Gergely ^{[10
]}

Goussetis, Evgenios ^{[11
]}

Staciuk, Raquel ^{[12
]}

Sedlacek, Petr ^{[13
]}

Pichler, Herbert ^{[3
,14
]}

Svec, Peter ^{[15
]}

Gabriel, Melissa ^{[16
]}

Guengoer, Tayfun ^{[17
]}

Bilic, Ernest ^{[18
]}

Buechner, Jochen ^{[19
]}

Renard, Marleen ^{[20
]}

Vettenranta, Kim ^{[21
,22
]}

Ifversen, Marianne ^{[23
]}

Diaz-de-Heredia, Cristina ^{[24
]}

Stein, Jerry ^{[25
,26
]}

Toporski, Jacek ^{[27
]}

Bierings, Marc ^{[28
]}

Peters, Christina ^{[3
,14
]}

Ansari, Marc ^{[29
,30
]}

Locatelli, Franco ^{[1
,31
]}

机构：

[1] Wroclaw Med Univ, Dept Pediat Hematol Oncol & Bone Marrow Transplant, Wroclaw, Poland

[2] Goethe Univ Frankfurt, Univ Hosp, Dept Pediat, Div Stem Cell Transplantat & Immunol, Frankfurt, Germany

[3] St Anna Childrens Canc Res Inst, Vienna, Austria

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

[5] Heinrich Heine Univ, Med Fac, Dept Pediat Oncol Hematol & Clin Immunol, Div Pediat Stem Cell Therapy, Dusseldorf, Germany

[6] Univ Paris Cite, Hop Paris Nord, Hematol & Immunol Pediat Unit, Grp Hosp Univ Assistance Publ, Paris, France

[7] Med Pk Antalya Hosp, Antalya, Turkiye

[8] Univ Milano Bicocca, Sch Med & Surg, Monza, Italy

[9] Fdn IRCCS San Gerardo Tintori, Unit Radiodiagnost, Monza, Italy

[10] Cent Hosp Southern Pest, Natl Inst Hematol & Infect Dis, Pediat Hematol & Stem Cell Transplantat Dept, Budapest, Hungary

[11] Agia Sofia Childrens Hosp, Stem Cell Transplant Unit, Athens, Greece

[12] Hosp Pediat Prof Dr Juan P Garrahan, Buenos Aires, Argentina

[13] Univ Hosp Motol, Dept Pediat Hematol & Oncol, Prague, Czech Republic

[14] Med Univ Vienna, St Anna Childrens Hosp, Dept Pediat Hematol Oncol, Vienna, Austria

[15] Comenius Univ, Natl Inst Childrens Dis, Dept Pediat Hematol & Oncol, Bratislava, Slovakia

[16] Childrens Hosp Westmead, Sydney, NSW, Australia

[17] Univ Childrens Hosp Zurich, Eleonore Fdn & Childrens Res Ctr, Div Hematol Oncol Immunol Gene Therapy & Stem Cell, Zurich, Switzerland

[18] Univ Hosp Ctr, Dept Pediat Zagreb, Div Hematol & Oncol, ZAGREB, Croatia

[19] Oslo Univ Hosp, Dept Pediat Hematol & Oncol, Oslo, Norway

[20] Univ Hosp Leuven, Dept Paediat Oncol, Leuven, Belgium

[21] Univ Helsinki, Helsinki, Finland

[22] Univ Helsinki, Hosp Children & Adolescents, Helsinki, Finland

[23] Copenhagen Univ Hosp, Rigshospitalet, Dept Pediat & Adolescent Med, Copenhagen, Denmark

[24] Hosp Univ Vall Hebron, Pediat Endocrinol Sect, Barcelona 08035, Spain

[25] Tel Aviv Univ, Petah Tiqwa, Israel

[26] Tel Aviv Univ, Sackler Fac Med, Petah Tiqwa, Israel

[27] Karolinska Univ Hosp, Dept Cellular Therapy Allogene Stem Cell Transpla, Stockholm, Sweden

[28] Princess Maxima Ctr Pediat Oncol, Utrecht, Netherlands

[29] Univ Geneva, Fac Med, Dept Pediat Gynecol & Obstet, CANSEARCH Res Platform Pediat Oncol & Hematol, Geneva, Switzerland

[30] Univ Geneva Hosp, Div Pediat Oncol & Hematol, Dept Women Child & Adolescent, Geneva, Switzerland

[31] Osped Pediatr Bambino Gesu, IRCCS, Dept Cardiol, I-00165 Rome, Italy

来源：

BLOOD ADVANCES | 2025年 / 9卷 / 04期

关键词：

STEM-CELL TRANSPLANTATION; ACUTE LYMPHOBLASTIC-LEUKEMIA; TOTAL-BODY IRRADIATION; LONG-TERM; PEDIATRIC-PATIENTS; REGIMEN; MARROW; TOXICITY; DISEASE; BLOOD;

D O I：

10.1182/bloodadvances.2024014548

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

The superiority of total body irradiation (TBI)-based vs chemotherapy conditioning for allogeneic hematopoietic stem cell transplantation (allo-HSCT) in children with acute lymphoblastic leukemia (ALL) has been established in the international, prospective phase- 3 FORUM study, randomizing 417 patients aged 4-18 years in complete remission (CR), who received allo-HSCT from HLA-matched sibling or unrelated donors. Because of the unavailability of TBI in some regions and to accommodate individual contraindications, this study reports the prespecified comparison of outcomes of patients receiving busulfan (BU)- or treosulfan (TREO)-based regimens from 2013 to 2018. Overall, 180 and 128 patients received BU/thiotepa (THIO)/fludarabine (FLU) or TREO/THIO/FLU, respectively. Data were analyzed as of February 2023, with a median follow-up of 4.2 years (range, 0.3-9.1). 3-year overall survival was 0.71 (BU, 95% confidence interval [0.64-0.77]) and 0.72 (TREO, [0.630.79]) and 3-year event-free survival was 0.60 (BU, [0.53-0.67]) and 0.55 (TREO, [0.46-0.63]). The 3-year cumulative incidence of relapse (BU, 0.31 [0.25-0.38]; TREO, 0.36 [0.27-0.44]); and nonrelapse mortality (BU, 0.08 [0.05-0.13]; TREO, 0.09 [0.05-0.15]) were comparable. One case of fatal veno-occlusive disease occurred in each group. No significant differences in acute and chronic graft-versus-host disease (GVHD) or 3-year GVHD-free and relapse-free survival (BU, 0.48 [0.41-0.55]; TREO, 0.45 [0.37-0.54]) were recorded. Outcomes for patients in first and second CR were similar irrespective of the regimen. In conclusion, BU/THIO/FLU or TREO/THIO/FLU regimens can be an alternative to TBI for patients with ALL aged >4 years with contraindications or lack of access to TBI. This trial was registered at www. ClinicalTrials.gov as #NCT01949129.

引用

页码：741 / 751

页数：11

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