Oussama Abla’s research while affiliated with SickKids and other places

The upcoming 5th edition of the World Health Organization (WHO) Classification of Haematolymphoid Tumours is part of an effort to hierarchically catalogue human cancers arising in various organ systems within a single relational database. This paper summarizes the new WHO classification scheme for myeloid and histiocytic/dendritic neoplasms and provides an overview of the principles and rationale underpinning changes from the prior edition. The definition and diagnosis of disease types continues to be based on multiple clinicopathologic parameters, but with refinement of diagnostic criteria and emphasis on therapeutically and/or prognostically actionable biomarkers. While a genetic basis for defining diseases is sought where possible, the classification strives to keep practical worldwide applicability in perspective. The result is an enhanced, contemporary, evidence-based classification of myeloid and histiocytic/dendritic neoplasms, rooted in molecular biology and an organizational structure that permits future scalability as new discoveries continue to inexorably inform future editions.

Objectives: First, we aimed to determine the feasibility of virtual reality (VR) distraction for children with cancer undergoing subcutaneous port (SCP) access. Second, we aimed to estimate preliminary treatment effects of VR compared to an active distraction control (iPad). Methods: A single-site pilot randomized controlled trial (RCT) comparing VR to iPad distraction was conducted. Eligible children were aged 8-18 years undergoing treatment for cancer with upcoming SCP needle insertions. Intervention acceptability was evaluated by child, parent, and nurse self-report. Preliminary effectiveness outcomes included child-reported pain intensity, distress, and fear. Preliminary effectiveness was determined using logistic regression models with outcomes compared between groups using pre-procedure scores as covariates. Results: Twenty participants (mean age 12▒y) were randomized to each group. The most common diagnosis was acute lymphocytic leukemia (n=23, 58%). Most eligible children (62%) participated, and one withdrew after randomization to the iPad group. Nurses, parents, and children reported the interventions in both groups to be acceptable, with the VR participants reporting significantly higher immersion in the distraction environment (P=0.0318). Although not statistically significant, more VR group participants indicated no pain (65% vs. 45%) and no distress (80% vs. 47%) during the procedure compared to the iPad group. Fear was similar across groups, with approximately 60% of the sample indicating no fear. Discussion: VR was feasible and acceptable to implement as an intervention during SCP access. Preliminary effectiveness results indicate that VR may reduce distress and distress compared to iPad distraction. These data will inform design of a future full-scale RCT.

Background A pediatric cancer diagnosis and its treatment can have a detrimental effect on the mental health of children and their families. Screening to identify psychosocial risk in families has been recognized as a standard of care in pediatric oncology, but there has been limited clinical application of this standard thus far. A significant impediment to the implementation of psychosocial screening is the dearth of information on how to translate psychosocial screening to clinical practice, and specifically, how to follow-up from screening results. This manuscript aims to describe a protocol of a new intervention examining the feasibility and acceptability of mapping via a Psychosocial Navigator (PSN) psychosocial screening results to specific recommendations of resources for families based on measured risk for psychosocial distress and mental health symptoms. Methods The pilot randomized control trial (RCT) consists of dyads of youth (10–17 years) newly diagnosed with cancer and their primary caregiver. This RCT includes two arms (intervention and control group), with each group completing measurements near diagnosis and 1 year later. After the initial assessment, dyads in the intervention group receive monthly screening results and recommendations from the study PSN that are tailored to these results. The patient’s primary healthcare team (nurse, social worker, oncologist) also receive the risk, distress, and mental health results as well as the recommendations from the PSN. Discussion This study addresses a significant barrier to the implementation of psychosocial screening in pediatric oncology: specifically, the limited knowledge of how to follow-up from screening results. Findings from this pilot will inform a future definitive RCT to test the effectiveness of the intervention on patient and family mental health outcomes. This project has implications for enhancing clinical care in pediatric oncology, as well as other pediatric populations. Strengths and limitations of this study This is the first study of screening and follow-up using a psychosocial navigator. This study involves both patient and caregiver report. The small sample size necessitates a future larger study to investigate the effects of intervention. Trial registration NCT04132856 , Registered 10 October 2019—retrospectively registered.

Background Central nervous system (CNS) relapse is rare in childhood anaplastic large cell lymphoma (ALCL) and is associated with a poor prognosis. Case We describe an 8‐year‐old boy with ALCL who developed an early CNS relapse without initial CNS disease. Despite aggressive medical management, the patient's neurological status deteriorated rapidly and he died shortly after. Conclusion Optimal treatment for children with relapsed ALCL involving the CNS remains unclear. Novel agents, including ALK inhibitors, that have CNS‐penetration might be helpful and pediatric studies are warranted.

This case report describes a four-year-old boy who presented with the diagnosis of LCH with liver involvement. This required a living-related liver transplant one year later. The primary disease recurred in the transplanted liver 6 months post-transplant and led to progressive biliary dilatation. A percutaneous trans-hepatic cholangiogram was performed five years after transplant, showing a pattern of multifocal biliary duct strictures mimicking the pattern of primary sclerosing cholangitis and a stenosis of the biliary-enteric anastomosis. Despite management with an internal-external biliary drain, the stenosis of the biliary-enteric anastomosis evolved to an occlusion one year after drain removal. This was associated with progression of the changes in the biliary tree, this time associated with significant saccular dilatations secondary to the multiple areas of stenosis. Due to these findings and progressive deterioration of the function of the graft, the patient required re-transplantation. This report illustrates the findings in imaging of the biliary tree secondary to the recurrence of LCH after liver transplantation, which may help to recognize this complication to physicians facing a similar clinical scenario.

In this issue of Blood, Goyal and colleagues present the second consensus guidelines for the clinical diagnosis and treatment of patients with Erdheim-Chester disease (ECD), in the era of targeted therapies.

Purpose: Needle procedures are among the most distressing aspects of pediatric cancer-related treatment. Virtual reality (VR) distraction offers promise for needle-related pain and distress given its highly immersive and interactive virtual environment. This study assessed the usability (ease of use and understanding, acceptability) of a custom VR intervention for children with cancer undergoing implantable venous access device (IVAD) needle insertion. Method: Three iterative cycles of mixed-method usability testing with semistructured interviews were undertaken to refine the VR. Results: Participants included 17 children and adolescents (8-18 years old) with cancer who used the VR intervention prior to or during IVAD access. Most participants reported the VR as easy to use (82%) and understand (94%), and would like to use it during subsequent needle procedures (94%). Based on usability testing, refinements were made to VR hardware, software, and clinical implementation. Refinements focused on increasing responsiveness, interaction, and immersion of the VR program, reducing head movement for VR interaction, and enabling participant alerts to steps of the procedure by clinical staff. No adverse events of nausea or dizziness were reported. Conclusions: The VR intervention was deemed acceptable and safe. Next steps include assessing feasibility and effectiveness of the VR intervention for pain and distress.

In this issue of Blood, Carrera Silva et al demonstrate that CD207⁺CD1a⁺ circulating cells are increased in the peripheral blood of children with active Langerhans cell histiocytosis (LCH), in comparison with children with nonactive LCH and with healthy adults. Furthermore, CD14⁺ monocytes express CD207 in these patients under the influence of thymic stromal lymphopoietin (TSLP) and transforming growth factor β (TGF-β)¹.

Relapsed/refractory acute myeloid leukemia (AML) has an extremely poor prognosis. We describe 17 children and adolescents with relapsed/refractory AML who received clofarabine, cyclophosphamide, and etoposide. Seven patients (41%) responded: 4 with a complete response (CR); 1 with CR with incomplete platelet recovery; and 2 with a partial response. Additionally, 4 developed hypocellular marrow without evidence of leukemia; 5 patients had resistant disease; and 1 suffered early toxic death. After further therapy including transplantation, 4 patients (24%) are alive without evidence of disease at a median of 60 months. This anthracycline-free regimen may be studied for relapsed or refractory AML, but due to the high risk of marrow aplasia reduced doses of clofarabine and cyclophosphamide should be used.