Pediatric Blood & Cancer最新文献

Recordings of patient-doctor interactions is a recommended method in communication research. However, concerns are expressed regarding audio-recording of conversations with vulnerable patients. Our study examined experiences of children, parents, and oncologists with recording diagnostic conversations in the pediatric acute leukemia setting. Results show that recording conversations is generally well received by virtually all children and parents. Pediatric oncologists seem to overestimate the expected emotional burden for children and parents, which may lead to gatekeeping by professionals. This in turn may lead to a decrease in patient autonomy and research quality when addressing relevant questions in communication science.

A 3-week-old neonate with glucose-6-phosphate dehydrogenase (G6PD) deficiency and primary lactic acidosis developed haemolytic jaundice and methaemoglobinaemia following treatment with dichloroacetate (DCA), a standard treatment for primary lactic acidosis. While this mechanism has been reported in the sheep model, it has not been described in humans. Our case reinforces the uncommon observation that a G6PD-deficient individual experiencing oxidative stress may develop concurrent methaemoglobinaemia. In this case, methylene blue, the standard treatment for methaemoglobinaemia, may result in further oxidative stress. The judicious use of blood transfusion to correct the oxygen-carrying capacity of our patient led to reversal of the methaemoglobinaemia.

Objective: To describe treatments and outcomes of French children treated for relapsed/refractory anaplastic lymphoma kinase-positive anaplastic large cell lymphoma (ALK+ ALCL).

Methods: We conducted the analysis of a series of 75 French children treated for a first relapsed/refractory ALK+ ALCL between 1999 and 2017.

Results: The median time to first relapse was 8.1 months from initial diagnosis (2.9 after end of treatment), with 12 relapses during frontline treatment or within 1 month of the end of treatment. Treatment of the first relapse varied according to the period of time and risk factors: 48 received multiagent chemotherapy, including 21 and 19 consolidated with allogeneic stem cell transplantation (SCT) and autologous-SCT, respectively. Twenty-one patients received weekly vinblastine, and six received ALK inhibitors (ALKi). Overall, 64/75 patients reached a second complete remission (CR2). Eight out of 11 patients who did not reach CR2 died and the other three were rescued with ALKi, vinblastine, and nivolumab. With a median follow-up of 8.2 years, 60 patients are alive, 43 in CR2, 15 in CR3, two in CR4; and 15 patients died, six from toxicity and nine from disease progression. The 5-year event-free survival and overall survival after first relapse were 51.7% (95% confidence interval [CI]: 39.6%-62.6%) and 80.7% (95% CI: 69.6%-88.1%), respectively. Time to relapse greater than 12 months from initial diagnosis was proven to be a prognostic factor in relapsed/refractory ALK+ ALCL.

Conclusion: In relapsed ALK+ ALCL, high survival rate can be reached with various therapeutic strategies. The main challenge remains to prevent subsequent relapses, and to lower long-term morbidity.

Background: In recent decades, new first and subsequent lines of anticancer treatment and supportive care have improved survival for children with cancer. We investigated recent temporal changes in the incidence of relapse and survival after relapse among children with cancer in Denmark.

Procedure: This register-based study included 2890 children diagnosed before age 15 years with haematological cancers and solid tumours (2001-2021) and central nervous system (CNS) tumours (2010-2021). We used the Aalen-Johansen and Kaplan-Meier estimators to assess cumulative incidence of relapse-defined as cancer recurrence or progression-and survival probability after relapse.

Results: Comparing the periods 2001-2010 and 2011-2021, the 5-year cumulative incidence of relapse decreased from 14% to 11% among children with haematological cancers (p = .07), and from 21% to 18% among children with solid tumours (p = .26). Concurrently, the 5-year survival after relapse increased among children with haematological cancers (from 44% to 61%, p = .03) and solid tumours (from 38% to 46%, p = .25). Among children with malignant CNS tumours, the 5-year cumulative incidence of relapse and the 5-year survival after relapse remained stable (49% and 51%, p = .82; and 20% and 18%, p = .90) comparing 2010-2015 and 2016-2021.

Conclusions: In recent decades in Denmark, improvements were observed in reducing relapse incidence and increasing survival after relapse in children with haematological cancers and solid tumours. However, the persistent survival gap between children who relapse and those who do not across all childhood cancers underlines the need for intensified and highly targeted treatments for children at high risk of relapse.

Background: Pediatric palliative care (PPC) is associated with improved end-of-life (EOL) outcomes. Inpatient and outpatient PPC have unique roles during the disease course. Yet, it is unknown whether the location of PPC receipt (inpatient vs. outpatient) is associated with healthcare utilization and EOL outcomes for pediatric and adolescent and young adult oncology patients.

Procedure: A retrospective single-institution chart review of pediatric patients (age 0-28) with cancer who died between January 2015 and December 2022 was performed to compare EOL outcomes and healthcare utilization metrics among inpatient PPC, any outpatient PPC, and non-PPC recipients. Demographics and clinical factors were analyzed by PPC receipt location.

Results: Among 450 patients, 292 (64.9%) received PPC (inpatient only 35%, any outpatient 65%). Patients who died without receiving PPC dropped from 69% to 22% following development of an outpatient PPC clinic (p < .001). In the last 6 months, 1 month, and last week of life, inpatient PPC recipients spent more days admitted to the hospital and intensive care unit (all p < .001), and had more intensive medical interventions performed (p < .01). Outpatient PPC recipients were less likely to receive intravenous (IV) chemotherapy (p < .01) or intubation (p = .05), and more likely to receive hospice, die at home, and have an outpatient do-not-resuscitate order (all p < .001).

Conclusions: PPC receipt substantially increased after the creation of an outpatient PPC clinic, suggesting that outpatient PPC is critical in the provision of PPC to children with cancer. Outpatient PPC was associated with fewer hospital days, IV chemotherapy, and intubation at EOL, while increasing hospice enrollment and home death.

Background: The Children's Oncology Group (COG)-AALL0434 trial investigated the addition of nelarabine to the augmented Berlin-Frankfurt-Münster (aBFM) protocol in patients (1.0-30.99 years) with newly diagnosed T-cell acute lymphoblastic leukemia (T-ALL). Despite demonstrating superior outcomes, nelarabine is not currently funded by many health systems, in part due to a lack of cost-effectiveness data. We estimated the cost-utility of nelarabine for this indication from a Canadian public healthcare payer perspective.

Methods: We developed a microsimulation model that followed hypothetical patients with newly diagnosed T-ALL from post-induction therapy to death. Three health states were modeled: relapse-free, post-relapse, and death. Efficacy was estimated using AALL0434 and retrospective data from Ontario, Canada. Costs were obtained from Canadian sources. Utility estimates and long-term mortality risks were sourced from literature. Total healthcare costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratio (ICER) were reported. Probabilistic and scenario analyses were conducted.

Results: Incorporating nelarabine in the aBFM protocol increased costs by $51,670 Canadian dollars per patient, but resulted in 1.97 more QALYs and an ICER of $26,184/QALY. Most of the identified cost and benefit were accrued within the AALL0434 trial period (first 11 years post diagnosis) and while patients were in the relapse-free health state. Across multiple scenarios, the ICER was stable under an assumed $50,000/QALY threshold.

Conclusion: Incorporating nelarabine into aBFM was cost-effective across different scenarios and assumptions. These results support its funding by public and private payers.

Background: The ability of transcranial Doppler (TCD) to detect asymptomatic cerebrovascular disease among childhood brain tumor survivors following exposure to cranial radiation therapy has not been established.

Methods: Survivors of childhood brain tumors, more than 3 years since diagnosis and exposed to greater than 30 Gy cranial radiation, underwent a history and physical exam, laboratory biomarkers of cerebrovascular disease (cholesterol, high-density lipoprotein cholesterol (HDL), low-density lipoprotein cholesterol (LDL), high-sensitivity CRP, hemoglobin A1C, apoprotein A, and apoprotein B), and a TCD evaluation of their cerebral arteries.

Results: In all 165 cerebral arteries from 13 patients (medulloblastoma = 10; germ cell tumor = 3; females = 5; mean age at diagnosis = 8.0 years; mean age at time of study = 20.9 years) were examined. Twenty-eight of 165 (17%) were considered abnormal by pre-specified criteria. Total 114 cerebral arteries from 13 patients were assessed for greater than 50% stenosis velocities. Arteries most likely to be considered abnormal included the distal bilateral vertebral arteries (right 38%, left 30%), basilar artery 30%, bilateral siphon internal carotid arteries (right 30%, left 23%), bilateral middle cerebral arteries (23% bilaterally), and bilateral anterior cerebral arteries (7% bilaterally). Two vessels had mean flow velocities consistent with $\ge$ 50% stenosis (1.8%). No vessels were found to have greater than 80% stenosis.

Conclusions: TCD may be a useful and practical tool to examine asymptomatic cerebrovascular disease among childhood brain tumor survivors after exposure to cranial radiation therapy. Posterior circulation vessels appear to have the highest burden of disease in this group of brain tumor survivors, a majority of whom had medulloblastoma.