Pre-transplantation Risks and Transplant-Techniques in Haematopoietic Stem Cell Transplantation for Acute Leukaemia
Background: The role of conditioning intensity and stem cell source on modifying pre-transplantation risk in allogeneic haematopoietic stem cell transplantation (HSCT) is a matter of debate, but crucial when benchmarking centres. Methods: This Retrospective, multicenter exploratory-validation analys...
| Main Authors: | , , , , , , , , , , , , , , |
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| Format: | Article |
| Language: | English |
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Elsevier
2019-10-01
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| Series: | EClinicalMedicine |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S2589537019301397 |
| _version_ | 1818310667492392960 |
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| author | Alois Gratwohl Rafael Duarte John A. Snowden Anja van Biezen Helen Baldomero Jane Apperley Jan Cornelissen Hildegard T. Greinix Eoin Mc Grath Mohamad Mohty Nicolaus Kroeger Arnon Nagler Dietger Niederwieser Hein Putter Ronald Brand |
| author_facet | Alois Gratwohl Rafael Duarte John A. Snowden Anja van Biezen Helen Baldomero Jane Apperley Jan Cornelissen Hildegard T. Greinix Eoin Mc Grath Mohamad Mohty Nicolaus Kroeger Arnon Nagler Dietger Niederwieser Hein Putter Ronald Brand |
| author_sort | Alois Gratwohl |
| collection | DOAJ |
| description | Background: The role of conditioning intensity and stem cell source on modifying pre-transplantation risk in allogeneic haematopoietic stem cell transplantation (HSCT) is a matter of debate, but crucial when benchmarking centres. Methods: This Retrospective, multicenter exploratory-validation analysis of 9103 patients, (55.5% male, median age 50 years; 1–75 years range) with an allogeneic HSCT between 2010 and 2016 from a matched sibling (N = 8641; 95%) or matched unrelated donor (N = 462; 5%) for acute myeloid (N = 6432; 71%) or acute lymphoblastic (N = 2671; 29%) leukaemia in first complete remission, and reported by 240 centres in 30 countries to the benchmark database of the European Society for Blood and Marrow Transplantation (EBMT) searched for factors associated with use of transplant techniques (standard N = 6375;70% or reduced intensity conditioning N = 2728;30%, respectively bone marrow N = 1945;21% or peripheral blood N = 7158;79% as stem cell source), and their impact on outcome. Findings: Treatment groups differed significantly from baseline population (p < 0.001), and within groups regarding patient-, disease-, donor-, and centre-related pre-transplantation risk factors (p < 0.001); choice of technique did depend on pre-transplantation risk factors and centre (p < 0.001). Probability of overall survival at 5 years decreased systematically and significantly with increasing pre-transplantation risk score (score 2 vs 0/1 HR: 1·2, 95% c.i. [1·1–1·.3], p = 0.002; score 3 vs 0/1 HR: 1·5, 95% c.i. [1·3–1·7], p < 0.001; score 4/5/6 vs 0/1 HR: 1·9, 95% c.i. [1·6–2·2], p < 0.001) with no significant differences between treatment groups (likelihood ratio test on interaction: p = 0.40). Overall survival was significantly associated with selection steps and completeness of information (p < 0.001). Interpretation: Patients' pre-transplantation risk factors determine survival, independent of transplant techniques. Transplant techniques should be regarded as centre policy, not stratification factor in benchmarking. Selection criteria and completeness of data bias outcome. Outcomes may be improved more effectively through better identifying pre-transplantation factors as opposed to refinement of transplant techniques. Funding: The study was funded by EBMT. Keywords: Haematopoietic stem cell transplantation, Acute leukaemia, Risk factors, Conditioning, Stem cell source, Benchmarking |
| first_indexed | 2024-12-13T07:49:42Z |
| format | Article |
| id | doaj.art-4b3ac3890f7f46f5a47f20c03e58cd04 |
| institution | Directory Open Access Journal |
| issn | 2589-5370 |
| language | English |
| last_indexed | 2024-12-13T07:49:42Z |
| publishDate | 2019-10-01 |
| publisher | Elsevier |
| record_format | Article |
| series | EClinicalMedicine |
| spelling | doaj.art-4b3ac3890f7f46f5a47f20c03e58cd042022-12-21T23:54:43ZengElsevierEClinicalMedicine2589-53702019-10-01153341Pre-transplantation Risks and Transplant-Techniques in Haematopoietic Stem Cell Transplantation for Acute LeukaemiaAlois Gratwohl0Rafael Duarte1John A. Snowden2Anja van Biezen3Helen Baldomero4Jane Apperley5Jan Cornelissen6Hildegard T. Greinix7Eoin Mc Grath8Mohamad Mohty9Nicolaus Kroeger10Arnon Nagler11Dietger Niederwieser12Hein Putter13Ronald Brand14Hematology, Medical Faculty, University of Basel, Basel, Switzerland; Corresponding author at: Hematology, Medical Faculty, University of Basel, Dittingerstrasse 4, CH-4053 Basel, Switzerland.Department of Hematology, University Hospital Puerta de Hierro Majadahonda, Madrid, SpainDepartment of Haematology, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, United KingdomDepartment of Biomedical Data Sciences, Section Medical Statistics, Leiden University Medical Centre, Leiden, the NetherlandsEBMT activity survey office, University Hospital, Basel, SwitzerlandCentre for Haematology, Hammersmith Hospital, Imperial College London, United KingdomHematology, Erasmus University Medical Center, Rotterdam, the NetherlandsDivision of Hematology, Medical University of Graz, Graz, AustriaJACIE Accreditation Office, Barcelona, SpainHematology, Hôpital St. Antoine, Paris, France; Sorbonne University, Paris, FranceDepartment of Stem Cell Transplantation, University Hospital Eppendorf, Hamburg, GermanyChaim Sheba Medical Center, Tel-Hashomer, IsraelHematology and Oncology, University Hospital, Leipzig, GermanyDepartment of Biomedical Data Sciences, Section Medical Statistics, Leiden University Medical Centre, Leiden, the NetherlandsDepartment of Biomedical Data Sciences, Section Medical Statistics, Leiden University Medical Centre, Leiden, the NetherlandsBackground: The role of conditioning intensity and stem cell source on modifying pre-transplantation risk in allogeneic haematopoietic stem cell transplantation (HSCT) is a matter of debate, but crucial when benchmarking centres. Methods: This Retrospective, multicenter exploratory-validation analysis of 9103 patients, (55.5% male, median age 50 years; 1–75 years range) with an allogeneic HSCT between 2010 and 2016 from a matched sibling (N = 8641; 95%) or matched unrelated donor (N = 462; 5%) for acute myeloid (N = 6432; 71%) or acute lymphoblastic (N = 2671; 29%) leukaemia in first complete remission, and reported by 240 centres in 30 countries to the benchmark database of the European Society for Blood and Marrow Transplantation (EBMT) searched for factors associated with use of transplant techniques (standard N = 6375;70% or reduced intensity conditioning N = 2728;30%, respectively bone marrow N = 1945;21% or peripheral blood N = 7158;79% as stem cell source), and their impact on outcome. Findings: Treatment groups differed significantly from baseline population (p < 0.001), and within groups regarding patient-, disease-, donor-, and centre-related pre-transplantation risk factors (p < 0.001); choice of technique did depend on pre-transplantation risk factors and centre (p < 0.001). Probability of overall survival at 5 years decreased systematically and significantly with increasing pre-transplantation risk score (score 2 vs 0/1 HR: 1·2, 95% c.i. [1·1–1·.3], p = 0.002; score 3 vs 0/1 HR: 1·5, 95% c.i. [1·3–1·7], p < 0.001; score 4/5/6 vs 0/1 HR: 1·9, 95% c.i. [1·6–2·2], p < 0.001) with no significant differences between treatment groups (likelihood ratio test on interaction: p = 0.40). Overall survival was significantly associated with selection steps and completeness of information (p < 0.001). Interpretation: Patients' pre-transplantation risk factors determine survival, independent of transplant techniques. Transplant techniques should be regarded as centre policy, not stratification factor in benchmarking. Selection criteria and completeness of data bias outcome. Outcomes may be improved more effectively through better identifying pre-transplantation factors as opposed to refinement of transplant techniques. Funding: The study was funded by EBMT. Keywords: Haematopoietic stem cell transplantation, Acute leukaemia, Risk factors, Conditioning, Stem cell source, Benchmarkinghttp://www.sciencedirect.com/science/article/pii/S2589537019301397 |
| spellingShingle | Alois Gratwohl Rafael Duarte John A. Snowden Anja van Biezen Helen Baldomero Jane Apperley Jan Cornelissen Hildegard T. Greinix Eoin Mc Grath Mohamad Mohty Nicolaus Kroeger Arnon Nagler Dietger Niederwieser Hein Putter Ronald Brand Pre-transplantation Risks and Transplant-Techniques in Haematopoietic Stem Cell Transplantation for Acute Leukaemia EClinicalMedicine |
| title | Pre-transplantation Risks and Transplant-Techniques in Haematopoietic Stem Cell Transplantation for Acute Leukaemia |
| title_full | Pre-transplantation Risks and Transplant-Techniques in Haematopoietic Stem Cell Transplantation for Acute Leukaemia |
| title_fullStr | Pre-transplantation Risks and Transplant-Techniques in Haematopoietic Stem Cell Transplantation for Acute Leukaemia |
| title_full_unstemmed | Pre-transplantation Risks and Transplant-Techniques in Haematopoietic Stem Cell Transplantation for Acute Leukaemia |
| title_short | Pre-transplantation Risks and Transplant-Techniques in Haematopoietic Stem Cell Transplantation for Acute Leukaemia |
| title_sort | pre transplantation risks and transplant techniques in haematopoietic stem cell transplantation for acute leukaemia |
| url | http://www.sciencedirect.com/science/article/pii/S2589537019301397 |
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