Director of the Pediatric Hematology Diagnosis and Treatment Center
Chinese Academy of Medical Sciences Hematology Hospital (Institute of Hematology)
The 28th European Hematology Association (EHA) Annual Congress was held in Frankfurt, Germany, from June 8 to 11, 2023. Dr Xiaofan Zhu and his team from the Children’s Hematology Center at the Chinese Academy of Medical Sciences and Peking Union Medical College presented four abstracts at this EHA congress. These abstracts covered various aspects of research, including the prognostic factors for relapsed childhood acute lymphoblastic leukemia (ALL) treated with the CCLG-ALL2008 protocol in China, the use of Blinatumomab for 14 days in combination with chemotherapy to achieve minimal residual disease (MRD) conversion in pediatric B-ALL patients, the integration of transcriptomic features to improve prognosis prediction in pediatric acute myeloid leukemia (AML) with KMT2A rearrangements, and the effective treatment of pediatric non-sickle cell anemia (NSAA) with Hydroxyurea in combination with CSA. To gain a deeper understanding of these studies, Dr. Zhu has provided interpretations of the research findings, which are summarized below.
Abstract: P391
PROGNOSTIC ANALYSIS OF RELAPSED PEDIATRIC ACUTE LYMPHOBLASTIC LEUKEMIA TREATED BY CCLG-ALL2008 PROTOCOL IN CHINA
Relapse in childhood ALL mainly occurs in the very early phase and is mostly isolated bone marrow relapse, resulting in poorer survival rates. Central nervous system involvement and immunophenotype at diagnosis do not significantly affect the median survival of relapsed patients. HSCT after relapse improves the survival of high-risk patients, but further confirmation is needed to determine whether new treatment strategies such as cellular immunotherapy will further improve outcomes.
Abstract: P1402
INFUSION OF 14 DAYS BLINATUNOMAB IN COMBINATION WITH CHEMOTHERAPY FOR 46-DAY MRD+ PEDIATRIC B-ALL PATIENTS IN INTERMEDIATE/HIGH-RISK GROUP RESULTS IN MRD CONVERSION
Initial data from this exploratory study suggest that Blin-14 treatment in pediatric B-ALL patients with MRD+ on day 46 in the IR/HR group shows promising clinical activity and manageable safety. Rapid decline and subsequent rebound of T cells at a relatively high level suggest good efficacy. The study is ongoing, with further follow-up.
Abstract: P511
INTEGRATION OF TRANSCRIPTOMIC FEATURES IMPROVES PROGNOSIS PREDICTION OF PEDIATRIC ACUTE MYELOID LEUKEMIA WITH KMT2A REARRANGEMENTS
At the transcriptomic level, pediatric KMT2A-r AML is a heterogeneous disease, and transcriptomic features can improve prognosis prediction. This risk stratification can better differentiate patients with different risks, providing clinical utility and laying the foundation for further research into the pathophysiology of pediatric KMT2A-r AML.
Abstract: P792
CLINICAL STUDY OF HETROMBOPAG COMBINED WITH CYCLOSPORINE A IN THE TREATMENT OF CHILDREN WITH NON-SEVERE APLASTIC ANEMIA
Eltrombopag combined with CSA is effective in treating pediatric NSAA patients and shows efficacy in TD-NSAA children.
Researcher’s Remarks
Dr. Xiaofan Zhu: The four research studies presented at this EHA congress address challenging issues in the field of pediatric hematology. Through data analysis, we have established new prognostic models, guided treatment decisions, evaluated new treatment approaches, and aimed to improve treatment outcomes.
The study with Abstract P391 analyzed the clinical characteristics of relapsed childhood ALL patients, providing valuable insights into the timing and types of relapse, which can inform follow-up strategies and treatment decisions for these patients.
Abstract P511 explored new prognostic factors for pediatric KMT2A rearranged AML at the transcriptomic level, and the newly developed pKMT2A7 scoring system improves risk assessment and provides clues for better understanding the characteristics of this challenging disease.
The research described in Abstract P1402 investigated a novel approach to using Blinatumomab to reduce side effects while maintaining efficacy in pediatric B-ALL patients. Early efficacy results are promising, and further long-term follow-up is needed.
Lastly, the study presented in Abstract P792 examined the use of eltrombopag in combination with CSA for the treatment of pediatric NSAA, aiming to better adapt treatment for children and minimize the impact of the disease on their normal lives. The results show significant improvements in PLT, HGB, and ANC, with many transfusion-dependent patients becoming transfusion-independent. Therefore, eltrombopag combined with CSA treatment is worthy of further exploration.
In summary, these four studies represent the culmination of years of effort by the Children’s Hematology Center at the Chinese Academy of Medical Sciences and Peking Union Medical College to improve prognosis prediction and treatment for rare and refractory diseases. They provide preliminary evidence for clinical practice and lay the foundation for larger-scale research efforts.
