Pediatric Blood & Cancer最新文献

SARS-CoV-2 seroprevalence reflects the efficacy of pandemic infection prevention and control measures. We performed anti-spike IgG serological testing on residual sera of children 1-11 years old at a tertiary care referral center between October and November 2021. Immunocompromised patients had the highest SARS-CoV-2 seroprevalence, at 40.5%, compared to 19.3% in non-immunocompromised patients. Targeted infection prevention and public health interventions are warranted for pediatric immunocompromised patients in future pandemics.

Background: Hemophilia A (HA) is an X-linked bleeding disorder diagnosed by a deficiency in factor VIII (FVIII). For severe HA (SHA), prophylaxis clotting factor concentrates (CFC) has become the standard of care; however, it imparts a high treatment burden and typically results in an annualized bleeding rate (ABR) of 2-6. Emicizumab, a subcutaneously administered FVIII substitute, has become the de facto standard-of-care prophylaxis for children with SHA in many countries. Previous clinical trials of emicizumab have assessed ABR in patients greater than 12 years without inhibitors, and in children less than 12 years with inhibitors; however, there is little information published regarding the ABR of emicizumab compared to CFC in non-inhibitor SHA children.

Methods: Using a retrospective electronic medical record chart review, we conducted a self-control analysis of 15 patients less than 12 years of age during equivalent periods of CFC versus emicizumab prophylaxis.

Results: The mean ABR on CFC and emicizumab was 1.79 and 1.13 (p = .092), respectively, with a substantially decreased rate of joint bleeds (CFC 0.94; emicizumab 0.33; p = .001) and spontaneous bleeds (CFC 0.79; emicizumab 0.23; p = .008). No safety events were recorded for patients while administering emicizumab. The mean annual cost of CFC prophylaxis was $515,340 (SD $199,540), compared to $328,410 (SD $137,230) for emicizumab prophylaxis (p < .001).

Conclusion: Emicizumab resulted in an improved ABR compared to CFC, especially for joint and spontaneous bleeds, had fewer administration complications, and was substantially less expensive compared to CFC prophylaxis; however, more research is necessary for a complete understanding of the effect of emicizumab on joint health and muscle bleeds.

Pediatric surgeons engaged in oncology will inevitably treat patients receiving palliative care, but their role in this context is poorly described. This article identifies some of the challenges and opportunities of surgical involvement in pediatric oncology palliative care, underscoring how the surgeon's expertise can be exploited to significantly benefit children with cancer. Specific examples of skills (procedural, communication, and coordination) that surgeons can provide to the multidisciplinary palliative care teams are described and the importance of collaboration is highlighted.

Background: Thrombocytopenia is a common hematologic finding in children and adolescents. Immune thrombocytopenia (ITP) is the most common cause of this finding, but the differential diagnosis includes a growing list of genetic disorders. We aimed to report differences in phenotypes of patients with ITP, inherited platelet disorder (IPD)/primary immunodeficiency disorder (PID), and other causes, with a focus on differentiating ITP from inherited thrombocytopenia.

Procedure: This retrospective, population-based observational cohort from 2006 to 2020 involved 506 Finnish children under 16 years of age presenting with isolated thrombocytopenia.

Results: Of the 506 participants, 79.7% had ITP, 6.7% had IPD/PID, and 13.6% had other causes of thrombocytopenia. A platelet count of ≤12 × 10⁹/L best distinguished between ITP and other reasons with a sensitivity of 60% and a specificity of 80%. Among patients with the lowest platelet count of less than 10 × 10⁹/L, 95.9% had ITP, 3.3% had IPD/PID, and 0.8% had other causes. Severe bleeding events were reported in 20 patients (4.0%), but there were no cases of intracranial or fatal bleeding due to thrombocytopenia. Up to 50% of patients with a high suspicion of inherited thrombocytopenia remained without a specific diagnosis despite genetic testing.

Conclusions: ITP remains the most common cause of thrombocytopenia. A platelet count of ≤12 × 10⁹/L often leads to an ITP diagnosis. Genetic disorders are rare but should be suspected in patients with persisting thrombocytopenia, especially with platelet counts constantly above 12 × 10⁹/L, a positive family history, or atypical clinical features.

Background: Optimal nutrition in pediatric oncology can influence cancer-related outcomes. To establish an understanding of nutrition practice and perceptions of best practice, we queried nutrition providers practicing in pediatric oncology care centers in high-income countries.

Methods: An electronic, multidisciplinary, cross-sectional survey of nutrition practices was conducted among pediatric oncology nutrition practitioners. Final analysis included 110 surveys from 71 unique institutions and included practitioners from Europe, the United States, Canada, Australia/New Zealand, South America, and the Middle East/Asia.

Results: The majority of institutions (97%) reported having dietitians; 72% had designated oncology dietitians. Approximately half of the practitioners (47%) reported feeling their institutions were inadequately staffed. The majority (78%) of institutions completed nutrition risk screening, but there was no consensus on specific screening practices. Half (50%) of the institutions that screened for nutrition risk did so in both inpatient and outpatient settings. The majority (80%) of institutions completed a nutrition assessment close to the time of diagnosis. Those that did not cite lack of staff and/or lack of time, lack of standardized approach, and consult only level of nutritional care as primary barriers. The most common topic of nutrition education provided to patients/families was nutrition-related symptom management (68%).

Conclusion: While most institutions reported having pediatric oncology dietitians, we found a lack of standardized practice and perceived inadequate staffing. In addition, what providers perceived to be best practice did not always align with day-to-day clinical practice. Ongoing efforts are needed to develop evidence-based guidelines, including staffing recommendations, to support specialized care in this population.

Objectives: To investigate whether (cluster of differentiation) CD40-1C>T (rs1883832) contributes to predisposition and treatment response of primary immune thrombocytopenia (pITP) in children.

Methods: A case-control study that included 100 children with newly diagnosed pITP and 50 age- and sex-matched healthy controls. CD40 rs1883832 was genotyped using TaqMan allele discrimination real-time polymerase chain reaction (PCR). Patients were categorized into responders and non-responders according to their response to corticosteroids and thrombopoietin-receptor agonists (TPO-RA) at 3-month intervals.

Results: The genotypic distribution of the CD40 rs1883832 was significantly different among cases and controls (CC 48% vs. 30%; CT 44% vs. 42%; TT 8% vs. 28%; p = .003). Compared with controls, children with newly diagnosed pITP had significantly higher C allele frequency (70% vs. 51%; odds ratio [OR] 2.2, 95% confidence interval [CI]: 1.3-3.8; p = .001). The association between C allele frequency and pITP risk was evident in females (OR 4.3, 95% CI: 2.1-8.8; p < .001), but not in males (OR 0.9, 95% CI: 0.4-2.1; p = .822). Compared with responders, the C allele frequency was significantly higher among non-responders to corticosteroids (87% vs. 66%; OR 3.4, 95% CI: 1.2-11.7; p = .012), but not to TPO-RA (92% vs. 85%; OR 2, 95% CI: 0.2-107; p = .550).

Conclusion: CD40 rs1883832 polymorphism may contribute to predisposition and response to upfront corticosteroids therapy of pediatric pITP. These findings improve our understanding of the compound pathophysiology of ITP, suggest important clinical potentials, and open the door for further research on the mechanistic role of CD40 rs1883832 in ITP development and progression.

Background: Optic pathway gliomas (OPG) are rare tumors in children. Lesion extent, visual functions, neurofibromatosis 1 (NF1), and age are factors that guide treatment. This study evaluates the clinical characteristics, treatment, and outcome of children and adolescents with OPG treated over a 31-year period in a single center.

Methods: Ninety-five patients with OPG diagnosed between January 1990 and December 2021 were retrospectively evaluated. First-line chemotherapy regimen consisted of vincristine and carboplatinum for 1 year. Radiotherapy was not used as first-line treatment and tried to be avoided in the ones who progressed after first-line treatment.

Results: Ninety-five children (44 male, 51 female) with a median age of 52 (1-216) months were evaluated. Sixty-three (66.3%) had NF1 and 10 (10,5%) diencephalic syndrome. The most common presenting symptoms were visual abnormalities and/or proptosis, nistagmus, and behavioral changes. Twenty-one (22.1%) patients with NF1 had stable disease throughout the follow-up period and received no treatment. Sixty-three of 74 patients received treatment at diagnosis and 11 due to progression during follow-up. Only one adolescent received radiotherapy at progression. Patients who progressed, received further line systemic treatment (vinblastine; bevacizumab; vincristine-cisplatinum-etoposide). Ten-year overall survival in all patients, in patients with NF1, and without NF1 were 97.2%, 98%, and 95.8% (p > .05), respectively; 10-year progression-free survival (PFS) in all patients, in patients with NF1, and without NF1 were 71.6%, 85.7%, and 54.2% (p = .001), respectively.

Conclusions: In children with symptomatic/progressive OPG, chemotherapy consisting of vincristine-carboplatinum (VC) is effective. Radiotherapy may be avoided, especially in patients with NF1.