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Les enfants atteints de maladies chroniques invalidantes et douloureuses utilisent de plus en plus le cannabis medical, particulierement lorsque les options therapeutiques classiques sont peu efficaces ou provoquent des effets [...]

Children with chronic debilitating illness and pain are increasingly using cannabis for medical purposes, particularly when conventional treatment options have limited benefit or substantial adverse effects. Caregivers are becoming aware [...]

•Compared with historical studies, 3-year survival for patients with pHLH improved to 77% for patients receiving first-line etoposide.•Contemporary outcome of patients with pHLH is better than anticipated, providing a benchmark for evaluation of novel treatment regimens.

Medical cannabis (MC) may offer therapeutic benefits for children with complex neurological conditions and chronic diseases. In Canada, parents, and caregivers frequently report encountering barriers when accessing MC for their children. These include negative preconceived notions about risks and benefits, challenges connecting with a knowledgeable healthcare provider (HCP), the high cost of MC products, and navigating MC product shortages. In this manuscript, we explore several of these barriers and provide recommendations to decision-makers to enable a family-centered and evidence-based approach to MC medicine and research for children.

As manager of telecommunications for Telecommunications Marketing Corporation (TMC), I have seen our company grow at an average rate of 33 percent per year for the last four years. This [...]

Introduction:Children with Down Syndrome (DS) have a 20-fold increased incidence of acute lymphoblastic leukemia (ALL) and a 70% higher risk of relapse than children with ALL and no DS. Treatment for DS children with relapsed ALL is challenging due to the high treatment-related mortality associated with both intensive chemotherapy and hematopoietic stem cell transplantation (HSCT) as well as high rates of second relapse. Outcomes for relapsed ALL in DS are dismal, with recent studies citing an event-free survival (EFS) as low as 17%. Alternative treatment approaches are needed for this patient population. Treatment with anti-CD19 chimeric antigen receptor T-cells (CARTs) have shown high remission rates in patients with multiple relapsed/refractory ALL with an acceptable safety profile. CARTs are generally not indicated for first relapse of ALL; DS-specific indications for CARTs do not exist. In Ontario, due to the specific susceptibility of children with DS-ALL, CARTs were made available to this population upon first relapse. We describe the population-based Canadian experience using CARTs for first relapse of DS-ALL.

A retrospective study was performed to collect information regarding efficacy and toxicity of cidofovir (CDV) in allogeneic stem cell transplant patients. Data were available on 82 patients. The indications for therapy were cytomegalovirus (CMV) disease in 20 patients, primary preemptive therapy in 24 patients, and secondary preemptive therapy in 38 patients. Of the patients, 47 had received previous antiviral therapy with ganciclovir, foscarnet, or both drugs. The dosage of CDV was 1 to 5 mg/kg per week followed by maintenance every other week in some patients. The duration of therapy ranged from 1 to 134 days (median, 22 days). All patients received probenecid and prehydration. Ten of 20 (50%) patients who were treated for CMV disease (9 of 16 with pneumonia) responded to CDV therapy, as did 25 of 38 (66%) patients who had failed or relapsed after previous preemptive therapy and 15 of 24 (62%) patients in whom CDV was used as the primary preemptive therapy. Of the patients, 21 (25.6%) developed renal toxicity that remained after cessation of therapy in 12 patients. Fifteen patients developed other toxicities that were potentially due to CDV or the concomitantly given probenecid. No toxicity was seen in 45 (61.6%) patients. Cidofovir can be considered as second-line therapy in patients with CMV disease failing previous antiviral therapy. However, additional studies are needed before CDV can be recommended for preemptive therapy.

TERCvariant telomere biology disorders are a rare and heterogenous group of disorders that arise from germline variants in TERC, a gene that encodes for the RNA component of telomerase. Variants in TERClead to accelerated telomere attrition and can manifest as a wide array of clinical phenotypes affecting multiple organ systems. In this case series, we aim to add to the literature describing TERCvariant telomere biology disorders by reporting cases from two unrelated families from Eastern Canada, one of whom was found to have a novel germline TERCvariant and the other had a unique phenotypic presentation of previously described TERCvariant.

ABSTRACTAdenovirus causes disseminated disease following bone marrow transplantation (BMT). We report a child who underwent T-cell-depleted BMT. Adenovirus subgenus F serotype 41 was detected antemortem by PCR in cerebrospinal fluid and postmortem in other tissues. Serotypes 40 and 41, associated with gastrointestinal disease, have not previously been implicated in disseminated disease.

BACKGROUND: Hematopoietic stem cell transplant (HSCT) recipients are at a high risk for late bloodstream infection (BSI). Controversy exists regarding the benefit of surveillance blood cultures in this immunosuppressed population. Despite the common use of this practice, the practical value is not well established in non-neutropenic children following HSCT.METHODS: At the IWK Health Centre (Halifax, Nova Scotia), weekly surveillance blood cultures from central lines are drawn from children following HSCT until the line is removed. A retrospective chart review was performed to determine the utility and cost of this practice. Eligible participants were non-neutropenic HSCT recipients with central venous access lines. The cost of laboratory investigations, nursing time, hospital stay and interventions for positive surveillance cultures was calculated.RESULTS: Forty-three HSCTs were performed in 41 children. Donors were allogenic in 33 cases (77%) and autologous in 10 cases (23%). There were 316 patient contacts for surveillance cultures (mean seven per patient) and 577 central line lumens sampled. Three of 43 patients (7%) had clinically significant positive surveillance blood cultures. Bacteria isolated were Klebsiella pneumoniae (n=2) and Corynebacterium jeikeium (n=1). All follow-up cultures before initiation of antimicrobial therapy were sterile. All three patients were admitted for antimicrobial therapy if they were not already hospitalized and/or had an uncomplicated course. The estimated total cost of BSI surveillance and management of asymptomatic infection over six years was $27,989.CONCLUSION: The present study suggests that BSI surveillance in children following HSCT engraftment has a very low yield and significant cost. It is unclear whether it contributes to improved patient outcomes.

Background: HSCT recipients are at high risk of late bloodstream infection (BSI). Controversy exists regarding the benefit of surveillance blood cultures in this immunosuppressed population. Despite the common use of this practice, its practical value is not well established in non-neutropenic children following HSCT. Methods: At the IWK Health Centre, a tertiary health care center serving 2.5 million in the three Maritime provinces of Canada, weekly surveillance blood cultures from central venous access lines (CVLs) are drawn from children following HSCT until CVL removal. We reviewed data from a prospectively collected CVL database in association with a health record review to determine the utility and cost of this practice. Eligible participants were non-neutropenic pediatric HSCT recipients with CVLs followed at the IWK between 1999 and 2005. Patients routinely received PCP and penicillin prophylaxis. Cultures were considered to be for surveillance if the child was afebrile without focus of infection. The cost of laboratory investigations, nursing time, hospital stay, and interventions for positive surveillance cultures was calculated. Results: 43 HSCTs were performed in 41 patients of whom 21 were male (49%). The median age was 7 years (range 1–18). Donors were related in 15 cases (35%), unrelated in 18 (42%) and autologous in 10 cases (23%). The most common underlying illness leading to HSCT was leukemia (19/41; 46%) followed by solid tumour (7; 17%), inborn errors of metabolism and Fanconi Anemia (4 each; 10%) and other (4;10%). The median length of study eligibility, defined as the point at which the patient returned to the IWK from the transplant centre until the time of central line removal, was 70 days (range 7–176). There were 316 patient contacts for surveillance cultures (mean 7 per transplant) and 577 central line lumens sampled. Three patients had potentially clinically significant surveillance blood cultures that were positive (3/43; 7%). The bacteria isolated were Klebsiella pneumoniae (n=2) and Corynebacterium jeikium (n=1). Repeat cultures were done prior to initiation of antimicrobial therapy and all were sterile. Patients were admitted for antimicrobial therapy if they were not already hospitalized for HSCT care. Median hospitalization for treatment of the positive culture was 5 days. All three patients had an uncomplicated course. The estimated total cost of BSI surveillance and treatment of asymptomatic infection over 6 years was $27,989. Conclusion: This study suggests that BSI surveillance in children post engraftment has a very low yield and significant cost. It is unclear that it contributes to improved patient outcomes.

Background. Hurler’s syndrome is a lysosomal storage disease with devastating neurological sequelae if left untreated. The negative correlation between age and IQ is significant (r = −0.82, p< 0.0003). These children have a normal intelligence at birth but on average decline by 2 standard deviations within the first 2 years of life (represents 30 point decrease in IQ). The underlying abnormality is an absence of the enzyme alpha-L-iduronidase, which is available as a recombinant enzyme but does not cross the blood-brain barrier. Currently, bone marrow transplant (BMT) is the only way to prevent the expected neurological deterioration. It is believed that higher levels of enzyme activity are associated with better neurological outcomes. Case report: We report on a 4 year 6 month old boy who was diagnosed with Hurler’s syndrome at 9 months of age. At 15 months of age, he received a matched related transplant with a preparative regimen of cyclophosphamide, busulfan and low dose total body irradiation (300 cGy). In the post transplant period, he had some minor complications but no neurological insults. He received Cyclosporin A for GVHD prophylaxis, which was discontinued after 5 months. Initially there was complete engraftment and normal enzyme activity but this was temporary (See Table 1). Table 1 Alpha-L-iduronidase Chimerism % * believed to be secondary to assay problems Pretransplant 0 Post transplant Normal 14–41 nmol/h/mg protein Ideal > 90% 1 month 28 90 4 months 0 45 6 months 14 30 1 year 3 15 1 year 5 months 2 15 1 year 8 months 3 10 2 year 1 month 73* 15 2 year 6 months 4 10 3 year 2 months 3 10 He had an excellent clinical response to BMT with softening of the characteristic facial features and resolution of airway obstruction. Prior to BMT he was report to be developing normally. He sat at 61/2 months, stood at 7 month, had single words at 10 months, and walked at 13 months. Despite mixed chimerism and sub-optimal enzyme activity, his development since BMT has been impressive. 2 years post-BMT (age 3 yr 3 mo), he underwent a formal neuropsychological evaluation. Full Scale intelligence testing revealed he had average intelligence (45th percentile), with a discrepancy between his verbal and performance intelligence. His language skills were within normal limits. He was reported to have some difficulties with visual spatial testing (in block design). Clinical assessment, at this time, revealed that he was doing well in school and had been fully toilet trained. At his last assessment at age 4 yr 5 mo (3 yr 2 mo post BMT), he had made dramatic improvements on the Peabody Developmental Motor Skills, with an age equivalent of 41 months. This was likely hampered somewhat by flexion contractures of his fingers. He continued to do well in school and was speaking both English and French. He had clinically normal development for age. Formal neuropsychological tests will be presented in poster format. Conclusions : This case is the first in the literature with halting of the characteristic neurological decline in Hurler’s syndrome despite poor engraftment and low enzyme activity following BMT. Full donor chimerism and normal alpha-L-iduronidase expression may not be needed to ameliorate the devastating consequences of Hurler’s syndrome.