Proposed Therapeutic Range of Treosulfan in Reduced Toxicity Pediatric Allogeneic Hematopoietic Stem Cell Transplant Conditioning: Results From a Prospective Trial.


Journal

Clinical pharmacology and therapeutics
ISSN: 1532-6535
Titre abrégé: Clin Pharmacol Ther
Pays: United States
ID NLM: 0372741

Informations de publication

Date de publication:
08 2020
Historique:
received: 07 08 2019
accepted: 28 10 2019
pubmed: 9 11 2019
medline: 25 5 2021
entrez: 9 11 2019
Statut: ppublish

Résumé

Treosulfan is given off-label in pediatric allogeneic hematopoietic stem cell transplant. This study investigated treosulfan's pharmacokinetics (PKs), efficacy, and safety in a prospective trial. Pediatric patients (n = 87) receiving treosulfan-fludarabine conditioning were followed for at least 1 year posttransplant. PKs were described with a two-compartment model. During follow-up, 11 of 87 patients died and 12 of 87 patients had low engraftment (≤ 20% myeloid chimerism). For each increase in treosulfan area under the curve from zero to infinity (AUC

Identifiants

pubmed: 31701524
doi: 10.1002/cpt.1715
pmc: PMC7484914
doi:

Substances chimiques

Myeloablative Agonists 0
treosulfan CO61ER3EPI
Vidarabine FA2DM6879K
Busulfan G1LN9045DK
fludarabine P2K93U8740

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

264-273

Subventions

Organisme : Medical Research Council
ID : MR/P014534/1
Pays : United Kingdom
Organisme : Medical Research Council
ID : MR/M008665/1
Pays : United Kingdom

Informations de copyright

© 2019 The Authors. Clinical Pharmacology & Therapeutics published by Wiley Periodicals, Inc. on behalf of American Society for Clinical Pharmacology and Therapeutics.

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Auteurs

Robert Chiesa (R)

Bone Marrow Transplantation Department, Great Ormond Street Hospital for Children, NHS Foundation Trust, London, UK.

Joseph F Standing (JF)

Pharmacy Department, Great Ormond Street Hospital for Children, NHS Foundation Trust, London, UK.
Infection, Immunity, and Inflammation, Great Ormond Street Institute of Child Health, University College London, London, UK.

Robert Winter (R)

Chemical Pathology Department, Great Ormond Street Hospital for Children,, NHS Foundation Trust, London, UK.

Zohreh Nademi (Z)

Bone Marrow Transplantation Department, Great Ormond Street Hospital for Children, NHS Foundation Trust, London, UK.
Bone Marrow Transplantation Department, Great North Children's Hospital, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.

Jan Chu (J)

Bone Marrow Transplantation Department, Great Ormond Street Hospital for Children, NHS Foundation Trust, London, UK.

Danielle Pinner (D)

Bone Marrow Transplantation Department, Great Ormond Street Hospital for Children, NHS Foundation Trust, London, UK.

Frank Kloprogge (F)

Institute for Global Health, University College London, London, UK.

Susan McLellen (S)

Clinical Biochemistry, Integrated Laboratory Medicine Directorate, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.

Persis J Amrolia (PJ)

Bone Marrow Transplantation Department, Great Ormond Street Hospital for Children, NHS Foundation Trust, London, UK.
Infection, Immunity, and Inflammation, Great Ormond Street Institute of Child Health, University College London, London, UK.

Kanchan Rao (K)

Bone Marrow Transplantation Department, Great Ormond Street Hospital for Children, NHS Foundation Trust, London, UK.

Giovanna Lucchini (G)

Bone Marrow Transplantation Department, Great Ormond Street Hospital for Children, NHS Foundation Trust, London, UK.

Juliana Silva (J)

Bone Marrow Transplantation Department, Great Ormond Street Hospital for Children, NHS Foundation Trust, London, UK.

Oana Ciocarlie (O)

Bone Marrow Transplantation Department, Great Ormond Street Hospital for Children, NHS Foundation Trust, London, UK.

Arina Lazareva (A)

Bone Marrow Transplantation Department, Great Ormond Street Hospital for Children, NHS Foundation Trust, London, UK.

Andrew R Gennery (AR)

Bone Marrow Transplantation Department, Great North Children's Hospital, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.

Bilyana Doncheva (B)

Pharmacy Department, Great Ormond Street Hospital for Children, NHS Foundation Trust, London, UK.

Andrew J Cant (AJ)

Bone Marrow Transplantation Department, Great North Children's Hospital, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.

Sophie Hambleton (S)

Bone Marrow Transplantation Department, Great North Children's Hospital, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.

Terence Flood (T)

Bone Marrow Transplantation Department, Great North Children's Hospital, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.

Elizabeth Rogerson (E)

Bone Marrow Transplantation Department, Great North Children's Hospital, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.

Kirsty Devine (K)

Bone Marrow Transplantation Department, Great North Children's Hospital, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.

Helen Prunty (H)

Chemical Pathology Department, Great Ormond Street Hospital for Children,, NHS Foundation Trust, London, UK.

Simon Heales (S)

Chemical Pathology Department, Great Ormond Street Hospital for Children,, NHS Foundation Trust, London, UK.

Paul Veys (P)

Bone Marrow Transplantation Department, Great Ormond Street Hospital for Children, NHS Foundation Trust, London, UK.
Infection, Immunity, and Inflammation, Great Ormond Street Institute of Child Health, University College London, London, UK.

Mary Slatter (M)

Bone Marrow Transplantation Department, Great North Children's Hospital, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.

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Classifications MeSH