Report of the Paediatric Diseases Working Party Educational Course on conventional hematopoietic stem cell transplantation, various gene editing techniques for autologous stem cells, or CAR-T cell therapies.
20 – 22 June 2025, Wrocław, Poland
Summary report written by Dalila Paoli, University Hospital Regensburg, Germany
From 20 to 22 June 2025, the beautiful Silesian city of Wrocław hosted the annual Educational Meeting of the EBMT Paediatric Diseases Working Party (PDWP). Under the guidance of PDWP Chair Krzysztof Kałwak, PDWP Secretary Katharina Kleinschmidt, and Nurse Coordinator Ida Bremer Ophorst, more than 130 delegates from 24 countries—including physicians, nurses, researchers, and industry partners—gathered for three intense days devoted to the frontiers of hematopoietic stem cell transplantation (HSCT), gene therapy, and cell-based treatments in children.
Educational Programme – Physicians’ Track
Day 1 – Friday, 20 June 2025
The welcome words from PDWP Chair Krzysztof Kałwak and Katharina Kleinschmidt opened the meeting, which began immediately with a fascinating session.
Session I, chaired by Christina Peters and Jochen Büchner, focused on the evolving landscape of HSCT and cellular therapy in pediatric acute lymphoblastic leukemia (ALL), bridging past evidence from FORUM I with future directions outlined in FORUM II.
The opening session revisited key findings from the FORUM I study. Christina Peters reaffirmed the importance of total body irradiation (TBI) combined with etoposide as the most effective conditioning regimen for children aged two and older. Despite side effects, this approach offers the best balance between survival and toxicity.
However, the focus quickly shifted to the future: Franco Locatelli introduced FORUM II, an ambitious effort to reduce long-term toxicity through strategies such as reduced-dose TBI, early GVHD management with ruxolitinib, and post-transplant immunotherapy with blinatumomab—especially in younger patients. In the same session, Peter Bader delivered a thorough update on CAR-T cell therapy for relapsed or refractory B-ALL. Real-world studies conducted since the approval of tisagenlecleucel confirm its remarkable potency: complete remission is achieved in approximately 85–87% of treated patients, most of which are MRD-negative. One- and two-year event-free survival rates hover around 45–50%. A high disease burden at the time of infusion worsens outcomes, whereas cytogenetic risk factors appear to have little impact on response. Despite these encouraging figures, prognosis remains poor for children who relapse within six months of an HSCT—underscoring the urgent need for next-generation strategies.
Adriana Balduzzi completed the discussion by emphasizing the importance of minimal residual disease (MRD) in guiding transplant decisions, reinforcing the move toward personalized, risk-adapted treatment plans.
Session II turned to hemoglobinopathies and inherited bone marrow failure syndromes (BMF), where curative options are evolving rapidly. Selim Corbacioglu opened the session with long-term data on HSCT and lentiviral gene-addition therapies for sickle cell disease and β-thalassemia. Carlo Dufour echoed this personalized approach in his discussion of BMF syndromes, highlighting how early transplantation guided by molecular diagnosis is becoming the new standard of care. Roland Meisel closed the session with an exciting overview of non-chemotherapy-based conditioning, especially antibody-directed regimens that could dramatically reduce long-term toxicity in children with non-malignant diseases.
The first day concluded with a focused scientific meeting. The PDWP committee reviewed its current study portfolio, approved four new international trials, and officially welcomed early-career investigators into the group.
Day 2 – Saturday, 21 June 2025
The second day opened with Session III, chaired by Elif Ince and Jan Styczyński, dedicated to infectious complications—an ongoing challenge in pediatric HSCT. The session addressed CMV and EBV prophylaxis strategies, including viral load–guided pre-emptive therapy and the potential of off-the-shelf virus-specific T cells (VSTs). Marek Ussowicz presented the clinical development of tabelecleucel, a third-party EBV-specific T-cell product that is now transitioning from experimental use to clinical practice. The discussion highlighted the need for pediatric-specific protocols and timely interventions to reduce viral morbidity.
Session IV then shifted to conditioning regimens in malignant and non-malignant disorders and the essential topic of fertility preservation. In a well-balanced pro-con debate, Tayfun Güngör and Krzysztof Kałwak examined busulfan versus treosulfan, with the latter emerging as a promising, less toxic alternative. Andrea Jarisch discussed the gonadotoxic effects of conditioning, underlining the importance of long-term reproductive follow-up. Jean-Hugues Dalle concluded with a comprehensive overview of fertility preservation strategies, emphasizing that reproductive health must be systematically addressed in pediatric transplant planning.
The afternoon resumed with Session V, focused on graft-versus-host disease (GvHD). Talks explored mesenchymal stromal cells in steroid-refractory acute GvHD, the integration of JAK inhibitors such as ruxolitinib into standard practice, and emerging biomarkers for risk stratification. Chronic GvHD in adolescents was also discussed, highlighting the importance of age-adapted, patient-centered care.
Session VI spotlighted recent advances in academic CAR-T cell trials. Franco Locatelli presented exciting data from Rome’s in-house CAR-T program to reduce manufacturing times and increase accessibility. Robert Chiesa introduced genome-edited, off-the-shelf CAR-T cells targeting antigens like CD19, CD7, and CD33 to overcome the limitations of autologous approaches and broaden therapeutic indications.
Session VII, chaired by Robert Wynn and Dominik Turkiewicz, explored advances in HSCT for AML. Amir Hamidieh opened remotely from Iran with a striking pilot study using cord-blood platelet gel to heal stubborn GVHD-related skin ulcers. Robert Wynn emphasized MRD and cytogenetics as key drivers of transplant timing. Eva Rettinger showed how FLT3 and menin inhibitors are integrated into peri-transplant regimens to reduce relapse risk. Dominik Turkiewicz’s early NOPHO-SCRIPT data demonstrated that genetics-guided timing is already feasible across Nordic–Baltic centers. Collectively, the talks outlined a future defined by sharper timing, wider donor options, and precision-guided therapy.
Session VIII concluded the day with a focus on sinusoidal obstruction syndrome (SOS/VOD) and other endothelial complications. Katharina Kleinschmidt discussed data on VOD in non-transplant settings, particularly among patients exposed to hepatotoxic agents. Zofia Szmit presented insights from the Wrocław Center on diagnostic strategies and the use of defibrotide. Bernd Gruhn closed the session with risk prediction models combining clinical and genomic data for early intervention and prevention.
Day 3 – Sunday, 22 June 2025
Sunday opened with a detailed look at myeloid disorders, including CML, MDS, and JMML. Suttorp and Strahm explored the still-relevant role of transplantation in the age of tyrosine kinase inhibitors (TKIs), especially for high-risk or TKI-resistant patients. Molecular diagnostics increasingly guide transplant decisions, particularly in germline predisposition syndromes. Roswitha Lüftinger presented data on reduced-toxicity conditioning with treosulfan, showing promise for preserving organ function and fertility.
The final scientific session—"Pretty Kettle of Fish"—featured a lively, interactive format. Young investigators from across Europe and Asia presented rare, complex, and sometimes dramatic clinical cases that challenged current assumptions and sparked animated discussions. Topics included cancer predisposition syndromes, post-transplant immune dysregulation, life-threatening infections, and novel desensitization protocols in sensitized transplant recipients. This session highlighted the critical importance of multidisciplinary collaboration and clinical intuition, while fostering an open dialogue between trainees, early-career professionals, and senior experts.
Conclusions & Future Directions
The 2025 PDWP Educational Meeting fostered new network connections and renewed motivation to improve outcomes for children undergoing advanced therapies.
Across all sessions, several key themes emerged:
- Precision medicine is no longer the future—it is the present.
- Conditioning regimens are becoming safer and smarter.
- Supportive care—from infection prevention to fertility preservation—is essential to transplant success.
- Collaboration between centers, disciplines, and countries is vital to continued progress.
Educational Programme – Nurses’ Track
Summary report written by Ida Bremer Ophorst, Nurses Group Paediatric Committee Chair and PDWP nurse, Marjola Gjergji and Maria Finch, Paediatric Committee members.
Zuzanna Cepowska was the local Poland nurse who did a great job hosting and chairing several sessions.
The nurses sessions were held in parallel of the physicians’ track. Twenty four nurses from Poland, Slovenia, Spain, Italy, France, Denmark, UK, and the Netherlands participated in the meeting. The diversity of the group, with different levels of knowledge and experiences in caring of the HCT patients, made the meeting a success.
Session I: Transplant preparation
Hilda Mekelenkamp, from the Netherlands, started this session and talked about the “Shared decision-making” in HSCT where clinicians and patients work together to make informed choices. She emphasizes the importance of this process in order to better understand what matters mostly to the patient and their families making the best choice for the patient.
Maria Finch from UK, talked about the “Donor search/care of donor” describing the donor path with HLA typing from the siblings, to the parents and to the potential donors showing the UK reality with UK donor centres. An important aspect has emerged is the donor’s emotional support before, during and after.
Session II: Transplant complications
The first talk was on “Infection following HSCT and nursing intervention” and was performed by Laura di Russo from Italy. The nursing interventions were focused on how to prevent the infections like the hand hygiene, neutropenic diet and isolating roles but also during the treatment process with bacterial, virus and fungal infections.
Thomas Jezequel from France, talked about the VOD in children as a post-transplant challenge going through the risk factors and the diagnostic criteria. The nurses key role remains in early detection of symptoms in order to alert the medical team.
Acute GvHD was performed by Majken Høeg Olsen from Denmark. She talked about how the conditioning regimes impact on the aGvHD, so as the donor risk factors such as female donor to male recipient and the increased age. She ended her talk with a relevant case on skin aGvHD, how the nursing interventions were important for the success of the treatment.
At the end, Hilda Mekelenkamp gave a nice refresh how to write and submit a case abstract.
Session III: Supportive Care “developments or strategies”
Zuzanna Cepowska presented a “Single Centre Pilot Study on the implementation of Patient Reported Pain Assessment by Nurses in a Paediatric Oncology Inpatient Setting in Poland”. The study shows that the nurse’s pain reassessment increased more than 20% for patients and caretakers and it inherences the quality of pain assessment.
Ida Bremer Ophorst from the Netherlands talked about the “Oral care and Photo Bio Modulation”. She shows how by using the PBM at the right wavelength and intensity, it stimulates wound healing, regeneration and immune responses to inflammation reducing pain.
“Fatigue Management” was a talk from Sara Francis from UK. Fatigue is a very common problem, especially post BMT, so it’s very important to introduce it pre/during/post BMT by using an appropriate tool. In order to avoid it, it’s important to keep patient active with basic daily activities and self-management strategies but also focusing on living well with fatigue.
As last speaker of this session, Veronique van Noort from the Netherlands introduced the “Comfort care”, a type of medical care based on activities focused on relieving symptoms such as pain and emotional and psychological support, improving quality of life of the patients. She shows some examples how this activity was also helpful for the daily nursing practise.
Session IV: Dilemma
In session 4 the nurses group was dived in two groups and discussed a dilemma in Ethical and Moral considerations vs Shared Decision-Making and Quality of Live vs Quantity of live…… It is always valuable to discuss with each other our nursing perspectives especially in an international group of HCT care.
Session V: EBP & innovations
Our first speaker o this session, Agnieszka Majk from Poland presented (virtually) the experience of the Bydgoszcz Centre in MSC therapy in children post HSCT. She explained the benefits of MSC in treating GvHD and how they have been used in her centre on an experimental basis. The potential complications and the safety and administration of the cells was highlighted. Results and a case study was discussed as well as a conclusion, stating that long term clinical trials are needed to understand the optimal timing, doses, long term efficacy and risk of toxicity.
Marta Brzykowska also from Poland presented her centre’s experience of administering CART cells, focusing on the nurse’s role in this. She discussed how they educate and council patient and families about the procedure as well as coordinating the treatment. Nurses monitor the patient post infusion as well as daily care and symptom management. They also plan the patient’s discharge and provide education as well as the long term follow up. There was some discussion within the group about the physician’s role, as in this case they administer the cells whereas in a lot of countries this is a nurse’s role.
Rebecca Ongaro from Italy presented understanding, treatment approaches and nursing care and innovations in Fanconi Anaemia. She explained what Fanconi Anaemia is, how it’s diagnosed and potential treatment options available. She highlighted that there are many different roles of nurses through the patient journey such as the HSCT Research Nurse, the Nurse Coordinator, the Specialized Nurse and the Transitional/Discharge Nurse and described how these roles work. To conclude, early diagnosis is essential, and multidisciplinary collaboration is critical to ensure coordinated and holistic care. Incorporating the family in this is critical and the nurse plays a fundamental role in education, psychological support, ongoing patient care and in clinical research.
The last speaker of the session was Tamara Jimenez from Spain. She presented immunodeficiency from a nursing perspective. She explained about primary immunodeficiency and severe combined immunodeficiency (SCID). She then presented a case study of a patient with SCID and the genetic component of the condition. She gave a background of how the diagnosis was made and the initial treatment course as well as the HSCT journey. The support, burden and adaption of the family was highlighted as well as emerging therapies in treating SCID patients. To conclude nurses, have a key role as a guide, caregiver and support throughout the process and key recommendations for nurses is to listen, educate and to detect clinical and emotional signs.
Session VI: Challenges in care for the foreign patient
Zuzanna Cepowska from Poland presented her involvement in the SAFER Ukraine initiative of the evacuation of paediatric cancer and haematology patients from the war in Ukraine. This was initially to Poland and then on to other countries once patients had been triaged in a specialist centre that was set up especially for this purpose. She explained the processes involved and the challenges the teams faced in getting patients and their families to the most appropriate place to be safe for treatment to continue. Over 1500 patients were evacuated with the collaboration of 40 nurses as well as medics and interpreters. This generated lots of questions and discussion from the group who were fascinated and impressed by the process.
Maria Finch from the UK presented her centre’s experience in looking after non-UK born patients. She explained three different types of patients and families, the non-UK born but with citizenship, families in the UK as asylum seekers or as student visa holders and those in UK for treatment only. The consideration and challenges involved in caring for these patients was highlighted as well as a closer look at a case study which emphasised the issues raised. This generated some questions and discussion amongst the group.
We finished the day with a panel discussion including Ida Bremer Ophorst, Hilda Mekelenkamp, Zuzanna Cepowska and Maria Finch. We invited the participants to ask us questions, and they didn’t disappoint! Lots of questions were aimed at Zuzanna about her involvement with the evacuation of Ukrainian patients. Maria was asked about some potential solutions to the challenges faced with looking after non-UK patients.
Overall, the day brought together a group of nurses who were able to share their experiences and knowledge and have the opportunity to network with nurses from other countries.
