The chronic nature of chronic myeloid leukemia (CML) has a negative impact on patients’ health-related quality of life (HRQOL) and the need for prolonged oral tyrosine kinase inhibitors (TKIs) poses a risk to low or non-adherence overtime. This systematic review seeks to analyze HRQOL outcomes and adherence to TKIs among pediatric, adolescent and young adult (AYA) patients with CML. Full-text screening ultimately led to the inclusion of 12 articles focused on HRQOL, patient reported outcomes (PRO) and medication adherence. There was heterogeneity in the findings among the included studies, especially in HRQOL and PROs. TKIs adherence ranged from 45-78% using self-report and 58-100% using medical and pharmacy records. Increased TKI adherence was significantly associated with better clinical outcomes, including overall survival. Our findings could inform efforts to develop behavioral interventions to optimize TKI adherence and improve HRQOL outcomes among AYA patients with CML.

Background: Low 6-mercaptopurine (6-MP) adherence (<95%) is associated with increased relapse in pediatric acute lymphoblastic leukemia (ALL). Stronger habit has been associated with higher adherence. Our objective was to examine the relationship of 6-MP adherence to habit strength and health-related quality of life (HRQOL) in children with ALL. Methods: A single-center, cross-sectional study of 52 participants: 11 patients (mean age 16±4, 45% Female) and 41 parents/caregivers (age 37±5, 80% Female). Participants completed Visual Analogue Scale (VAS dose), Patient Reported Outcomes Measurement Information System (PROMIS) Medication Adherence Scale (PMAS), Self-Regulated Habit Index (SRHI), and PROMIS HRQoL measures. Descriptive statistics and spearman’s rho correlations were used. Eleven semi-structured interviews of participants were conducted and analyzed using Grounded Theory and thematic analysis. Results: Overall, 81% (42/52) of participants reported high 6-MP adherence (VAS dose ≥95%): patients 91% (10/11) and parents 78% (32/41). Higher adherence was correlated with better peer relationship scores (r s=0.34, P=0.03). No significant correlation was found between adherence and habit strength. Reported facilitators for higher 6-MP included reminders, care team communications, developing personalized tools, experience with medication administration, self-efficacy, and access to social support. On the other hand, financial burden, scheduling conflicts, and medication access were common barriers cited by participants. Conclusions: One-fifth of participants reported low 6-MP adherence, and habit strength wasn’t associated with adherence rates. Variability of 6-MP routines over treatment may prohibit automaticity. Behavioral interventions aimed at promoting habit formation could address barriers to 6-MP adherence and improve health outcomes in pediatric ALL.

Background: While intravenous fluid (IVF) therapy in patients with sickle cell disease (SCD) admitted for a vaso-occlusive episodes (VOE) can help reduce red blood cell sickling, clinical practice varies across institutions. We examined the relationship between IVF therapy and hospital length of stay (HLOS), as well as adverse events, such as acute chest syndrome (ACS), pediatric intensive care unit (PICU) transfer and 28-day readmission. Methods: This is a single-center retrospective analysis of SCD VOE hospitalizations between January 2015 and April 2020. Patients with SCD, age 0-30, with consecutive hospitalizations for VOE were included. For the first 3 days of each admission, an “IVF ratio” was calculated by dividing actual IVF rate administered by weight-based maintenance IVF (mIVF) rate. Results: A total of 617 hospitalizations for 161 patients were included. Mean HLOS was 5.7 days, (SD 3.9), and mean IVF volume over the first 3 days of admission was 139.6 ml/kg/day (SD 57.8). Multivariate analysis showed that for each additional 0.5 times the mIVF rate, HLOS increased by 0.53 days (P<0.001; 95% CI: 0.609–0.989), but there was no significant association between IVF therapy and adverse events. History of chronic pain was associated with increased odds of readmission (OR, 6.4; 95% CI: 3.93 – 10.52). Conclusions: Despite the theoretical potential for IVF therapy to slow down the sickling process, our findings suggest that increased IVF therapy was associated with prolonged HLOS, which places a burden on patients, families, and the health system.

Nutritional deficiencies such as iron, vitamin B12 and folate are recognized as etiologies for several cytopenias; although copper’s role in multiple metabolic enzymes is well-established, copper deficiency is often overlooked as a contributing entity. Frequently diagnosis is delayed, patients may undergo bone marrow investigations with findings overlapping a myelodysplastic process, which can lead to further testing and treatment considerations including hematopoietic stem cell transplant referral. We present a case of a young boy with cystic fibrosis with biliary dysplasia corrected with hepato-portoenterostomy and distal intestinal obstruction syndrome resulting in jejunal resection, with severe anemia and thrombocytopenia requiring transfusion support. Initial evaluation had been unremarkable, ongoing pancytopenia prompted bone marrow studies, which revealed vacuolated granulocytic and erythroid precursors and ring sideroblasts, suggestive of copper deficiency. Serum copper and ceruloplasmin were consistent with severe deficiency, attributed to insufficient absorption intestinal resection, chronic parenteral nutrition and prior zinc supplementation. Following enteral copper supplementation, anemia, leukopenia and thrombocytopenia significantly improved, however upon cessation, counts again worsened and has since been maintained on daily copper supplementation without further transfusion needs. Our experience exemplifies the importance of early consideration for copper deficiency in children with cytopenias, especially within context of intestinal malabsorption or inadequate nutritional intake which often occurs in children with cystic fibrosis.

Title PageTitle : Pancytopenia in a child with cystic fibrosis and severe copper deficiency: Insight from bone marrow evaluationAuthors : Maggie D. Seblani1,2; Susanna A. McColley2,3,4; Shunyou Gong5; Lee M. Bass2,6; Sherif M. Badawy1,2Affiliations : 1 Division of Hematology, Oncology and Stem Cell Transplant, Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL, USA; 2 Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, IL, USA; 3 Division of Pulmonary and Sleep Medicine, Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL, USA; 4 Stanley Manne Children’s Research Institute, Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, Illinois, USA; 5 Division of Pediatric Pathology, Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL; 6 Division of Gastroenterology, Hepatology and Nutrition, Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL.Correspondence: Sherif M. Badawy, MD, MS, MBBCh, 225 E. Chicago Ave., Box #30, Chicago, IL, 60611, office: 312-227-4836, fax: 312-227-9376, e-mail: sbadawy@luriechildrens.orgWord count: 498Number of Tables, Figures, and Supplemental files : 1Running title: Bone marrow findings with severe copper deficiencyKeywords: pancytopenia, anemia, leucopenia, neutropenia, thrombocytopenia, copper deficiency, ring sideroblasts, copper supplement, cystic fibrosis, malabsorptionConflict of Interest: Author has no conflicts of interest to disclose.

Infertility secondary to chemotherapy and/or myeloablative conditioning regimens prior to stem cell transplantation is an important cause of morbidity and psychosocial distress among pediatric cancer patients. Known options exist for fertility preservation; however, knowledge among providers varies. We conducted a pilot study with an educational intervention over one-hour for hematology-oncology faculty, fellows, and advanced practice providers. Participants completed pre-/post-test assessment on fertility preservation knowledge. Participants’ pre-test mean (SD) score was 53% (17%), which significantly increased to 72% (11%) in the post-test (P=0.0004). We demonstrated that a fertility education intervention could improve knowledge regarding infertility risk assessment and fertility preservation options.