John Wiernikowski

Background: Venous thromboembolism (VTE) is a significant cause of morbidity in children, particularly among hospitalized patients and those with chronic medical conditions. There is a lack of consensus on anticoagulant prophylaxis strategies. Objective: These evidence-based guidelines from the American Society of Hematology (ASH) and the International Society on Thrombosis and Haemostasis (ISTH) are intended to support patients and health care professionals in decisions about anticoagulant prophylaxis for pediatric VTE prevention. Methods: ASH formed a multidisciplinary guideline panel that included 1 patient representative. The University of Kansas Health System supported the guideline development process, including systematic evidence reviews up to April 2025. Clinical questions and outcomes were prioritized according to their importance for clinicians and patients. The panel used the GRADE (Grading of Recommendations Assessment, Development, and Evaluation) approach to assess certainty in the evidence and make recommendations. Results: The panel agreed on 12 recommendations. For pediatric patients with solid cancer, who have experienced trauma, or who are critically ill, the panel issued conditional recommendations suggesting no anticoagulant prophylaxis. For pediatric patients with antiphospholipid antibody syndrome, or those on long-term total parenteral nutrition, the panel issued conditional recommendations suggesting the use of anticoagulant prophylaxis. Other pediatric subgroups addressed included patients with acute lymphoblastic leukemia or lymphoma, surgical and hospitalized patients, and those with a central venous access device. Conclusions: High-quality data on anticoagulant prophylaxis for pediatric VTE prevention are scarce. Key research priorities include the development and validation of subgroups-specific VTE risk assessment models, and evaluation of the safety and efficacy of risk-stratified anticoagulant prophylaxis strategies across different pediatric subgroups.

Background: The American Society of Hematology (ASH) guidelines on treatment of pediatric venous thromboembolism (VTE) were published in 2018. In the last 6 years, there has been a 10-fold increase in the number of children involved in VTE treatment trials. Objective: The ASH Committee on Quality and Guidelines agreed to update the pediatric guidelines in conjunction with the International Society on Thrombosis and Haemostasis (ISTH). These ASH/ISTH evidence-based guidelines are intended to support patients, clinicians, and other health care professionals in the management of pediatric patients with VTE. Methods: ASH/ISTH formed a multidisciplinary guideline panel to minimize potential bias from conflicts of interest. An unconflicted patient representative was not identified. The University of Kansas Health System supported the guideline development process, updating or performing systematic evidence reviews up to 2024. The panel focused specifically on the 2018 questions for which there was the greatest amount of interim data. The panel used the GRADE (Grades of Recommendation, Assessment, Development, and Evaluation) approach, including GRADE Evidence-to-Decision frameworks, to assess evidence and make recommendations, which were subject to public comment. Results: The panel agreed on 20 recommendations and also provided implementation guidance on the optimal use of anticoagulants in pediatric patients. Key recommendations of these guidelines include the role of DOACs in the treatment of a variety of pediatric VTEs. Conclusions: Further research is required. Key priorities are understanding the natural history of clinically unsuspected thrombosis across a range of patient subpopulations and obtaining real-world data on the use of DOACs in children.

Globally there are numerous impediments, both logistical, regulatory and more recently global drug shortages, hindering pediatric access to therapeutic drugs of all types. Efforts to reduce barriers are ongoing and are especially important in low and middle income countries and for children requiring treatment of conditions such as those encountered in pediatric oncology characterized by the risk of life threatening treatment failures. Progress has been made through the efforts of the World Health Organization and regulators in the US and Europe to encourage the development of therapeutic agents for use in pediatrics and measures taken have fostered the availability of stronger pediatric data to guide therapeutic decisions. Nonetheless, pharmaceuticals remain a global commodity, subject to regulation by the World Trade Organization and this has often had detrimental effects in low and middle income countries. This article emphasizes the need for closer international collaboration to address the barriers currently impeding access to antineoplastic and supportive care medicines for children. Pediatr Blood Cancer 2014; 61:1513–1517.

Although bendamustine has been used to treat lymphoproliferative disorders for decades, it has only recently been approved for use in Canada. Thus, Canadian recommendations on the administration of bendamustine and the management of common adverse events (aes) are needed. This article highlights effective management and assessment strategies recommended by Canadian nurses and pharmacists for the most common aes arising from the use of bendamustine in patients with chronic lymphocytic leukemia and indolent non-Hodgkin lymphoma. Those strategies include administering bendamustine over 60 minutes instead of 30 minutes, administering pre-medications to control infusion-related reactions and nausea, hydrating patients to minimize fatigue, and using free-flowing saline at the closest port to prevent phlebitis.

Controversies exist over the currently recommended guidelines for the use of low-molecular-weight heparin (LMWH) in neonates. We retrospectively studied 30 neonates treated with LMWH and found a poor therapeutic response to recommended doses as measured by anti-Xa levels. Sixty percent of the study participants required their doses to be increased because of subtherapeutic anti-Xa levels during the initial course of their treatment. The mean starting enoxaparin dose was 1.53 ± 0.38 mg/kg. The mean enoxaparin dose, once therapeutic anti-Xa levels had been achieved, was 1.86 ± 0.50 mg/kg. Preterm and term infants required doses of 2.06 ± 0.61 mg/kg and 1.67 ± 0.26 mg/kg, respectively, to achieve therapeutic anti-Xa levels. In summary, our results suggest that higher initial doses are required to achieve therapeutic anticoagulation in neonates.

The burden of cancer in children in low and middle income countries (LMICs) is substantial, comprising at least 80% of incident cases globally, and an even higher proportion of cancer‐related deaths. With survival rates exceeding 80% in high income countries, it is imperative to transfer these successes to LMICs. A major challenge is the poor availability of safe, cost‐effective chemotherapy. A list of 51 drugs—chemotherapeutics, infectious disease agents, and supportive care medications—is proposed as essential to improving the survival of children with cancer in LMICs with an additional 13 drugs identified as being of further value. Pediatr Blood Cancer 2013; 60: 889–891.

9005 Background: Survivorship care plans (SCPs) are recommended for patients who have completed primary treatment and are transitioning to routine follow-up care. SCPs are considered to have face validity but have never been tested in a randomized trial. The objective of this study was to evaluative a SCP for breast cancer survivors transitioning to routine follow-up care with their own family physician (FP). Methods: 408 early -stage breast cancer patients who completed primary treatment at least 3 months previously and without recurrent or new primary cancer were enrolled through tertiary care cancer centers throughout Canada and stratified into 2 groups: <24 months (n=180) and ≥ 24 months (n=228) since diagnosis. All patients were transferred to their own FP for follow-up care. In the intervention group, 200 patients received a SCP consisting of: a treatment summary; a patient-version of follow-up guidelines; brochures and information about local relevant supportive care resources. These were compiled ...

Osteopenia is a common consequence of the treatment of acute lymphoblastic leukemia (ALL) in children and adolescents, due predominantly to glucocorticosteroid therapy. The pathogenesis relates to an imbalance of resorption over formation of bone. Alendronate (Fosamax), an inhibitor of osteoclastic bone resorption, was administered for at least 6 months to 15 children with ALL during maintenance chemotherapy, after the diagnosis of osteopenia/osteoporosis by dual energy x-ray absorptiometry. The height velocity was also measured during the administration of alendronate and again 2 years later. Areal bone mineral density Z scores of the lumbar spine had a median value of -1.32 before administration of alendronate and a median gain of +0.64, with 14/15 children showing improvement. There was no adverse effect of alendronate on height velocity, and the drug was well tolerated with no short-term toxicity. This preliminary experience suggests a potential value in the use of alendronate for the treatment of osteopenia/osteoporosis in children with ALL and points to the need for a randomized controlled trial of this intervention.