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. 2023 Jun;58(6):659-666.
doi: 10.1038/s41409-023-01924-6. Epub 2023 Mar 9.

Benchmarking of survival outcomes following Haematopoietic Stem Cell Transplantation (HSCT): an update of the ongoing project of the European Society for Blood and Marrow Transplantation (EBMT) and Joint Accreditation Committee of ISCT and EBMT (JACIE)

Affiliations

Benchmarking of survival outcomes following Haematopoietic Stem Cell Transplantation (HSCT): an update of the ongoing project of the European Society for Blood and Marrow Transplantation (EBMT) and Joint Accreditation Committee of ISCT and EBMT (JACIE)

Riccardo Saccardi et al. Bone Marrow Transplant. 2023 Jun.

Abstract

From 2016 EBMT and JACIE developed an international risk-adapted benchmarking program of haematopoietic stem cell transplant (HSCT) outcome to provide individual EBMT Centers with a means of quality-assuring the HSCT process and meeting FACT-JACIE accreditation requirements relating to 1-year survival outcomes. Informed by previous experience from Europe, North America and Australasia, the Clinical Outcomes Group (COG) established criteria for patient and Center selection, and a set of key clinical variables within a dedicated statistical model adapted to the capabilities of the EBMT Registry. The first phase of the project was launched in 2019 to test the acceptability of the benchmarking model through assessment of Centers' performance for 1-year data completeness and survival outcomes of autologous and allogeneic HSCT covering 2013-2016. A second phase was delivered in July 2021 covering 2015-2019 and including survival outcomes. Reports of individual Center performance were shared directly with local principal investigators and their responses were assimilated. The experience thus far has supported the feasibility, acceptability and reliability of the system as well as identifying its limitations. We provide a summary of experience and learning so far in this 'work in progress', as well as highlighting future challenges of delivering a modern, robust, data-complete, risk-adapted benchmarking program across new EBMT Registry systems.

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Conflict of interest statement

AMSB declares honoraria from Takeda, BMS/Celgene, MSD, Janssen, Amgen, Novartis, Gilead Kite, Sanofi, Roche, Alexion and Consultancy: Takeda, BMS/Celgene, Novartis, Janssen, Gilead, Sanofi, Speaker’s Bureau: Takeda and Research Support from Takeda, BMS/Celgene. GA declares paid consultancy project for Pfizer in 2021. MK declares honoraria for educational events from Jazz and Mallinkrodt, for advisory board membership from Jazz. RPdL declares Expert consultant/speaker: Alexion, Amgen, Gilead, Jazz, Keocyte, MSD, Novartis, Pfizer, Roche, Samsung & Therakos. Research grant: Alexion, Amgen, Jazz pharmaceutical, Novartis, Pfizer. Rémy Dulery declares personal fees from Takeda, Novartis, Incyte and Biotest and non-financial support from Gilead, (not related the submitted work). S-G, Fermindeclares honoraria and/or research support from Novartis, Kite/Gilead, Celgene/BMS, Pfizer, Incyte, Amgen, Takeda and Roche. RS declares honoraria and/or research support from Novartis, Kite/Gilead, Jazz Pharmaceuticals, Mallinkrodt, Sanofi and Cytiva. JS declares honoraria for educational events from Gilead and Janssen, for advisory board membership from Medac, and for trial IDMC membership from Kiadis Pharma.

Figures

Fig. 1
Fig. 1. Patients selection.
Selection process of patients to be analysed in the second phase of the Benchmarking Project.
Fig. 2
Fig. 2. Patient Follow up per country.
The bars report the percentage of reported 1-year follow-up for Centers in countries with at least 5 Centers, divided into allogeneic (left) and autologous (right) transplants in the second phase of the project (observation interval 2015–2019). On the right side of the plots, the number of Centers per country is reported. Centers with very good follow-up (>90% of transplanted patients) were classified as “Green”; Centers with a borderline completeness (80–90%) were classified as “Amber”; Centers with a Follow-up <80% were classified as “Red” and were not selected for the outcome analysis.
Fig. 3
Fig. 3. Autologous Transplants One-year mortality Funnel plot.
The Funnel plot shows 1-year mortality after an autologous transplant in Centers selected for a reliable follow-up in the 2015–2019 interval (second phase) comparing observed over expected mortality, adjusted for case mix and Center follow-up.
Fig. 4
Fig. 4. Allogeneic Transplants One-year mortality Funnel plot.
The Funnel plot shows 1-year mortality after an allogeneic transplant in Centers selected for a reliable follow-up in the 2015–2019 interval (second phase) comparing observed over expected mortality, adjusted for case mix and Center follow-up.
Fig. 5
Fig. 5. Data completeness.
In the second phase of the projects (2015–2019 transplants), the lack of case-mix data is reported in allogeneic transplants (a) and autologous (b), respectively. HCT-CI was calculated based on the raw data entered in ProMISe data field; blank fields were assumed as negative. The trend to data completeness in the two composite indexes HCT-CI (c) and DRI (d) in allogeneic HSCT in the period 2015–2019 is shown in the lower panels.
Fig. 6
Fig. 6. Reporting of molecular markers.
The bars represent the percentage of MED-A forms reporting 1 (blue bars), more than 1 (green bars) or no molecular markers (red bars), per year of transplant. The horizontal bars above indicate the time intervals analysed in the two phases of the Benchmarking project.

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