This strategy of brief intensive chemotherapy for young children with non-metastatic medulloblastoma eliminated the need for craniospinal irradiation 52% of the patients, and may preserve QoL and intellectual functioning. The excellent survival rates are somewhat dampened by high toxic mortality.

A significant proportion of children with malignant brain tumors can avoid radiotherapy and prolonged maintenance chemotherapy yet still achieve durable remission with this brief intensive chemotherapy regimen.

BACKGROUND Convection-enhanced delivery of chemotherapeutics for the treatment of malignant glioma is a technique that delivers drugs directly into a tumor and the surrounding interstitium through continuous, low-grade positive-pressure infusion. This allows high local concentrations of drug while overcoming the limitations imposed by toxicity and the blood-brain barrier in systemic therapies that prevent the use of many potentially effective drugs. OBJECTIVE To examine the safety profile of a conventional chemotherapeutic agent, topotecan, via convection-enhanced delivery in the treatment of recurrent malignant gliomas and secondarily to assess radiographic response and survival. METHODS We performed a prospective, dose-escalation phase Ib study of the topoisomerase-I inhibitor topotecan given by convection-enhanced delivery in patients with recurrent malignant gliomas. RESULTS Significant antitumor activity as described by radiographic changes and prolonged overall survival with minimal drug-associated toxicity was demonstrated. A maximum tolerated dose was established for future phase II studies. CONCLUSION Topotecan by convection-enhanced delivery has significant antitumor activity at concentrations that are nontoxic to normal brain. The potential for use of this therapy as a generally effective treatment option for malignant gliomas will be tested in subsequent phase II and III trials.

Although this trial did not report a benefit of inhalation aromatherapy for reducing anxiety, nausea, or pain when added to standard supportive care, it provides the first experimental rather than descriptive report on testing a single therapeutic essential oil among children and adolescents undergoing stem cell infusion. Future research may consider exploring the cutaneous application of essential oil through massage or other psychoeducational counseling interventions among parents with elevated anxiety and patients with greater information seeking coping styles during SCT.

Sickle cell disease (SCD) is a hereditary hemoglobinopathy that affects over 100,000 people in the United States. Patients with SCD are known to experience suboptimal health-related quality of life (HRQoL). In addition to the physical manifestations of SCD, psychological and social stress, along with academic difficulties, secondary to the chronicity of the disease and its complications often affect patients with SCD. Although medical therapy of SCD has improved, allogeneic hematopoietic cell transplantation (allo-HCT) remains the only curative therapy. The objective of this study was to measure HRQoL before and after allo-HCT by assessing physical, psychological, and social functioning in patients with SCD who have undergone reduced-toxicity conditioning (busulfan/fludarabine/alemtuzumab) followed by allo-HCT. Patients < 21 years of age undergoing allo-HCT (matched siblings and unrelated donors) for SCD and their primary caregiver were enrolled using either the English or Spanish version of the PedsQoL 4.0. Data were collected at 3 time points: before allo-HCT and on days 180 and 365 after allo-HCT. The change in HRQoL from baseline was assessed with unadjusted and adjusted mixed-effects models in which subjects were treated as random effects, and variance component structure was used. Seventeen patients and 23 primary caregivers were enrolled and reported a mean overall HRQoL of 66.05 (SD, 15.62) and 72.20 (SD, 15.50) at baseline, respectively. In the patient-reported analysis with adjusted mixed-effects models, the estimated improvements in overall HRQoL were 4.45 (SE, 4.98; P = .380) and 16.58 (SE, 5.06; P = .003) at 180 and 365 days, respectively, after allo-HCT. For parent-reported overall HRQoL, the estimated improvements were 1.57 (SE, 4.82; P = .747) and 9.28 (SE, 4.62; P = .053) at 180 and 365 days, respectively, after allo-HCT. Similar results were found across the physical, social, and emotional HRQoL domains with mixed-effects models after adjustment of demographic and medical variables. In addition to the alleviation of clinical manifestations of SCD, these patients demonstrated significant improvement in most aspects of HRQoL by 1 year after allo-HCT. These data represent the trajectory of HRQoL during the initial year of follow-up within this population and should be integrated into the decision-making process when considering allo-HCT in patients with SCD.

Objective Providing care to one’s child during and after a hematopoietic stem cell transplant (HSCT) is a universally stressful experience, but few psychological interventions have been developed to reduce caregiver distress. The goal of this study was to test the efficacy of a brief cognitive–behavioral intervention delivered to primary caregivers. Method Two hundred eighteen caregivers were assigned either best-practice psychosocial care (BPC) or a parent social-cognitive intervention program (P-SCIP). The 5 session P-SCIP was delivered during the HSCT hospitalization. Caregivers completed measures of distress, optimism, coping, and fear appraisals preintervention, 1, 6 months, and 1 year. Results P-SCIP reduced caregiver’s distress significantly more than BPC between the pretransplant assessment (Time 1) and 1-month follow-up assessment (Time 2). P-SCIP had a stronger effect than BPC among caregivers who began the hospitalization reporting higher depression and anxiety, and among caregivers whose children developed graft-versus-host disease (GvHD). Long-term treatment effects of P-SCIP were seen in traumatic distress among caregivers who reported higher anxiety pretransplant as well as among caregivers whose children had GvHD at HSCT discharge. Conclusions Screening caregivers for elevations in pretransplant anxiety and targeting interventions specifically to these caregivers, as well as targeting caregivers to children who develop GvHD, may prove beneficial.

Induction, with or without intensification using intravenous methotrexate, followed by myeloablative consolidation chemotherapy with AuHCT, may avoid or delay CSI, with possible stabilization of neuropsychological functioning, including those younger at diagnosis. Continued follow-up is necessary to determine the preservation of neuropsychological, academic, social-emotional and behavioral functioning.

Background Although corticosteroids remain a mainstay of treatment for acute lymphoblastic leukemia (ALL), they can cause troublesome neurobehavioral changes during active treatment, especially in young children. We evaluated acute neurobehavioral side effects of corticosteroid therapy in preschool versus school-age children by obtaining structured reports weekly for one month. Procedure Parents of 62 children (2 to 17 years at diagnosis) treated on Dana-Farber Cancer Institute (DFCI) ALL Consortium Protocol 00-01 participated during the continuation phase of treatment. Patients received cyclical twice-daily 5-day courses of prednisone (40 mg/m2/day) or dexamethasone (6 mg/m2/day). Parents completed behavior rating scales about their child weekly during one steroid cycle [baseline (Day 0), active steroid (Day 7), post-steroid (Days 14 and 21)]. Results Behavioral side effects increased significantly (p < .001) during the steroid week for preschool children (< 6 years) on measures of emotional control, mood, behavior regulation, and executive functions, returning to baseline during the two ‘off-steroid’ weeks. In contrast, school-age children (≥ 6 years) did not demonstrate an increase in side effects during the steroid week. Steroid type (prednisone vs. dexamethasone) was not a significant predictor of neurobehavioral side effects. Conclusions Preschool children are at greater risk for neurobehavioral side effects during active steroid treatment for ALL than school age children and adolescents. Dexamethasone was not associated with more neurobehavioral side effects than prednisone. Counseling of families about side-effects should be adapted according to age. The observed effects, moreover, were transient, reducing concerns about longer-term neurobehavioral toxicities.