The EBMT-ADWP and the CIBMTR

Abstract

Hematopoietic stem cell transplantation (HSCT) has evolved over the last 25 years as a specific treatment of patients with severe neurologic autoimmune diseases (ADs), through eradication of the pathologic, immunologic memory, and profound immune "resetting." HSCT for ADs is recently facing a unique developmental phase across transplant centers. Data from patients undergoing HSCT and cellular therapies have been captured through the established major transplant registries, such as the European Society for Blood and Marrow Transplantation (EBMT) and the Center for International Blood and Marrow Transplant Research (CIBMTR). The EBMT Autoimmune Diseases Working Party (ADWP) is central to bringing together HSCT and disease-specialist communities. The AD section of the EBMT registry is the largest database of its kind worldwide, reporting more than 3700 transplants. Multiple sclerosis (MS) covers approximately 50% of transplants in AD, HSCT being an integral and standard-of-care part of the treatment algorithm. In the Americas, at least a subset of HSCT is reported to the CIBMTR, as reporting is voluntary. A total of 1400 recipients of autologous HSCT were reported and 1030 were performed for the treatment of neurologic conditions. MS accounts for 96% of all diagnoses among neurologic indications for HSCT. Although the activity of HSCT for MS is low in the United States in relation to its prevalence, the number of transplants has increased in recent years. In contrast, Mexico has reported a sharp increase in the number of these transplants. This chapter provides an overview of the EBMT and CIBMTR registries, then offers the current status and publication outputs in relation to neurologic AD.

Keywords: Center for International Blood and Marrow Transplant Research; European Society for Blood and Marrow Transplantation; Hematopoietic stem cell transplantation; Neurologic autoimmune diseases.

Fig. 19.1.

Autologous HSCT distribution in neurologic AD, per 1995–2021: EBMT-ADWP registry data. Abbreviations: AD, autoimmune diseases; ADWP, autoimmune diseases working party; EBMT, European Society for Blood and Marrow Transplantation; HSCT, hematopoietic stem cell transplantation; MS, multiple sclerosis; CIDP, chronic inflammatory demyelinating polyradiculoneuropathy; NMO, neuromyelitis optica.

Fig. 19.2.

Autologous HSCT distribution (1995–2021) in neurologic AD, per country (only countries with ≥10 autologous HSCTs): EBMT-ADWP registry data. Abbreviations: AD, autoimmune diseases; ADWP, Autoimmune Diseases Working Party; EBMT, European Society for Blood and Marrow Transplantation; HSCT, hematopoietic stem cell transplantation; MS, multiple sclerosis; CIDP, chronic inflammatory demyelinating polyradiculoneuropathy; NMO, neuromyelitis optica.

Fig. 19.3.

Autologous HSCT distribution, according to 1995–2021, in RR-MS versus other MS forms: EBMT-ADWP registry data. Abbreviations: ADWP, autoimmune diseases working party; EBMT, European Society for Blood and Marrow Transplantation; HSCT, hematopoietic stem cell transplantation; MS, multiple sclerosis; RR, relapsing-remitting.

Fig. 19.4.

Epoch analysis of trends in nonrelapse mortality (NRM) from 2000 to 2004, 2005 to 2009, 2010 to 2014, and 2015 to 2020: EBMT-ADWP registry data (only MS patients). Abbreviations: AD, autoimmune diseases; ADWP, autoimmune diseases working party; EBMT, European Society for Blood and Marrow Transplantation; MS, multiple sclerosis.

Fig. 19.5.

Autologous HSCT distribution in neurologic AD, per 1995–2020 for all reporting countries (US, Mexico, Canada, and Brazil): CIBMTR registry data. Abbreviations: AD, autoimmune diseases; auto-HSCT, autologous HSCT; CIBMTR, Center for International Blood and Marrow Transplant Research; HSCT, hematopoietic stem cell transplantation; US, United States.

Fig. 19.6.

Autologous HSCT distribution in neurologic AD, per 1995–2020, US only: CIBMTR registry data. Abbreviations: AD, autoimmune diseases; auto-HSCT, autologous HSCT; CIBMTR, Center for International Blood and Marrow Transplant Research; HSCT, hematopoietic stem cell transplantation; US, United States.