Oncogenic and immunological targets for matched therapy of pediatric blood cancer patients: Dutch iTHER study experience.

Over the past 10 years, institutional and national molecular tumor boards have been implemented for relapsed or refractory pediatric cancer to prioritize targeted drugs for individualized treatment based on actionable oncogenic lesions, including the Dutch iTHER platform. Hematological malignancies form a minority in precision medicine studies. Here, we report on 56 iTHER leukemia/lymphoma patients for which we considered cell surface markers and oncogenic aberrations as actionable events, supplemented with ex vivo drug sensitivity for six patients.

The Development of New Agents for Post-Hematopoietic Stem Cell Transplantation Non-Infectious Complications in Children.

Hematopoietic stem cell transplantation (HSCT) is often the only curative treatment option for patients suffering from various types of malignant diseases and some non-cancerous conditions. Nevertheless, it is associated with a high risk of complications leading to transplant-related mortality and long-term morbidity. An increasing number of therapeutic and prevention strategies have been developed over the last few years to tackle the complications arising in patients receiving an HSCT.

Implementation of paediatric precision oncology into clinical practice: The Individualized Therapies for Children with cancer program 'iTHER'.

iTHER is a Dutch prospective national precision oncology program aiming to define tumour molecular profiles in children and adolescents with primary very high-risk, relapsed, or refractory paediatric tumours. Between April 2017 and April 2021, 302 samples from 253 patients were included. Comprehensive molecular profiling including low-coverage whole genome sequencing (lcWGS), whole exome sequencing (WES), RNA sequencing (RNA-seq), Affymetrix, and/or 850k methylation profiling was successfully performed for 226 samples with at least 20% tumour content.

Disaster zones-should we be clowning around?

Unlabelled: Medical clowns have an important role in helping patients cope with their pain and distress. This is especially true in the pediatric population. However, their activity in a disaster area is unheard of.

Therapy with eculizumab for patients with CD59 p.Cys89Tyr mutation.

Objective: The objective of this work was to report on the outcome of eculizumab treatment in pediatric patients with recurrent acute predominantly motor, demyelinating neuropathy with conduction block, and chronic hemolysis attributed to p.Cys89Tyr mutation in the CD59 gene.

Methods: Four patients were recruited from our new registry of patients with homozygosity for the p.

Procedural sedation and analgesia in the emergency room of a field hospital after the Nepal earthquake.

Procedural sedation and analgesia (PSA) should be a priority in the emergency care of injured children. This humanistic approach is particularly important in paediatric victims of disaster, because these patients are prone to psychological distress secondary to the traumatic event. Following the Nepal earthquake, an Israeli field hospital (IFH) was deployed in Kathmandu.

Unexplained complaints in primary care: evidence of action bias.

Purpose: Primary care physicians sometimes encounter patients with clinical complaints that do not fit into a recognized diagnostic pattern. This study was undertaken to assess the way physicians respond to patients whose symptoms are unusual or unexplained that is, what approach they take in the absence of a working hypothesis.

Methods: We surveyed 130 primary care physicians affiliated with 3 academic centers in Israel, presenting 5 clinical vignettes describing patients who had unusual complaints, no clear diagnosis, and no apparent need for urgent care.

Underdiagnosed menorrhagia in adolescents is associated with underdiagnosed anemia.

Objective: To test the hypothesis that adolescent girls with menorrhagia rarely seek medical attention.

Study Design: A total of 705 adolescent girls attended a lecture on menorrhagia, completed an initial anonymous questionnaire, and were asked to participate in a more comprehensive study comprising a detailed bleeding questionnaire, a pictorial blood loss assessment chart, and blood tests.

Results: A total of 105 adolescents (15%) reported they had heavy periods on the initial questionnaire.