Management of adults and children receiving CAR T-cell therapy: 2021 best practice recommendations of the European Society for Blood and Marrow Transplantation (EBMT) and the Joint Accreditation Committee of ISCT and EBMT (JACIE) and the European Haematology Association (EHA).

BACKGROUND: Several commercial and academic autologous chimeric antigen receptor T-cell (CAR-T) products targeting CD19 have been approved in Europe for relapsed/refractory B-cell acute lymphoblastic leukemia, high-grade B-cell lymphoma and mantle cell lymphoma. Products for other diseases such as multiple myeloma and follicular lymphoma are likely to be approved by the European Medicines Agency in the near future. DESIGN: The European Society for Blood and Marrow Transplantation (EBMT)-Joint Accreditation Committee of ISCT and EBMT (JACIE) and the European Haematology Association collaborated to draft best practice recommendations based on the current literature to support health care professionals in delivering consistent, high-quality care in this rapidly moving field. RESULTS: Thirty-six CAR-T experts (medical, nursing, pharmacy/laboratory) assembled to draft recommendations to cover all aspects of CAR-T patient care and supply chain management, from patient selection to long-term follow-up, post-authorisation safety surveillance and regulatory issues. CONCLUSIONS: We provide practical, clinically relevant recommendations on the use of these high-cost, logistically complex therapies for haematologists/oncologists, nurses and other stakeholders including pharmacists and health sector administrators involved in the delivery of CAR-T in the clinic.

Early Pre-B Lymphoblastic Transformation in A Patient with Refractory Anemia.

A 77 year-old male with refractory anemia receiving only supportive therapy presented with an acute transformation six years following diagnosis. The lineage of the blast cells could not be ascertained by morphological and cytochemical studies. Nevertheless, immunophenotypical studies, including monoclonal antibodies against myeloid, monocytic, erythroid, megakaryo-cytic and T and B lymphoid antigens, and analysis of immunoglobulin and T-cell receptors genes demonstrated the early pre-B nature of the blasts. Lymphoblastic transformation is extremely rare in myelodysplastic syndromes (MDS). The case presented here and other similar cases previously reported are consistent with the concept that MDS are clonal disorders arising from a pluripotent stem cell.