Reduced-toxicity myeloablative conditioning regimen using fludarabine and full doses of intravenous busulfan in pediatric patients not eligible for standard myeloablative conditioning regimens: Results of a multicenter prospective phase 2 trial.


Journal

Bone marrow transplantation
ISSN: 1476-5365
Titre abrégé: Bone Marrow Transplant
Pays: England
ID NLM: 8702459

Informations de publication

Date de publication:
11 2022
Historique:
received: 18 01 2022
accepted: 21 07 2022
revised: 18 07 2022
pubmed: 27 8 2022
medline: 5 11 2022
entrez: 26 8 2022
Statut: ppublish

Résumé

Data regarding the safety and efficacy of reduced-toxicity conditioning regimen (RTC) prior to allogeneic stem cell transplantation (allo-SCT) to treat hematological malignancies in pediatric patients are limited. This prospective multicenter, phase 2 trial investigated a RTC regimen based on the combination of intravenous busulfan (3.2 mg/kg/d x 4 days), fludarabine (30 mg/m

Identifiants

pubmed: 36028757
doi: 10.1038/s41409-022-01769-5
pii: 10.1038/s41409-022-01769-5
doi:

Substances chimiques

Busulfan G1LN9045DK
fludarabine P2K93U8740
Vidarabine FA2DM6879K

Banques de données

ClinicalTrials.gov
['NCT01572181']

Types de publication

Clinical Trial, Phase II Journal Article Multicenter Study Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

1698-1703

Informations de copyright

© 2022. The Author(s), under exclusive licence to Springer Nature Limited.

Références

Mohty M, Labopin M, Volin L, Gratwohl A, Socié G, Esteve J, et al. Reduced-intensity versus conventional myeloablative conditioning allogeneic stem cell transplantation for patients with acute lymphoblastic leukemia: A retrospective study from the European Group for blood and marrow transplantation. Blood. 2010;116:4439–43. https://doi.org/10.1182/blood-2010-02-266551
doi: 10.1182/blood-2010-02-266551 pubmed: 20716774
Pulsipher MA, Levine JE, Hayashi RJ, Chan KW, Anderson P, Osunkwo I, et al. Safety and efficacy of allogeneic PBSC collection in normal pediatric donors: The pediatric blood and marrow transplant consortium experience (PBMTC) 1996-2003. Bone Marrow Transpl. 2005;35:361–7. https://doi.org/10.1038/sj.bmt.1704743
doi: 10.1038/sj.bmt.1704743
Pulsipher MA, Nagler A, Iannone R, Nelson RM. Weighing the risks of G-CSF administration, leukopheresis, and standard marrow harvest: Ethical and safety considerations for normal pediatric hematopoietic cell donors. Pediatr Blood Cancer. 2006;46:422–33. https://doi.org/10.1002/pbc.20708
doi: 10.1002/pbc.20708 pubmed: 16411207
Pulsipher MA, Boucher KM, Wall D, Frangoul H, Duval M, Goyal RK, et al. Reduced-intensity allogeneic transplantation in pediatric patients ineligible for myeloablative therapy: results of the Pediatric Blood and Marrow Transplant Consortium Study ONC0313. Blood. 2009;114:1429–36. https://doi.org/10.1182/blood-2009-01-196303
doi: 10.1182/blood-2009-01-196303 pubmed: 19528536
Satwani P, Cooper N, Rao K, Veys P, Amrolia P. Reduced intensity conditioning and allogeneic stem cell transplantation in childhood malignant and nonmalignant diseases. Bone Marrow Transpl. 2008;41:173–82. https://doi.org/10.1038/sj.bmt.1705923
doi: 10.1038/sj.bmt.1705923
Blaise D, Vey N, Faucher C, Mohty M. Current status of reduced-intensity-conditioning allogeneic stem cell transplantation for acute myeloid leukemia. Haematologica. 2007;92:533–41. https://doi.org/10.3324/haematol.10867
doi: 10.3324/haematol.10867 pubmed: 17488664
Przepiorka D, Weisdorf D, Martin P, Klingemann HG, Beatty P, Hows J, et al. 1994 Consensus Conference on Acute GVHD Grading. Bone Marrow Transpl. 1995;15:825–8.
Rossoff J, Jacobsohn D, Kwon S, Kletzel M, Duerst RE, Tse WT, et al. Reduced-toxicity conditioning regimen with busulfan, fludarabine, rATG, and 400 cGy TBI in pediatric patients undergoing hematopoietic stem cell transplant for high-risk hematologic malignancies. Pediatr Blood Cancer. 2021;68:e29087 https://doi.org/10.1002/pbc.29087
doi: 10.1002/pbc.29087 pubmed: 34022093
Yaniv I, Krauss AC, Beohou E, Dalissier A, Corbacioglu S, Zecca M, et al. Second hematopoietic stem cell transplantation for post-transplantation relapsed acute leukemia in children: A retrospective EBMT-PDWP study. Biol Blood Marrow Transpl. 2018;24:1629–42. https://doi.org/10.1016/j.bbmt.2018.03.002
doi: 10.1016/j.bbmt.2018.03.002
Sorror ML, Maris MB, Storb R, Baron F, Sandmaier BM, Maloney DG, et al. Hematopoietic cell transplantation (HCT)-specific comorbidity index: a new tool for risk assessment before allogeneic HCT. Blood. 2005;106:2912–9. https://doi.org/10.1182/blood-2005-05-2004
doi: 10.1182/blood-2005-05-2004 pubmed: 15994282 pmcid: 1895304
Broglie L, Ruiz J, Jin Z, Kahn J, Bhatia M, George D, et al. Limitations of Applying the Hematopoietic Cell Transplantation Comorbidity Index in Pediatric Patients Receiving Allogeneic Hematopoietic Cell Transplantation. Biol Blood Marrow Transplant J Am Soc Blood Marrow Transplant. Published online October 8, 2020. https://doi.org/10.1016/j.bbmt.2020.10.003
Friend BD, Tang K, Markovic D, Elashoff D, Moore TB, Schiller GJ. Identifying risk factors associated with worse outcomes in adolescents and young adults undergoing hematopoietic stem cell transplantation. Pediatr Blood Cancer. 2019;66:e27940 https://doi.org/10.1002/pbc.27940
doi: 10.1002/pbc.27940 pubmed: 31429528
Peters C, Dalle JH, Locatelli F, Poetschger U, Sedlacek P, Buechner J, et al. Total body irradiation or chemotherapy conditioning in childhood ALL: A Multinational, Randomized, Noninferiority Phase III Study. J Clin Oncol J Am Soc Clin Oncol. 2021;39:295–307. https://doi.org/10.1200/JCO.20.02529
doi: 10.1200/JCO.20.02529
Eapen M, Horowitz MM, Klein JP, Champlin RE, Loberitza FR Jr, Ringden O, et al. Higher mortality after allogeneic peripheral-blood transplantation compared with bone marrow in children and adolescents: The Histocompatibility and Alternate Stem Cell Source Working Committee of the International Bone Marrow Transplant Registry. J Clin Oncol J Am Soc Clin Oncol. 2004;22:4872–80. https://doi.org/10.1200/JCO.2004.02.189
doi: 10.1200/JCO.2004.02.189
Lucchini G, Labopin M, Beohou E, Dalissier A, Dalle JH, Cornish J, et al. Impact of conditioning regimen on outcomes for children with acute myeloid leukemia undergoing transplantation in first complete remission. an analysis on behalf of the pediatric disease working party of the European group for blood and marrow transplantation. Biol Blood Marrow Transpl J Am Soc Blood Marrow Transpl. 2017;23:467–74. https://doi.org/10.1016/j.bbmt.2016.11.022
doi: 10.1016/j.bbmt.2016.11.022
Sauer MG, Lang PJ, Albert MH, Bader P, Creutzig U, Eyrich M, et al. Hematopoietic stem cell transplantation for children with acute myeloid leukemia—results of the AML SCT-BFM 2007 trial. Leukemia. 2020;34:613–24. https://doi.org/10.1038/s41375-019-0584-8
doi: 10.1038/s41375-019-0584-8 pubmed: 31578451
Ishida H, Adachi S, Hasegawa D, Okamoto Y, Goto H, Inagaki J, et al. Comparison of a fludarabine and melphalan combination-based reduced toxicity conditioning with myeloablative conditioning by radiation and/or busulfan in acute myeloid leukemia in Japanese children and adolescents. Pediatr Blood Cancer. 2015;62:883–9. https://doi.org/10.1002/pbc.25389
doi: 10.1002/pbc.25389 pubmed: 25545836
Balduzzi A, Dalle JH, Wachowiak J, Yaniv I, Yesilipek A, Sedlacek P, et al. Transplantation in Children and Adolescents with Acute Lymphoblastic Leukemia from a Matched Donor versus an HLA-Identical Sibling: Is the Outcome Comparable? Results from the International BFM ALL SCT 2007 Study. Biol Blood Marrow Transpl. 2019;25:2197–210. https://doi.org/10.1016/j.bbmt.2019.07.011
doi: 10.1016/j.bbmt.2019.07.011
Hirabayashi K, Nakazawa Y, Sakashita K, Kurata T, Saito S, Yoshikawa K, et al. Reduced-toxicity myeloablative conditioning consisting of 8-Gy total body irradiation, cyclophosphamide and fludarabine for pediatric hematological malignancies. Sci Rep. 2014;4:6942 https://doi.org/10.1038/srep06942
doi: 10.1038/srep06942 pubmed: 25373730 pmcid: 4221788
Ueda M, de Lima M, Caimi P, Tomlinson B, Little J, Creger R, et al. Concurrent blinatumomab and donor lymphocyte infusions for treatment of relapsed pre-B-cell ALL after allogeneic hematopoietic cell transplant. Bone Marrow Transpl. 2016;51:1253–5. https://doi.org/10.1038/bmt.2016.104
doi: 10.1038/bmt.2016.104
Zahler S, Bhatia M, Ricci A, Roy S, Morris E, Harrison L, et al. A Phase I Study of Reduced-Intensity Conditioning and Allogeneic Stem Cell Transplantation Followed by Dose Escalation of Targeted Consolidation Immunotherapy with Gemtuzumab Ozogamicin in Children and Adolescents with CD33+ Acute Myeloid Leukemia. Biol Blood Marrow Transplant J Am Soc Blood Marrow Transpl. 2016;22:698–704. https://doi.org/10.1016/j.bbmt.2016.01.019
doi: 10.1016/j.bbmt.2016.01.019

Auteurs

Fanny Rialland (F)

CHU de Nantes, Nantes, France. fanny.rialland@chu-nantes.fr.

Audrey Grain (A)

CHU de Nantes, Nantes, France.

Myriam Labopin (M)

Sorbonne University, Saint-Antoine Hospital, AP-HP, INSERM UMRs 938, Paris, France.

Gerard Michel (G)

Department of Pediatric Immunology, Hematology and Oncology, Timone Enfants Hospital and Aix-Marseille University, Marseille, France.

Virginie Gandemer (V)

University hospital of Rennes, University of Rennes1, Rennes, France.

Catherine Paillard (C)

Department of Paediatric Haematology and Oncology, Strasbourg University Hospital, Strasbourg, France.

Cécile Pochon (C)

Hôpital Brabois, Nancy, France.

Laurence Clement (L)

CHU Bordeaux, Bordeaux, France.

Eolia Brissot (E)

Sorbonne University, Saint-Antoine Hospital, AP-HP, INSERM UMRs 938, Paris, France.

Charlotte Jubert (C)

CHU Bordeaux, Bordeaux, France.

Anne Sirvent (A)

University Hospital, Montpellier, France.

Pierre Simon Rohrlich (PS)

Nice University Hospital, Nice, France.

Dominique Plantaz (D)

University Hospital of Grenoble, La Tronche, France.

Jean-Hugues Dalle (JH)

Hôpital Robert Debré, GH APHP Nord - Université de Paris, Paris, France.

Mohamad Mohty (M)

Sorbonne University, Saint-Antoine Hospital, AP-HP, INSERM UMRs 938, Paris, France.

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