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Tisagenlecleucel (Kymriah™) and axicabtagene ciloleucel (Yescarta™) are the first representatives of a new class of gene therapies produced by ex-vivo genetic modification of human autologous T lymphocytes, now using viral vectors. In 2020, there are three independent CAR-T cell databases in France: DESCAR-T (database supported by LYSARC, GRAALL and the IFM), ProMISe (EBMT database) and ATIH (database of the Agence Technique de l'Information sur l'Hospitalisation). Only the EBMT database is common to France and the French-speaking countries that are members of the SFGM-TC. In 2019, a workshop was held to draft a manual for entering data specific to CAR-T cells in the EBMT ProMISe database. As a follow-up to this article, we present a medical report template containing all the data required to enter the data of patients treated with CAR-T in the EBMT registry, in the CRF of the DESCAR-T registry and in the ATIH registry. This document aims to improve the completeness and quality of the data while optimizing data entry time.

Resume Tisagenlecleucel (Kymriah™) et axicabtagene ciloleucel (Yescarta™) sont les premiers representants d’une nouvelle classe de therapies cellulaires produites par modification genetique ex vivo de lymphocytes T autologues humains, aujourd’hui a l’aide de vecteurs viraux. Dans le cadre d’une demarche globale visant a identifier des registres transnationaux sur des groupes de malades homogenes en termes de diagnostic ou d’approches therapeutiques, l’Agence Europeenne du Medicament (EMA) a emis une opinion favorable a l’endroit du registre existant de l’EBMT, comme outil susceptible d’etre utilise pour collecter de facon prospective les donnees d’efficacite et de toxicite a court et long terme survenant chez les patients traites en Europe avec des « CAR-T Cells » ; en raison de leur statut de « therapies geniques », le suivi clinique des « CAR-T Cells » doit etre organise au cours des 15 prochaines annees. L’utilisation d’un registre europeen a l’avantage de permettre de disposer d’une vision globale, transnationale et « transpathologies » et evitera la multiplication des registres de donnees. Pour repondre aux exigences de l’EMA, des laboratoires pharmaceutiques developpant des « CAR-T cells » et autres therapies cellulaires ou geniques, et des autres parties interessees, l’EBMT a cree un nouveau formulaire en ligne permettant de saisir les informations essentielles sur le medicament de therapie innovante administre, sur la pathologie traitee et sur le devenir du patient. Dans cet article, nous presentons les recommandations de la SFGM-TC pour aider les data manageurs a mieux saisir les donnees des patients traites par cellules CAR-T dans le registre de l’EBMT.

In an effort to standardize hematopoietic stem cell allograft procedures, the Francophone bone marrow transplantation and Cell Therapy Society (SFGM-TC) organized the 9th Allograft Harmonization Practice Workshop in Lille in September 2018. The purpose of these workshops is to propose a consensual attitude to the centers that wish it. In this workshop, we discuss how to capture the cytogenetic and molecular abnormalities of acute leukaemias, myelomas, myelodysplasias, myeloproliferative syndromes and myelodysplastic/myeloproliferative syndromes in the database common to all European transplant centers called ProMISe and managed by the European Society for Blood and Marrow Transplantation (EBMT). The complexity of cytogenetic and molecular data makes it difficult to enter data into the ProMISe registry. This workshop proposes a tool for input assistance, in tabular form by pathology. The main recommendation for the karyotype remains that of the complex karyotype that must be entered in "Full caryotype". Concerning the molecular anomalies, it is necessary to enter all the items proposed by ProMISe. In reviewing all the sheets proposed by ProMise, we note the absence of some relevant elements that can be added later.

International audience; Invasive aspergillosis (IA) occurs in up to 23% of allogeneic hematopoietic stem cell transplantation (HSCT) patients. Although transplant procedures have changed over time, more late cases of IA are being observed. The objective of this study was to identify the pre-and post-transplant factors of IA in a large cohort of HSCT patients mainly transplanted with reduced-intensity conditioning. This multicenter, case-control study was carried out using data collected between 2005 and 2010 by the Surveillance des Aspergilloses Invasives en France program (Institut Pasteur, Paris) and the European Society for Blood and Marrow Transplantation ProMISe registry. Four control subjects without IA were individually matched to each case based on the center, patient age, and year of the transplant. We identified 185 cases of probable and proven IA and 651 control subjects. The median date of IA after the transplant was 133 days, with 35 cases (19%) of early IA (before day 40), 33 cases (18%) of late IA (days 40 to 100), and 117 cases (63%) cases of very late IA (after day 100). In the multivariate analysis early IA was significantly associated with a lack of engraftment, whereas late and very late IA were significantly associated with more than grade II acute graft-versus-host disease (GVHD); very late IA was also significantly associated with relapse and secondary neutropenia. Two-thirds of IA cases occurred more than 100 days after HSCT with different risk factors from those occurring earlier. Prophylactic strategies should consider the specific risk factors for late and very late IA, especially GVHD, relapse after transplant, and secondary neutropenia.

The quality of the information provided in post-transplant follow-up is necessary to obtain a coherent and exploitable database. Since the beginning of 2017, three forms (Med-B-allograft) have been available: the first month (Day 0), Day 100 (second report) and an annual follow-up report. Recommendations for follow-up were addressed in the 2014 harmonization workshop, "Harmonization of Data Coding…". However, it is sometimes difficult to determine which data to specify in ProMISe for post-transplantation. The objective of this workshop was to clarify certain situations and/or items.

As part of the 7th Annual francophone workshop series on the harmonization of clinical practices in allogeneic stem cell transplantation held in Lille in September 2016, our workgroup discussed how transplant centers might follow a collective approach to coding data. This was done mainly by analyzing the study results found in the literature that do not provide clear answers. In addition, we discuss practical ways of coding for both donor and recipient HLA typing in the European bone marrow transplantation database called ProMISe which is managed by the European Society for Blood and Marrow Transplantation (EBMT).

Resume Les difficultes des etudes prospectives et retrospectives issues de la base des donnees europeenne (ProMISe), et des etudes propres a chaque centre sont dues a des donnees insuffisamment mises a jour, manquantes ou incoherentes. Si les donnees sont erronees, les resultats eux-memes seront errones. Lors des sixiemes ateliers d’harmonisation des pratiques, un groupe forme des data managers a travaille dans le but d’apporter une aide pour parvenir a une base de donnees « propre », avec un minimum d’erreurs a transmettre, et pour harmoniser la saisie des donnees. Le sujet de cet atelier etait le codage des diagnostics d’un certain nombre de maladies ainsi que l’etat hematologique a la greffe.