-
Something wrong with this record ?
Haematopoietic stem cell transplantation after reduced intensity conditioning in children and adolescents with chronic myeloid leukaemia: A prospective multicentre trial of the I-BFM Study Group
H. Pichler, P. Sedlacek, R. Meisel, R. Beier, M. Faraci, K. Kalwak, M. Ifversen, I. Müller, J. Stein, K. Vettenranta, G. Kropshofer, A. Kolenova, S. Karlhuber, E. Glogova, U. Poetschger, C. Peters, M. Suttorp, S. Matthes-Leodolter, A. Balduzzi
Language English Country England, Great Britain
Document type Journal Article, Multicenter Study, Clinical Trial
PubMed
38803040
DOI
10.1111/bjh.19535
Knihovny.cz E-resources
- MeSH
- Leukemia, Myelogenous, Chronic, BCR-ABL Positive * therapy mortality MeSH
- Child MeSH
- Protein Kinase Inhibitors therapeutic use MeSH
- Humans MeSH
- Adolescent MeSH
- Graft vs Host Disease etiology prevention & control MeSH
- Child, Preschool MeSH
- Transplantation Conditioning * methods MeSH
- Prospective Studies MeSH
- Hematopoietic Stem Cell Transplantation * methods MeSH
- Treatment Outcome MeSH
- Check Tag
- Child MeSH
- Humans MeSH
- Adolescent MeSH
- Male MeSH
- Child, Preschool MeSH
- Female MeSH
- Publication type
- Journal Article MeSH
- Clinical Trial MeSH
- Multicenter Study MeSH
This prospective multicentre trial evaluated the safety and the efficacy of a thiotepa/melphalan-based reduced intensity conditioning (RIC) haematopoietic stem cell transplantation (HSCT) in children and adolescents with chronic myeloid leukaemia (CML) in chronic phase (CP). Thirty-two patients were transplanted from matched siblings or matched unrelated donors. In 22 patients, HSCT was performed due to insufficient molecular response or loss of response to first- or second-generation tyrosine kinase inhibitor (TKI), with pretransplant BCR::ABL1 transcripts ranging between 0.001% and 33%. The protocol included a BCR::ABL1-guided intervention with TKI retreatment in the first year and donor lymphocyte infusions (DLI) in the second-year post-transplant. All patients engrafted. The 1-year transplant-related mortality was 3% (confidence interval [CI]: 0%-6%). After a median follow-up of 6.3 years, 5-year overall survival and event-free survival are 97% (CI: 93%-100%) and 91% (CI: 79%-100%) respectively. The current 5-year leukaemia-free survival with BCR::ABL1 <0.01% is 97% (CI: 88%-100%) and the current TKI- and DLI-free survival is 95% (CI: 85%-100%). The incidence of chronic graft-versus-host disease (GvHD) was 32%, being severe in four patients (13%). At last follow-up, 31 patients are GvHD-free and have stopped immunosuppression. RIC HSCT following pretreatment with TKI is feasible and effective in children and adolescents with CP-CML with an excellent disease-free and TKI-free survival.
Children's Cancer Research Institute Medical University of Vienna Vienna Austria
Department of Hematology Oncology Schneider Children's Medical Center of Israel Petah Tikva Israel
Department of Paediatric Haematology and Oncology St Anna Children's Hospital Vienna Austria
Department of Paediatrics and Adolescent Medicine Medical University of Vienna Vienna Austria
Department of Pediatric Hematology and Oncology Hannover Medical School Hannover Germany
Department of Pediatrics Medical University of Innsbruck Innsbruck Austria
Paediatric Haemato Oncology University of Helsinki Helsinki Finland
Pediatric Transplant Unit Fondazione IRCCS San Gerardo Dei Tintori Monza Italy
School of Medicine and Surgery University of Milano Bicocca Milan Italy
References provided by Crossref.org
- 000
- 00000naa a2200000 a 4500
- 001
- bmc24019897
- 003
- CZ-PrNML
- 005
- 20241024111013.0
- 007
- ta
- 008
- 241015s2024 enk f 000 0|eng||
- 009
- AR
- 024 7_
- $a 10.1111/bjh.19535 $2 doi
- 035 __
- $a (PubMed)38803040
- 040 __
- $a ABA008 $b cze $d ABA008 $e AACR2
- 041 0_
- $a eng
- 044 __
- $a enk
- 100 1_
- $a Pichler, Herbert $u Department of Paediatric Haematology and Oncology, St. Anna Children's Hospital, Vienna, Austria $u Department of Paediatrics and Adolescent Medicine, Medical University of Vienna, Vienna, Austria $u Children's Cancer Research Institute, Medical University of Vienna, Vienna, Austria $1 https://orcid.org/0000000151075123
- 245 10
- $a Haematopoietic stem cell transplantation after reduced intensity conditioning in children and adolescents with chronic myeloid leukaemia: A prospective multicentre trial of the I-BFM Study Group / $c H. Pichler, P. Sedlacek, R. Meisel, R. Beier, M. Faraci, K. Kalwak, M. Ifversen, I. Müller, J. Stein, K. Vettenranta, G. Kropshofer, A. Kolenova, S. Karlhuber, E. Glogova, U. Poetschger, C. Peters, M. Suttorp, S. Matthes-Leodolter, A. Balduzzi
- 520 9_
- $a This prospective multicentre trial evaluated the safety and the efficacy of a thiotepa/melphalan-based reduced intensity conditioning (RIC) haematopoietic stem cell transplantation (HSCT) in children and adolescents with chronic myeloid leukaemia (CML) in chronic phase (CP). Thirty-two patients were transplanted from matched siblings or matched unrelated donors. In 22 patients, HSCT was performed due to insufficient molecular response or loss of response to first- or second-generation tyrosine kinase inhibitor (TKI), with pretransplant BCR::ABL1 transcripts ranging between 0.001% and 33%. The protocol included a BCR::ABL1-guided intervention with TKI retreatment in the first year and donor lymphocyte infusions (DLI) in the second-year post-transplant. All patients engrafted. The 1-year transplant-related mortality was 3% (confidence interval [CI]: 0%-6%). After a median follow-up of 6.3 years, 5-year overall survival and event-free survival are 97% (CI: 93%-100%) and 91% (CI: 79%-100%) respectively. The current 5-year leukaemia-free survival with BCR::ABL1 <0.01% is 97% (CI: 88%-100%) and the current TKI- and DLI-free survival is 95% (CI: 85%-100%). The incidence of chronic graft-versus-host disease (GvHD) was 32%, being severe in four patients (13%). At last follow-up, 31 patients are GvHD-free and have stopped immunosuppression. RIC HSCT following pretreatment with TKI is feasible and effective in children and adolescents with CP-CML with an excellent disease-free and TKI-free survival.
- 650 _2
- $a lidé $7 D006801
- 650 12
- $a transplantace hematopoetických kmenových buněk $x metody $7 D018380
- 650 _2
- $a mladiství $7 D000293
- 650 _2
- $a dítě $7 D002648
- 650 12
- $a příprava pacienta k transplantaci $x metody $7 D019172
- 650 12
- $a chronická myeloidní leukemie $x terapie $x mortalita $7 D015464
- 650 _2
- $a mužské pohlaví $7 D008297
- 650 _2
- $a ženské pohlaví $7 D005260
- 650 _2
- $a předškolní dítě $7 D002675
- 650 _2
- $a prospektivní studie $7 D011446
- 650 _2
- $a nemoc štěpu proti hostiteli $x etiologie $x prevence a kontrola $7 D006086
- 650 _2
- $a výsledek terapie $7 D016896
- 650 _2
- $a inhibitory proteinkinas $x terapeutické užití $7 D047428
- 655 _2
- $a časopisecké články $7 D016428
- 655 _2
- $a multicentrická studie $7 D016448
- 655 _2
- $a klinické zkoušky $7 D016430
- 700 1_
- $a Sedlacek, Petr $u Department of Paediatric Haematology and Oncology, University Hospital Motol, Charles University, Prague, Czech Republic
- 700 1_
- $a Meisel, Roland $u Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology and Clinical Immunology, Medical Faculty, Heinrich-Heine-University, Duesseldorf, Germany
- 700 1_
- $a Beier, Rita $u Department of Pediatric Hematology and Oncology, Hannover Medical School, Hannover, Germany
- 700 1_
- $a Faraci, Maura $u Hematopoietic Stem Cell Transplantation Unit, Department of Hematology and Oncology, IRCCS Institute G. Gaslini, Genoa, Italy $1 https://orcid.org/000000015715501X
- 700 1_
- $a Kalwak, Krzysztof $u Clinical Department of Paediatric Bone Marrow Transplantation, Oncology and Haematology, Wrocław Medical University, Wrocław, Poland
- 700 1_
- $a Ifversen, Marianne $u Department of Paediatrics and Adolescent Medicine, Copenhagen University Hospital Rigshospitalet, Copenhagen, Denmark $1 https://orcid.org/0000000225581935
- 700 1_
- $a Müller, Ingo $u Division of Pediatric Stem Cell Transplantation and Immunology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany
- 700 1_
- $a Stein, Jerry $u Department of Hematology-Oncology, Schneider Children's Medical Center of Israel, Petah Tikva, Israel
- 700 1_
- $a Vettenranta, Kim $u Paediatric Haemato-Oncology, University of Helsinki, Helsinki, Finland
- 700 1_
- $a Kropshofer, Gabriele $u Department of Pediatrics, Medical University of Innsbruck, Innsbruck, Austria
- 700 1_
- $a Kolenova, Alexandra $u Department of Pediatric Hematology and Oncology, Faculty of Medicine, Children's Haematology and Oncology Clinic, Comenius University in Bratislava, Bratislava, Slovakia
- 700 1_
- $a Karlhuber, Susanne $u Children's Cancer Research Institute, Medical University of Vienna, Vienna, Austria
- 700 1_
- $a Glogova, Evgenia $u Children's Cancer Research Institute, Medical University of Vienna, Vienna, Austria
- 700 1_
- $a Poetschger, Ulrike $u Department of Paediatric Haematology and Oncology, St. Anna Children's Hospital, Vienna, Austria $u Department of Paediatrics and Adolescent Medicine, Medical University of Vienna, Vienna, Austria $u Children's Cancer Research Institute, Medical University of Vienna, Vienna, Austria
- 700 1_
- $a Peters, Christina $u Department of Paediatric Haematology and Oncology, St. Anna Children's Hospital, Vienna, Austria $u Department of Paediatrics and Adolescent Medicine, Medical University of Vienna, Vienna, Austria $u Children's Cancer Research Institute, Medical University of Vienna, Vienna, Austria $1 https://orcid.org/0000000303698515
- 700 1_
- $a Suttorp, Meinolf $u Paediatric Haemato-Oncology, Faculty of Medicine Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany $1 https://orcid.org/0000000295228838
- 700 1_
- $a Matthes-Leodolter, Susanne $u Department of Paediatric Haematology and Oncology, St. Anna Children's Hospital, Vienna, Austria $u Department of Paediatrics and Adolescent Medicine, Medical University of Vienna, Vienna, Austria $u Children's Cancer Research Institute, Medical University of Vienna, Vienna, Austria
- 700 1_
- $a Balduzzi, Adriana $u Pediatric Transplant Unit, Fondazione IRCCS San Gerardo Dei Tintori, Monza, Italy $u School of Medicine and Surgery, University of Milano-Bicocca, Milan, Italy
- 773 0_
- $w MED00009374 $t British journal of haematology $x 1365-2141 $g Roč. 205, č. 1 (2024), s. 268-279
- 856 41
- $u https://pubmed.ncbi.nlm.nih.gov/38803040 $y Pubmed
- 910 __
- $a ABA008 $b sig $c sign $y - $z 0
- 990 __
- $a 20241015 $b ABA008
- 991 __
- $a 20241024111007 $b ABA008
- 999 __
- $a ok $b bmc $g 2202230 $s 1231870
- BAS __
- $a 3
- BAS __
- $a PreBMC-MEDLINE
- BMC __
- $a 2024 $b 205 $c 1 $d 268-279 $e 20240527 $i 1365-2141 $m British journal of haematology $n Br J Haematol $x MED00009374
- LZP __
- $a Pubmed-20241015
