The meeting was attended by 119 virtual attendees, representing 34 countries.
The programme focused on current advances and future developments in HCT, gene therapies and novel cellular therapies across a range of indications, including CAR-T cell therapies for autoimmune diseases. The meeting featured both clinical and scientific sessions, fostering interdisciplinary exchange and cross-fertilization between haematology, paediatrics and autoimmune research. The five best selected abstracts were presented during the Best Abstracts session.
The following summarises the oral presentations made during these sessions from the ADWP and PDWP's perspectives.
Special thanks to the editors for preparing this report:
Rosamaria Nitti, ADWP Task Force Representative, Italy
Tobias Alexander, ADWP Chair, Germany
Virginia Meda Spacamela, clinician-scientist, Division of HCT and Cell Therapy, University Children’s Hospital Zurich, Switzerland
Krzysztof Kałwak, PDWP Chair, Poland
The ADWP view by Tobias Alexander and Rosamaria Nitti
“From Immunology and Autoimmunity to Haematology and back“, building upon solid foundations and moving towards the future with the latest evidences.
In the first session, an overview of the current indications and recommendations for autologous HSCT and CAR-T therapy in autoimmune diseases was provided. Elisa Roldan (Sheffield, UK) highlighted systemic sclerosis and multiple sclerosis as established standard indications for auto-HSCT, with cornerstones on patient selection as outlined in the recent EBMT recommendations (Alexander T, BMT 2025). Of note, ADs are the only growing indications to autologous HSCT in the EBMT registry. Raffaella Greco (Milan, IT) provided an update on the EBMT clinical practice recommendations for the use of CAR-T cell therapy in ADs, focusing on patient selection and supportive care as discussed in a recently published paper (Greco R, eBioMedicine 2024), currently being updated. The picture is different in the context of pediatric patients treated with HSCT, as presented by Julia Silva (London, UK), who demonstrated a gradual shift from auto- to allo-HSCT among cases reported to the EBMT registry, with hematologic indications and juvenile systemic arthritis representing the most common indications.
The session on rheumatologic and neurological autoimmune diseases focused on clinical evidence, translational aspects, and novel developments in the field. Johanna Richter (Hamburg, DE) provided an update on CD19- and BCMA-directed CAR-T cell therapies in multiple sclerosis based on the few patients treated to date. This increasing experience demonstrates feasibility and a favorable safety profile, although clinical outcome data remain limited. Doron Rimar (Haifa, IL) presented emerging evidence on predictive biomarkers, including the neutrophil-to-lymphocyte ratio and inflammatory versus fibrotic immune signatures, for outcomes following auto-HSCT in systemic sclerosis. He also introduced the concept of pre-HSCT bridging therapy with rituximab and MMF in severely affected patients. Finally, Elise Siegert (Berlin, DE) presented emerging evidence for the use of bispecific T-cell engagers in systemic sclerosis, with only a few, mostly single-center studies currently available, utilizing T-cell engagers directed against CD19 (blinatumomab) and BCMA (teclistamab). Initial clinical data are promising, with some patients achieving treatment-free remission; however, further studies are required to evaluate the risk–benefit profile before broader clinical implementation can be considered.
A message of great hope in the context of fertility after CAR-T cell therapy emerged with the first reports of successful pregnancies in women treated with CAR-Ts, both in the presentations by Giorgio Orofino (Milan, IT), who presented an EBMT study, and by Melanie Hagen (Erlangen, DE), who showed data on women treated for ADs, both reporting spontaneous pregnancies with healthy babies.
An emerging and recurrent question in the field of HSCT and cellular therapies for ADs is the challenge of choice or sequencing of therapies: as speakers agreed, today, it is wise to prefer the therapy with the strongest evidence for which the patient is fit, but as evidence grows, this approach will necessary evolve, and we look forward to face this challenge.
The PDWP view by Krzysztof Kałwak and Virginia Meda Spaccamela
The Joint Meeting of the EBMT ADWP, IEWP and PDWP, titled 'From Immunology and Autoimmunity to Haematology and Back', took place online on 22nd and on 23rd May 2026. The meeting focused on different cell therapies, such as CAR-T and gene therapy, in the treatment of immunological and autoimmune diseases. Specialists from various adult and pediatric disciplines came together to discuss the various elements involved in new approaches to treating these diseases. All possible aspects were covered, including official indications and guidelines, appropriate conditioning regimens, management of complications, fertility issues and regulatory aspects. Attending this joint meeting enabled participants to join a large community dedicated to developing and improving cell therapies for diseases of the immune system.
The first session, chaired by Mathieu Puyade and Krzysztof Kałwak, gave an overview on the indications of autologous/allogenic HSCT and CAR-T in adult and pediatric patients with autoimmune disorders.
Elisa Roldan, secretary of ADWP, spoke about the indications for autologous HSCT in adult patients with autoimmune diseases such as Multiple Sclerosis, Systemic Sclerosis, Systemic Lupus Erythematosus (SLE) and Chron’s disease. She pointed out the importance of a multidisciplinary approach to these patients, such as the involvement of neurologists, cardiologists and nephrologists in the preparation and management of these patients before and after autologous HSCT. She concluded her talk by mentioning the importance of randomized studies, which are at the moment ongoing, to improve patient selection, optimize conditioning regimes and graft manipulation.
Raffaella Greco started the second talk of the session by presenting the new EBMT Guidelines of CAR-T for Autoimmune Diseases in adult patients, including the indications, the patient screening, the different types of CAR-T products, the monitoring of B cell subsets and the management of toxicities. A very interesting overview of the last published papers about CAR-T for autoimmune diseases was also presented, showing very promising results with depletion of pathological antibody titers in blood and in CSF after CAR-T treatment, element that persist also after B cell immune reconstitution takes place. Like pointed out from Elisa Roldan for autologous HSCT also for CAR-T cells a multidisciplinary approach before and after the treatment is necessary. A mandatory point before starting a CAR-T treatment is the absence of any active infections, a disease assessment and a cardiological and nephrological evaluation. A pregnancy must be excluded and a fertility counselling is also recommended. Raffella Greco concluded her talk by mentioning the importance of register data for the follow-up of patients that underwent CAR-T therapy.
As last presentation of the first session Juliana Silva gave an overview of the indications for allogenic HCT in pediatric patients with autoimmune disorders. While modern targeted therapies have significantly improved overall outcomes, a small subset of patients remains highly refractory to standard treatments or suffers from severe drug toxicity. Allogenic HCT can be a valid therapeutic option for different reasons, but especially due to the Graft-versus-Autoimmunity (GVA) Effect, an unique, allogeneic-specific phenomenon that actively combats the patient's autoimmune activity.
In the second session a broad overview of CAR-T therapy in adult and pediatric settings was presented.
Melanie Hagen started her talk by mentioning the five years history of CAR-T therapy in autoimmune diseases, which started by treating an adult patient with SLE in March 2021. Currently there are 40 different ongoing trials that are including around 300 treated patients. An important point that Melanie Hagen underlined is the patient eligibility for CAR-T therapy after failing two conventional lines of treatment. Patients showed the highest efficacy when treated at the stage of early progression. preventing chronic organ damage. The Phase 1/2a CASTLE study was also presented, in which the safety and the efficacy of Zorpo-cel in patients with different clinical indications were evaluated. This study showed very promising results with less severe Cytokine Release Syndrome, very rare neurotoxicity and achievement of remission in SLE at 24 weeks after the treatment, pointing CAR-T therapy as one of the main treatments for patients with autoimmune diseases.
Marco Becilli continued the session by addressing CAR-T therapy in pediatric patients. In 2023 the first pediatric patients with SLE, systemic sclerosis and juvenile dermatomyositis were treated in Roma (IT) and in Erlangen (DE). All patients had highly active diseases affecting vital organs, such as severe lupus nephritis (one patient was on hemodialysis), interstitial lung disease, pulmonary hypertension and alveolar hemorrhage. Although patients had highly active diseases prior CAR-T therapy, the treatment was well tolerated and only mild toxicity was reported in some patients, allowing to conclude that CAR-T therapy is a feasible and well tolerated treatment with patient substantial benefits.
In the session about gene therapy a broad overview of this treatment for Inborn Errors of Immunity (IEIs) was presented by Claire Booth, pointing out its benefits and comparing it to allogenic HCST. Different trials were presented, such as the one for p47phox-CGD patients (London, UK) and the one for patients affected by Wiskott Aldrich Syndrome (name of the trial Waskyra), which was recently opened by the Telethon Foundation in Milano (IT). In particular the results of the multi-center gene therapy trial for patients with X-linked SCID were presented, showing a 100% patient overall survival. 16/17 patients presented a stable and good amount of T-cells one year after the treatment. Also the vector copies were stable in the different immune cell subsets, showing promising results for a treatment that should be more accessible in the future.
Like Claire Booth did for IEIs, Maria Ester Bernardo presented the different metabolic diseases for which gene therapy can be an option, such as the lysosomal storage disorders (LSDs). The results of the clinical trial for Metachromatic Leukodystrophy (MLD) were presented, showing a stable bone marrow engraftment (median follow up: 7 years), normalization of enzyme levels in blood and in the cerebrospinal fluid (CSF) and a preservation of motor and cognitive functions compared to the disease's natural decline. Similar results were also reported in the clinical trial for Mucopolysaccharidosis Type 1 (MPS1 - Hurler Syndrome).
As closing talk of the first day Alessanndro Aiuti pointed out the importance of different administrative aspects of gene therapy trials such as regulation, sustainability and accessibility. At the moment the legislation landscape is complicated due to the multiple overlapping EU legislations, situation that should improve once the new European pharmaceutical legislation of 2026 will be from the different EU countries fully adopted. The centralized approval by the European Medicine Agency does not guarantee patient access, since companies must still negotiate pricing and reimbursement on a country-by-country basis. Several approved gene therapies have been withdrawn from the European market entirely due to reimbursement barriers, high costs, and commercial unviability. It is also important to mention that for rare diseases it is unstainable to have treatment centers in all 27 European centers, raising the importance to localize this type of treatments in few highly specialized centers in Europe.
On the second day of the congress, after an exciting session about different type of treatments for rheumatological and neurological autoimmune diseases, the fifth session, chaired by Fulvio Porta, focusing on optimizing conditioning regimes in pediatric settings, was presented.
Adrian Lankester provided an overview of the history and current state of serotherapy in the context of bone marrow transplantation. An important study of Leyden and Utrecht performed a pharmacokinetic and pharmacodynamic analysis in a pediatric cohort, showing that different levels of Grafalon/ATG exposure have an impact on developing GvHD, the speed of immune reconstitution and the overall survival. Based on these significant findings the Utrecht team developed a dose algorithm based on body weight and lymphocyte count. Patients treated according to this algorithm showed a better immune reconstitution and overall survival. Also in the ALL-SCT Forum Study the exposure to Grafalon/ATG and Thymoglobulin/ATLG was analyzed, showing similar results. The exposure to ATG/ATLG showed a correlation to the incidence of GvHD and overall survival. A prolonged ATG exposure, particularly beyond 20 days after transplantion, showed an association with delayed immune recovery and an increased risk of CMV and EBV reactivation, but no negative impact on the risk to develop a relapse. Prolonged exposure to Alemtuzumab was also associated with a delayed immune reconstitution. A PK study of Alemtuzumab is at the moment ongoing.
Following the serotherapy talk Fulvio Porta gave a presentation on inborn error immunodeficiencies and immune dysregulation diseases, in particularly showing the different type of conditioning regimes and pointing out the growing importance of haploidentical transplants. The results of a Brescia study of patients with primary immunodeficiencies undergoing haploidentical transplantation were reported, showing very good overall survival. A complicated case study of a Brescia patient with LRBA deficiency was also presented. The patient was completely cured after being transplanted with based Treosulfan conditioning regime followed by MUD. Following this general overview, radiotherapy was addressed as a potential element of pediatric conditioning. Data of studies of patients with CGD, HLH, DOCK 8 and WAS that underwent radiotherapy showed an increase in late graft failure, but no relevant side effects and no increase of tumour cases, with a overall survival of 90%. Also radiotherapy as possible part of conditioning regime in patients with dysregulation of immune system was appointed. The Seattle experience and other recent trials of patients with non-malignant diseases, that were haploidentical transplanted following a condition regime with Cyclofosfamide, Fludarabine and TBI (200 or 400 cGy), were presented, showing good results in terms of overall survival and low percentage of transplantation related complications.
An active and interesting debate between by Krzysztof Kałwak, representing the Treosulfan party and Adrian Lankester, representing the Busulfan party took place and closed the fifth session. The pros and cons of these two conditioning regimes were compared, showing that each conditioning regime has its advantages and disadvantages. Treosulfan has a lower transplant-related mortality and a lower incidence of endothelial toxicity and veno-occlusive disease (VOD). Patients that underwent Treosulfan conditioning presented a better chronic GvHD-free survival and less gonadal toxicity. Due to its less toxicity Treosulfan is indicated for young children and for patients with high risk of transplant-related mortality such as patients undergoing a second transplantation. On the other side patients with non-malignant diseases undergoing a Treosulfan based conditioning have a higher risk of secondary graft failure and mixed chimerism, like it was reported in the study of Sykora of 2023. The Australian study by Rosser and Shaw was also mentioned, in which the addition of Thiotepa to Fludarabin-Treosulfan conditioning regime resulted in a better disease-free survival and a better chimerism. The use of Busulfan as conditioning regime is older compared to the one of Treosulfan and its pharmacokinetic guided dosing became mandatory to reduce transplant-related toxicity and gonadal toxicity and the risk of relapse in patients with ALL. The answer to the question “which conditioning regime is the best one between Busulfan and Treosulfan” remains unanswered, leading each center to decide depending on patient history and clinical conditions before transplantation.
Different other exciting topics were treated during the second day of the congress, such as complications after allogenic HSCT and CAR-T therapy, HLH and fertility issues. To conclude the congress an overview of the therapy access in lower/middle income countries was presented, pointing out the importance to improve the accessibility of these treatments worldwide.