Summary reports by Elisabetta Xue, Hematology and Bone Marrow Transplantation Unit, Vita-Salute San Raffaele University, Milan, Italy, and by Ariadna Domènech, Hematology and Bone Marrow Transplantation Unit, Hospital Clinic, Barcelona, Spain.
For the second consecutive year, the ADWP Educational Meeting was held on a virtual platform due to the ongoing Covid-19 pandemic.
The meeting was again very successful, attracting over 140 attendees from 28 countries, including haematologists, disease specialists, nurses, and trainees, and covered a wide range of topics, in relation to the pathophysiology and therapy of autoimmune diseases (ADs), including current and future indications, immunological mechanisms and novel targets.
HSCT activity - Update from the EBMT registry - Raffaella Greco
The meeting started with a comprehensive overview of the EBMT registry for ADs by Raffaella Greco. The registry now includes more than 3600 hematopoietic stem cell transplants (HSCT) performed for Ads. Remarkably, in some countries, the number of HSCT for these indications have increased in the last decade, mainly for autologous HSCT in multiple sclerosis and systemic sclerosis, now considered standard EBMT indications.
Infections and late effects of HSCT for AD. Chairs: Tobias Alexander & Zinaida Peric
Covid-19, overview and vaccination - Per Ljungman
The session started with Per Ljungman, discussing management on SarsCov2 in HSCT patients; he updated on several under-evaluation vaccines, and on recent EBMT recommendations regarding Covid-19 treatments in HSCT patients. He reported data from recent studies, outlying how age, performance status and immunodeficiency scoring index are still the main risk factors for Covid-19 related mortality.
CMV/EBV reactivation - Majid Kazmi
Subsequently, the focus moved on EBV and CMV reactivation; Majid Kazmi gave an overview on potential challenges in prompt diagnosis in patients transplanted for ADs, given the different clinical presentations these viral reactivations might have compared to other patients, and stressed the need of close viremia monitoring as per allogeneic transplants.
Late effects, retrospective analysis from ADWP - Kirill Kirgizov
Secondary Autoimmune Diseases after HSCT in MS - Joachim Burman
Afterwards, the session focused on late complications in patients with ADs performing HSCT. Kirill Kirgizov highlighted the need of more specific guidelines for long-term monitoring and management in these settings, whereas Joachim Burman reported on post-HSCT secondary ADs, overviewing hypotheses of their pathogenesis and potential treatments.
HSCT in MS and rare neurologic autoimmune diseases. Chairs: Riccardo Saccardi & John Snowden
HSCT for Multiple sclerosis: 507 real world patients - Richard Burt
Richard Burt reported detailed results on 507 patients with MS undergoing non-myeloablative HSCT, showing very low transplant-related mortality and low rate of neutropenic infections. Careful patient selection has been recommended, given the heterogeneous outcomes in different MS subtypes.
The 1,000th transplant for multiple sclerosis and other autoimmune disorders at the HSCT-México program - Iván Murrieta-Álvarez
Ivan Murrieta-Alvarez described outcomes of MS undergoing HSCT in Latin America, investigating non-frozen peripheral blood stem cells, a procedure on an outpatient basis, and employing a non-myeloablative regimen.
HSCT in CIDP, Neuromyelitis optica and Stiff person syndrome - Basil Sharrack
In conclusion, Basil Sharrack discussed on HSCT outcomes in rare neurological ADs. He reported long-term remission in treatment refractory CIDP patients, with overall low toxicity and good cost-effectiveness profile. For neuromyelitis optica spectrum disorder and stiff person syndrome, HSCT appears to a valid option but more trials are needed to determine long-term efficacy.
HSCT in rheumatologic and gastroenterological indications. Chairs: Dominique Farge & Tobias Alexander
Role of reduced intensity regimens, CD34 and ATG on outcomes in SSc - Jörg Henes
Jörg Henes discussed the role HSCT in patients with systemic sclerosis (SS), highlighting the need to minimize toxicity in this frail population, recommending the use of cyclophosphamide-based mobilizing protocols and remarking the benefit from CD34 selection.
Hematopoietic stem cell transplantation for Crohn’s disease: Gaps, doubts and perspectives - James Lindsay
James Lindsay reported the ASTIClite trial results in Crohn disease, focusing on toxicity profile and clinical results. Responses to HSCT are variable. Complete and impressive long-term medication-free responses have been achieved also in this difficult-to-treat population, although not in a high proportion of patients, at least not without reintroduction of treatment.
Outcomes of autoimmune cytopenia after HSCT - Nichola Cooper
Nichola Cooper reported, from past studies, considerable morbidity as well as high relapse rate in autoimmune cytopenia undergoing HSCT. However, new HSCT platforms will likely reduce toxicity and improve the overall outcome, warranting further studies in these indications.
HSCT in SLE - Tobias Alexander
Finally, Tobias Alexander reported high rate of durable treatment-free remission in systemic lupus erythematosus (SLE) patients undergoing HSCT, with demonstrated self-tolerance restoration, but pointed out the need to monitor carefully for very late relapses.
Immunologic mechanisms and novel targets for Cellular Therapy. Chairs: Raffaella Greco & Paolo Muraro
Fecal microbiota transplantation - Florent Malard
Florent Malard overviewed the use of fecal microbiota transplant (FMT). FMT strategy is well known in allogeneic setting, for its protective role against graft versus host disease, as well for its ability to restore microbiota diversity after transplant. Its role in the autologous setting is promising, although under investigation.
Application of CAR-T regs in autoimmune diseases - Matteo Doglio
Matteo Doglio gave updates on ongoing studies for CAR-T regs in SLE, a very promising and rapidly evolving field that might be expanded also in other ADs. In SLE mice models, CAR-T regs showed to be safe, significantly delayed lymphopenia and did not determine increase of proinflammatory cytokines; persistence of the cells is still a matter of debate.
Immunological mechanisms for tolerance induction after HSCT in multiple sclerosis - Peter Darlington
Finally, Peter Darlington reported data on immunologic studies in MS patients undergoing HSCT, suggesting a key role for NK cells regulatory function.
Patient, Nurses and Physician joint Session | Part I. Chairs: Helen Jessop, Majid Kazmi & Nicoletta Del Papa
Fertility issues following HSCT for Ads - Azza Ismail & Louise Willetts
The first speaker was Azza Ismail, who discussed on the long-term consequences of fertility in AD patients after HSCT. She presented the multicentre retrospective study of the ADWP whose primary endpoint is to determine the individual characteristics of patients with AD who underwent a pregnancy after HSCT and develop a consensus guideline on the pre-transplant counselling in patients and the subsequent management of pregnancy. She also shared the experience of a new joint service MS and Maternity Clinic that is carried out in her centre, in which she highlighted the importance of working with different specialists in a joined and coordinated way to improve care for patients.
The next speaker was Louise Willetts, who shared her experience when, after the transplant, she suffered early menopause and the whole process she lived to become a mother again. She highlighted the importance of patients not only receiving information about HSCT but also about long-term complications like the ones she experienced herself.
Patient perspective on HSCT during COVID-19 pandemic - Victoria Amory
Finally, we were able to hear the testimony of another patient, Victoria Amory, who explained how the pandemic delayed her HSCT and how the restrictions caused by it increased the feeling of isolation.
Patient, Nurses and Physician joint Session | Part II. Chairs: Ariadna Domenech, Basil Sharrack & Enrico Morello
Nutrition aspects in HSCT - Chiara Gandossi
The session started with Chiara Gandossi focusing on the specific nutritional aspects during the HSCT. The presentation highlighted the need to establish protocols concerning nutrition in patients based on the underlying diseases and not only about transplant and conditioning regimen toxicity.
Provision of HSCT for AD within Europe - Helen Jessop & Boaz Spermon
The next speaker was Helen Jessop presenting the survey of funding of AHSCT procedures for AD via the public health system in countries reporting to the EBMT. The results presented revealed significant differences between the member countries of the EBMT regarding the access to HSCT for AD patients.
The last speaker was Boaz Spermon, who contributed his experience as a patient and manager director at the Start2cure foundation. He explained his experience going abroad to underwent an HSCT, showing once again the differences between countries.
Finally, the role of patient organizations in the dissemination of quality and consensual information regarding AHSCT and its indications in AD was also addressed.
The meeting was very interesting, shared ideas on recent developments and gave new insights in this field. We look forward to encouraging physicians, nurses, trainees and as well as patients, to attend future meetings to promote education and foster future collaboration.