Two doctors from the Pediatric Oncology and Hematology Service of the Vall d'Hebron University Hospital and researchers from the Childhood Cancer and Hematological Diseases group of the Vall d'Hebron Research Institute (VHIR), won the first and second prizes for best communication at the national congress of the Sociedad Española de Hematología Oncología Pediátrica (SEHOP).

Throughout the three days of the congress, fourteen research papers were presented in ten-minute oral presentations. On the last day a SEHOP's jury decided which were the best. The first place went to Dr. Luz Uria for a study on the incidence of Avascular Bone Necrosis in patients who have received a bone marrow transplant. Second place went to Dr. Raquel Hladun for results about the efficacy of Larotrectinib in pediatric cancer patients with NTRK fusion.

A common complication in bone marrow transplants

The aim of the study presented by Dr. Luz Uria was to determine the incidence of cases of Avascular Bone Necrosis (ABN) among patients who had undergone a Hematopoietic Progenitor Transplant (HPT). HPT, popularly known as bone marrow transplants, are potentially curative treatments for some cancers and other severe primary immunodeficiencies. Hematopoietic progenitors are obtained primarily from bone marrow or umbilical cord blood.

AON is the death of bone tissue. Those affected present a lower bone density that can cause fractures and even total collapse of the bone, resulting in high long-term morbidity. To determine its prevalence among those who had undergone HPT, there was a review of the medical records of pediatric patients who had undergone surgery after more than 12 months have passed. In total, 22% developed a NOA. In view of the high incidence found, it was decided to implement an institutional protocol for the follow-up of transplant recipients with the collaboration of different diagnostic specialists. The goal is to improve prevention, early diagnosis, and find potential therapeutic treatments. This protocol has laid the foundations for the development of the prospective study "OssiPed" led by the Rheumatology and Pediatric Hemato-oncology Departments.
 

A new treatment for cancers with NTRK fusion

Dr. Raquel Hladun presented the results concerning the efficacy of Larotrectinib in pediatric patients with an extracerebral solid tumor with NTRK fusion, included in two international clinical trials: the pediatric phase ½ SCOUT trial and the phase 2 trial for adolescent and adult patients NAVIGATE. TRK proteins are essential for the development, function and maintenance of neuronal tissues. Recently it has been described how an abnormal function of these proteins has an oncogenic effect. This fusion of neurotrophic receptor tyrosine kinase (NTRK) genes is at the origin of some adult and pediatric tumors.

Larotrectinib is the first inhibitor of TRK proteins and is currently licensed in more than 40 countries for the treatment of adults and children with an extracerebral solid tumor with NTRK fusion, Its efficacy and safety in pediatric patients has been proven. The results were: an objective response rate of 88%, a progression-free survival of 60% at 36 months and an overall survival of 97% at 36 months. Given the great results of the trial, Dr. Hladun's team emphasizes the need to have and offer the required diagnostic techniques to detect NTRK fusions in pediatric tumors, thus being able to identify patients who can benefit from the new treatment.