Evidence map›Paper›PMID 37738088›Full record

Trial reportBlood advances2024

Low rate of nonrelapse mortality in under-4-year-olds with ALL given chemotherapeutic conditioning for HSCT: a phase 3 FORUM study.

Peter Bader, Ulrike Pötschger, Jean-Hugues Dalle, Laura M Moser, Adriana Balduzzi, Marc Ansari, Jochen Buechner, Tayfun Güngör, Marianne Ifversen, Gergely Krivan and 11 more

2 registry-linked trialsOpen access · goldAbstract readClinical Trial, Phase III
In one paragraph

Trial report in Blood advances, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 11 papers.

0numbers the graph read from it
0cells of the map it votes in
11citing papers in PubMed
6.4field-weighted citation impact, top 3% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT01949129 phase2 / phase3recruitingnot on this map

Allogeneic Stem Cell Transplantation for Children and Adolescents With Acute Lymphoblastic Leukaemia

TypeinterventionalSponsorSt. Anna KinderkrebsforschungRan2013 to 2030Enrolled1,800ConditionsAcute Lymphoblastic LeukaemiaArmsVP16, TBI, Thiotepa, Treosulfan, Fludarabine
NCT07297914 phase2 / phase3not yet recruitingnot on this mapstarted 2026, after this paper: background citation

Framework for Optimizing, Refining, and Unifying Management of HSCT in Pediatric ALL

TypeinterventionalSponsorBambino Gesù Hospital and Research InstituteRan2026 to 2032Enrolled1,000ConditionsAcute Lymphoblastic Leukemia (ALL), Stem Cell Transplant, Graft -Versus-host-diseaseArmsTotal Body Irradiation 8 Gy, Ruxolitinib, Blinatumomab, Cyclophosphamide, Total Body Irradiation 12 Gy
3 · Its place in the literature

Who cites it

11 citing papers in PubMed, 11 citations in OpenAlex.

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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

21 authors at 17 institutions in 16 countries.

Peter BaderGoethe University, University Hospital, Department of Pediatrics, Division for Stem Cell Transplantation, Immunology and Intensive Care Medicine, Frankfurt, Germany.ORCID 0000-0003-4554-0265
Ulrike PötschgerSt. Anna Children's Cancer Research Institute, Vienna, Austria.
Jean-Hugues DallePediatric Hematology and Immunology Department, Robert Debré Hospital, Groupe Hospitalo-Universitaire Assistance Publique Hôpitaux de Paris (GHU AP-HP) Nord, Université Paris Cité, Paris, France.
Laura M MoserGoethe University, University Hospital, Department of Pediatrics, Division for Stem Cell Transplantation, Immunology and Intensive Care Medicine, Frankfurt, Germany.
Adriana BalduzziUniversità degli Studi di Milano-Fondazione, FONDAZIONE MONZA E BRIANZA PER IL BAMBINO E LA SUA MAMMA (MBBM), Department for Pediatric Hematology and Oncology, Monza, Italy.ORCID 0000-0002-5879-0610
Marc AnsariCANSEARCH Research Platform for Pediatric Oncology and Hematology, Faculty of Medicine, Department of Pediatrics, Gynecology and Obstetrics, University of Geneva, Switzerland.
Jochen BuechnerDepartment of Pediatric Hematology and Oncology, Oslo University Hospital, Oslo, Norway.ORCID 0000-0001-5848-4501
Tayfun GüngörDepartment of Hematology/Oncology/Immunology, Gene Therapy, and Stem Cell Transplantation, University Children's Hospital Zürich, Eleonore Foundation & Children's Research Center, Zürich, Switzerland.ORCID 0000-0002-3261-1186
Marianne IfversenCopenhagen University Hospital Rigshospitalet, Department for Pediatric Hematology and Oncology, Copenhagen, Denmark.
Gergely KrivanPediatric Hematology and Stem Cell Transplantation Department, Central Hospital of Southern Pest, National Institute of Hematology and Infectious Diseases, Budapest, Hungary.
Herbert PichlerSt. Anna Children's Hospital, University Vienna, Vienna, Austria.ORCID 0000-0001-5107-5123
Marleen RenardDepartment of Paediatric Oncology, University Hospital Leuven, Leuven, Belgium.
Raquel StaciukHospital de Pediatría "Prof. Dr. Juan P. Garrahan," Buenos Aires, Buenos Aires, Argentina.
Petr SedlacekDepartment of Pediatric Hematology and Oncology, Motol University Hospital, Prague, Czech Republic.ORCID 0000-0001-7895-2886
Jerry SteinSchneider Children's Medical Center of Israel and Sackler Faculty of Medicine, Tel Aviv University, Petah Tikva, Israel.
Jan Robert HeuselGoethe University, University Hospital, Department of Pediatrics, Division for Stem Cell Transplantation, Immunology and Intensive Care Medicine, Frankfurt, Germany.ORCID 0000-0002-5753-0817
Tony TruongDivision of Pediatric Oncology and Cellular Therapy, Alberta Children's Hospital, Calgary, Alberta, Canada.
Jacek WachowiakDepartment of Pediatric Oncology, Hematology and Transplantology, Poznań University of Medical Sciences, Poznań, Poland.
Akif YesilipekMedical Park Antalya Hospital, Antalya, Turkey.
Franco LocatelliDepartment of Pediatric Hematology and Oncology, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ospedale Pediatrico Bambino Gesù, Catholic University of the Sacred Heart, Rome, Italy.
Christina PetersSt. Anna Children's Cancer Research Institute, Vienna, Austria.ORCID 0000-0003-0369-8515
Goethe University Frankfurt · DECancer Research Institute of the Slovak Academy of Sciences · SKAlberta Children's Hospital · CAAntalya IVF · TRCopenhagen University Hospital · DKFondazione Lanza · ITGarrahan Hospital · ARKU Leuven · BEOslo University Hospital · NOPoznan University of Medical Sciences · PLSt Anna Children's Hospital · ATTel Aviv University · ILUniversità Cattolica del Sacro Cuore · ITUniversité Paris Cité · FRUniversity Children's Hospital Zurich · CHUniversity Hospital in Motol · CZUniversity of Geneva · CH

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

abstractAllogeneic hematopoietic stem cell transplantation (HSCT) is highly effective for treating pediatric high-risk or relapsed acute lymphoblastic leukemia (ALL). For young children, total body irradiation (TBI) is associated with severe late sequelae. In the FORUM study (NCT01949129), we assessed safety, event-free survival (EFS), and overall survival (OS) of 2 TBI-free conditioning regimens in children aged <4 years with ALL. Patients received fludarabine (Flu), thiotepa (Thio), and either busulfan (Bu) or treosulfan (Treo) before HSCT. From 2013 to 2021, 191 children received transplantation and were observed for ≥6 months (median follow-up: 3 years). The 3-year OS was 0.63 (95% confidence interval [95% CI], 0.52-0.72) and 0.76 (95% CI, 0.64-0.84) for Flu/Thio/Bu and Flu/Thio/Treo (P = .075), respectively. Three-year EFS was 0.52 (95% CI, 0.41-0.61) and 0.51 (95% CI, 0.39-0.62), respectively (P = .794). Cumulative incidence of nonrelapse mortality (NRM) and relapse at 3 years were 0.06 (95% CI, 0.02-0.12) vs 0.03 (95% CI: <0.01-0.09) (P = .406) and 0.42 (95% CI, 0.31-0.52) vs 0.45 (95% CI, 0.34-0.56) (P = .920), respectively. Grade >1 acute graft-versus-host disease (GVHD) occurred in 29% of patients receiving Flu/Thio/Bu and 17% of those receiving Flu/Thio/Treo (P = .049), whereas grade 3/4 occurred in 10% and 9%, respectively (P = .813). The 3-year incidence of chronic GVHD was 0.07 (95% CI, 0.03-0.13) vs 0.05 (95% CI, 0.02-0.11), respectively (P = .518). In conclusion, both chemotherapeutic conditioning regimens were well tolerated and NRM was low. However, relapse was the major cause of treatment failure. This trial was registered at www.clinicaltrials.gov as #NCT01949129.

Indexed as

BusulfanGraft vs Host DiseaseHematopoietic Stem Cell TransplantationPrecursor Cell Lymphoblastic Leukemia-LymphomaChildChild, PreschoolHumansRecurrenceThiotepaTransplantation ConditioningBusulfanThiotepatreosulfan

Identifiers

PMID37738088
PMCPMC10827403
OpenAlexW4386945494

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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.