Report of the 8th International Transplant and Cellular Therapy Course - 8-10 September 2023 - Barcelona, Spain

Summary written by Carmelo Gurnari, Tor Vergata University, Rome, Italy

The first session was dedicated to conditioning regimens, graft preparation and donor’s choice with regards to HLA typing/compatibility and germline predisposition and was chaired by Drs. Florent Malard (France) and Carmelo Gurnari (Italy/US).

In the first talk, Neema Mayor (UK) gave a broad overview of the HLA system, its role in determining donor’s choice and the impact of ethnicity on the possibility of finding a match. Emphasis was given particularly to the role of mismatches in HLA class I and II and their impact on post-transplant outcomes. In the second talk, Dr. Florent Malard illustrated the history of conditioning regimens, their evolution over time and the new strategies including reduced-intensity/toxicity approaches, which are enabling more and more patients to access to transplant. The session continues with Dr. Carmelo Gurnari explaining how important it is to consider germline predisposition in the setting of allogeneic hematopoietic cell transplant. Briefly, Dr. Gurnari explained criteria to screen patients, to identify germline variants and what to do in case of specific instances such as the segregation of a germline variant in a family. Finally, Dr. Oscar M. Pello (Spain) displayed the methods to collect and cryopreserve hematopoietic stem cells, along with quality controls and assessments needed to ensure an optimal transplant procedure.

Summary written by Kristin Rathje, University Medical Center Hamburg-Eppendorf, Germany

Session 3 dealt with the prophylaxis and treatment of graft-versus-host disease (GvHD) and was chaired by Dr. Olaf Penack (Germany) and Kristin Rathje (Germany).

Dr. Florent Malard (France) started out by giving a broad overview of the history of GvHD prophylaxis and illustrating well-established and novel approaches in the prevention of GvHD. In his second talk, Dr. Malard displayed current options for first-, second- and third-line therapies of acute GvHD as well as frequent treatment complications. The session continued with Dr. Olaf Penack giving a comprehensive overview of pharmacological treatment options for patients with steroid-refractory chronic GvHD. He explained possible clinical challenges especially in patients requiring long-term steroid therapy and highlighted the importance of multidisciplinary care in the management of chronic GvHD. The session was concluded by two clinical cases. Dr. Ang Chieh Hwee (Singapore) presented the case of a patient who developed polymyositis after transplantation. She focused on challenges in the diagnostics and management of this rare manifestation of chronic GvHD. Lastly, Dr. Lucas Vogt Cota (Brazil) reported the clinical case of a young male patient who received peeling and laser therapy for the treatment of facial hyperpigmentation after chronic skin GvHD.

Summary written by Esra Gülderen, Resident Physician, Uludag University, Bursa, Turkey

The first session of the second day in the adults' track was dedicated to exploring the indications for hematopoietic cell transplantation (HCT) in adults, with a primary focus on acute myeloid leukaemia (AML) and acute lymphoblastic leukaemia (ALL). This session was chaired by Dr. Fabio Ciceri (Italy) and Dr. Esra Gülderen (Turkey)

Eolia Brissot (France) began the session by providing a comprehensive overview of transplant approaches in AML, taking into account risk stratification. She highlighted the critical role of Minimal Residual Disease (MRD) status in guiding therapy choices for AML patients. The importance of novel therapies and targeted approaches was emphasised in preventing AML relapse in the post-HCT period. Dr. Brissot also underlined the need for further research for refractory AML to enhance outcomes for this subgroup of patients.

Then, Dr. Sebastian Giebel (Poland) delved into the intricate subject of donor selection in ALL, addressing donor types, donor sources, therapeutic strategies, and Graft-versus-Host Disease (GvHD) prophylaxis. His insights shed light on the complexities involved in optimising donor choices for ALL patients.

The session continued with Dr. Piotr Pietowski (Poland) presenting a compelling case study that focused on the challenges posed by complex karyotype abnormalities in a young patient with AML. His presentation underscored the need for tailored approaches to address the unique clinical circumstances encountered in such cases.

Dr. Sally Thomas (UK) concluded the session with an intriguing case study. She highlighted the role of rapid turnaround WGS in donor selection for AML patients with specific genetic mutations, such as the CEBPA mutation. Her presentation exemplified the evolving role of genomics in optimising HCT outcomes.

This session provided a comprehensive exploration of the indications for HCT in adults, particularly focusing on AML and ALL. The diverse topics covered in this session, ranging from risk stratification to donor selection and the role of genomics, demonstrated the multifaceted nature of HCT in acute leukaemia patients. The insights shared by the speakers underline the importance of personalised approaches and ongoing research to improve the outcomes of HCT for these patient populations.

Summary written by Alexandros Rampotas, UCL Cancer Institute, London, UK

This session provided an excellent overview of the available data on transplantation in Myelodysplastic Syndromes (MDS) and Myelofibrosis (MF) delivered by Prof Kroger and Dr Donal McLornan. It also featured four compelling clinical cases that underscored the complexities of determining the optimal timing and the most suitable transplant candidates within this challenging patient cohort. 

Kristin Ratje's presentation highlighted the potential benefits of an improved and optimised scoring system. Such a system could effectively identify MDS patients who stand to gain the most from transplantation, enhancing the precision of patient selection. 

Angela Hwang's presentation shed light on the challenges involved in deciding between transplantation and conservative management in Myelofibrosis. Her insights into this decision-making process illuminated the intricate balance of risks and benefits that clinicians and patients must navigate. 

Alex Rampotas presented a captivating case study involving the successful salvage of a Myelofibrosis relapse post-transplant through the approach of Donor Lymphocyte Infusion (DLI). This case exemplified the potential for the underutilised DLI to yield sustained remission, offering a therapeutic option for an otherwise poor prognosis cohort. 

Deepesh Lad's presentation focused on the role of Haploidentical transplantation in Myelofibrosis, providing valuable insights into this transplantation modality's place within the treatment landscape. 

Throughout the session, there was a dynamic exchange of ideas and experiences between presenters and the engaged audience. This interaction not only facilitated knowledge consolidation but also challenged attendees to grapple with the intricate decisions involved in transplantation for MDS and MF patients and how available evidence could help in the decision making process.

Summary written by Rosamaria Nitti, Vita-Salute San Raffaele University, Milan, Italy

Session 7 dealt with Indications for HSCT in adults with a focus on plasma cell disorders and autoimmune diseases.

The plasma cell disorders lecture was introduced by a clinical case by Dr Edward Jones. This case highlighted the difficulties in the management of a fit patient with an emergency presentation of spinal cord compression by multiple myeloma, leading to the need to initiate treatment before well-established diagnosis and risk stratification. Moreover, first line treatment was changed after one cycle of therapy due to the new accessibility of Daratumumab in first line quadruplets in the country. In multiple myeloma, the treatment scenario is rapidly evolving, with introduction of new therapies with marvellous outcomes but lack of long-term follow up and lack of certainties about the best sequences. Dr Patrick Heyden went into details about available evidence on treatment schemes and the role of autologous HSCT in multiple myeloma, still standard of care in fit patients in first line today, given the impact on overall survival (still present if delayed to first relapse in standard risk patients) in previous studies. In recent studies with new therapies, overall survival is often not reached, and combined effect of other lines of therapy makes the role of autologous HSCT less evident. The role of allogeneic HSCT is becoming more limited to severe cases like plasma cell leukaemia or high risk, young patients with poor response to therapy, instead, the arrival of CAR-T cell therapy gives a new weapon to treat patients with advanced disease.

The session moved to autoimmune diseases, with an introductory case presented by Dr Andrea Acerbis. This case highlighted the peculiarities of dealing with patients with autoimmune diseases, who have frailties due to previous therapies and functional impact of the disease. This multiple sclerosis patient had a head trauma with intracranial bleeding during aplasia after cyclophosphamide and could not proceed straight with leukapheresis, so a new attempt at chemo-free mobilisation was necessary, with the addition of steroid to GCSF to reduce the risk of disease flares. Due to these events and to intercurrent SASRCoV2 infection, the conditioning regimen was remodulated to a reduced intensity conditioning. Dr Raffaella Greco gave an overview of current indications to HSCT in autoimmune diseases, starting from the standard indications in aggressive relapsing/remitting multiple sclerosis and in systemic sclerosis. Autologous HSCT has a specific role in autoimmune diseases, aiming at resetting/rebalancing the immune system and restoring immune tolerance. This mechanism is highly effective in inflammatory components of the disease, but cannot reverse fibrosis and tissue degeneration that is the consequence of inflammation. For this reason, patient selection is paramount, both by moving HSCT earlier in the disease course, in phases of active inflammation, and by identifying patients with severe organ dysfunction (like cardiac impairment in systemic sclerosis) that impact on the safety of the procedure. For other, less frequent autoimmune indications to HSCT, experience is gradually growing, including cases treated with allogeneic HSCT and first promising experiences with anti CD19 CAR-T cells.

The session ended with two interesting clinical cases. Dr Nicolò Rampi presented a case with difficult diagnosis and management of chronic GvHD, in which the unusual clinical presentation triggered the evaluation of autoantibody panels, critical to shift the diagnosis to MDA5 Autoantibody-associated dermatomyositis. Curiously, in spite of absence of family history and previous signs or symptoms, the family donor developed a similar but milder presentation, with positivity of autoantibodies, suggesting a genetic predisposition. Dr Paweł Kiciński presented a case of an adult patient undergoing allogeneic HSCT for immunodeficiency due to XIAP gene mutation, characterised by both severe recurrent infections and autoimmune manifestations. Given previous disappointing attempts with MAC in this disease, a RIC conditioning was chosen, combined with PBSCs from MUD donor and “reinforced” GvHD prophylaxis with ATG, MMF, MTX, tacrolimus. The procedure was well tolerated and full engraftment was obtained. Sharing experiences about successful HSCT in rare diseases is paramount to build evidence for the benefit of future patients.

Chairs: Hilda Mekelenkamp (NL), Michelle Kenyon (UK)

Summary by Marijke Quaghebeur (BE)

Right donor, right patient, right time - Annika Kisch (SW)

Annika Kisch guided us through the current guidelines and recommendations.  She highlighted that care and information to recipient and donor must be separated.  Each person involved, patients and (potential) donors, must be respected and treated as an autonomous person with autonomous choices. Beside this, the potential donors deserve honest and correct information.  Health care professionals should be aware that donors are exposed to drugs without personal therapeutic benefit.  We closed this talk with an inspiring discussion about choosing a relative donor.

Role of the apheresis nurse, more than just cell collection - Nathalia Lorente del Rio (SP)

Nathalia highlighted the role of the apheresis nurse. Nursing education is crucial in apheresis. They play a key role in the patient and donor assessment about eligibility and suitability.  Current guidelines and practical challenges were mentioned. Preparation and monitoring of the patient during the apheresis, followed by patient follow-up is essential. The talk was finalized with a holistic approach for more patient centered care.

Conditioning regimes, different approaches and why -    Marijke Quaghebeur (BE)

Marijke focused on how to choose the appropriate conditioning regimen. Patient, disease and graft factors play an important role.  The most frequent used conditioning regimens were discussed. How to evaluate patient fitness and nursing aspects plays a vital role in this process.  At least, she gave us a comprehensive overview of common toxicities of myeloablative and non-myeloablative conditioning regimens.

Cell infusion: stem cell, CART and lymphocyte infusions: exploring the differences - Julia Ruiz Pato (SP)

Julia shared her knowledge on safe cell infusion and important considerations for nurses.  Objectives and indications of the different infusions were mentioned. JACIE standards describe to follow institutional policy and SOPs. A well-trained team to provide education, safe administration of cellular therapy and prevent and recognize adverse events are crucial. Overall, an optimal communication between the processing unit and transplant unit is necessary.

Chair: Julia Ruiz Pato (SP), Daniel Kisielewski (DE)

Summary by Sandra Schönfeld (CH)

Nutritional support, the current guidelines – Katie Ahern (IE)

Session 2 covers topics related to post-infusion care. Katie Ahern from Ireland started the session with the topic Nutritional Support - the current guidance. The latest evidence shows that the guidelines will be significantly relaxed, as the strict low-germ diet does not seem to have any benefit for patients. Here a small discussion arose around the different handling of nutrition after HSCT.

CVCs – evidencing best practice in 2023 – Eline Öfverberg (SE)

Eline Öfverberg from Sweden then gave an overview of the current evidence of best practice in relation to CVCs.

Neutropenic fever and sepsis – Sandra Schönfeld (CH)

The topic of neutropenic fever and sepsis was summarized by Sandra Schönfeld from Switzerland. The focus here was on the basics of neutropenic fever and how nurses can recognize it.

Oral mucositis, prevention, assessment and care – Ida Breemer (NL)

Finally, Ida Breemer from the Netherlands gave us an overview of current knowledge related to oral mucositis. The focus was on prevention, assessment and nursing support.

Chair: Thomas Jezequel (FR), Sandra Schönfeld (CH)

Summary by Daniel Kisielewski (DE)

Big issues: sex and sexuality after transplant – Johan De Munter (BE)

The 3rd session dealt with topics related to supportive care. Johann de Munter began with insights into the topic of sex and sexuality after transplantation, as its relevance to patients is underestimated and structured integration into nursing care protocols still is not sufficient. 

Fungal infections and treatment – Daniel Kisielewski (DE)

Daniel Kisielewski gave an overview of fungal infections during and after transplantation, the importance of which could increase in the future due to multidrug-resistant fungal pathogens. 

Chimerism – Daphna Hutt (IL)

In the third lecture, Daphna Hutt spoke on "Understanding chimerism," where the focus was on the diagnosis and implications of chimerism, as well as on aspects of mixed chimerism. 

Advanced care planning conversations and palliative care: where they fit – Hilde Mekelenkamp (NL)

Lastly, Hilda Melekenkamp presented on "Advanced care planning conversations and palliative care." Here the focus was on distinctions and overlaps of both topics. Furthermore, she presented the evidence of symptom burden on pediatric and adult patients in the HSCT setting. Finally she talked about adequately timed beginning of ACP and PC activities and the question of when and how nurses can discuss these topics with their patients.

Chair: Lidia Hurtago (SP), Marijke Quaghebeur (BE)

Summary by Thomas Jézéquel (FR)

GvHD new therapies and old assessment – Kristin Rathje (DE)

This first presentation of the Update session is an opportunity to highlight a subject that remains an important concern for patients undergoing transplant procedures, and therefore for the nurses who accompany them. Dr. Kristin Rathje from Hambour-Eppendurf University Hospital in Germany, offers an overview of the grading methods and new treatments available to manage this post-allograft complication. Dr. Rathje's brilliant presentation focused on the issue of chronic GvHD, and provided an opportunity to discover new treatments such as Belumosudil.

Pediatric update – Thomas Jézéquel (FR)

This presentation suggested some updates in the field of pediatric HSCT. The FORUM trial was presented and suggest that TBI and etoposide is still the recommended conditioning regimen for children with ALL. An ancillary study suggest that MUD is no longer a risk factor compared to MSD in terms of overall survival and event-free survival. At least but not last, a recent retrospective German study has shown a remarkable improvement in survival rates for patients with refractory disease or who have experienced relapse after HSCT.

Advanced therapies: where are now with CART and BITEs – Maaike De Ruijter (NL)

Maaike de Ruijter, a nurse practitioner from Amsterdam, make a very useful presentation about advanced therapies such as CART and BITEs. After defining each treatment and highlighting their different mechanism of action, she was able to illustrate the results of studies evaluating their efficacy and suggest new perspectives. With the rapid expansion of these new therapies, nurse education must be at the heart of our institution to contribute to quality of care.

Haemorrhagic cystitis – the role of PTCy, other causes, care, and consequences – Styliana Bouziana (UK)

Haemorrhagic cystitis is a difficult-to-manage complication that occurs in the post-transplant period and can significantly alter patients' quality of life or even threaten their life expectancy. This very insightful presentation was given by Dr. Styliane Bouziana from King's College Hospital in London. Although this complication is well known to nurses, this presentation provided an opportunity to review the risk factors such as post-transplant cyclophosphamide or infection with the BK virus. Moreover, Dr Styliane Bouziana immersed us in the specific pathophysiological mechanisms of this complication, which are very interesting to improve our ability to recognize this complication and adapt our daily monitoring.