Patterns of surveillance for late effects of BCR-ABL tyrosine kinase inhibitors in survivors of pediatric Philadelphia chromosome positive leukemias.

BACKGROUND: Targeted anticancer therapies such as BCR-ABL tyrosine kinase inhibitors (TKIs) have improved outcomes for chronic myeloid leukemia (CML) and Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph + ALL). However, little is known about long-term risks of TKIs in children. Exposure-based survivorship guidelines do not include TKIs, thus surveillance practices may be variable. METHODS: We retrospectively examined surveillance for cardiac and endocrine late effects in children receiving TKIs for Ph + leukemias, diagnosed at < 21 years between 2000 and 2018. Frequency of echocardiogram (ECHO), electrocardiogram (EKG), thyroid stimulating hormone (TSH), dual-energy x-ray absorptiometry (DXA), and bone age testing were abstracted. Descriptive statistics were stratified by leukemia type. RESULTS: 66 patients (CML n = 44; Ph + ALL n = 22) met inclusion criteria. Among patients with CML, ≥1 evaluation was done: ECHO (50.0%), EKG (48.8%), TSH (43.9%), DXA (2.6%), bone age (7.4%). Among patients with Ph + ALL, ≥1 evaluation was done: ECHO (86.4%), EKG (68.2%), TSH (59.1%), DXA (63.6%), bone age (44.4%). Over a median 6.3 and 5.7 years of observation, respectively, 2% of patients with CML and 57% with Ph + ALL attended a survivorship clinic. CONCLUSIONS: Despite common exposure to TKIs in survivors of Ph + leukemias, patterns of surveillance for late effects differed in CML and Ph + ALL, with the latter receiving more surveillance likely due to concomitant chemotherapy exposures. Targeted therapies such as TKIs are revolutionizing cancer treatment, but surveillance for late effects and referral to survivorship clinics are variable despite the chronicity of exposure. Evidence based guidelines and longer follow-up are needed.

Features Associated With Weight Loss and Growth Stunting for Young Children During Cancer Therapy.

Features associated with malnutrition are poorly elucidated in pediatric cancer care. We aimed to better understand characteristics associated with weight-for-height (WHZ) and height-for-age (HAZ) changes for infants and young children during cancer treatment. This retrospective study included 434 patients diagnosed <3 years old from 2007 to 2015 at a large pediatric cancer center. Patients starting treatment outside our center, those with relapsed or secondary malignancies, or with inaccurate information were excluded. Abstracted weights and heights for a 24-month period after treatment initiation were converted to sex-specific and age-specific z scores. Although not statistically different at baseline, patients with hematologic malignancies gained weight over time, while other tumor types did not. Higher treatment intensity and younger age at diagnosis increased odds of clinically significant weight loss. Older children had higher HAZ at diagnosis and HAZ also significantly decreased over time for all examined risk factors, which is distinctly different from patterns in WHZ over time. In conclusion, WHZ and HAZ are affected differently by cancer treatment in infants and young children. We identify key risk factors for weight loss and growth stunting which will be necessary to develop prospective trials to examine anthropometric, biochemical, and patient recorded outcomes around nutrition.

Very late recurrence of B-cell acute lymphoblastic leukemia masquerading as a pituitary tumor.

Involvement of the pituitary gland by leukemic infiltration is exceedingly rare. Here, we describe a very late recurrence of B-cell acute lymphoblastic leukemia masquerading as a pituitary tumor and review the literature for previously reported cases. Our female patient presented 13 years after completion of therapy for B-ALL with headache, amenorrhea, galactorrhea and a pituitary mass. Subsequent studies revealed recurrence of her leukemia, and the pituitary lesion resolved after induction chemotherapy. Our case highlights the importance of considering leukemic infiltrate in the differential diagnosis of pituitary mass, particularly in a patient with a history of hematologic malignancy, sparing unnecessary surgical intervention and informing endocrine evaluation. In addition, the case also highlights difficulties with characterizing this recurrence as a very late relapse or clonal evolution of the original leukemia.

Management of chronic myeloid leukemia in children and adolescents: Recommendations from the Children's Oncology Group CML Working Group.

Chronic myeloid leukemia (CML) accounts for 2-3% of leukemias in children under 15 and 9% in adolescents aged 15-19. The diagnosis and management of CML in children, adolescents, and young adults have several differences compared to that in adults. This review outlines the diagnosis and management of the underlying disease as well as challenges that can occur when dealing with CML in this patient population.

Assessment of ovarian function in adolescents and young adults after childhood cancer treatment-How accurate are young adult/parent proxy-reported outcomes?

BACKGROUND: Providers often rely on self-reported ovarian function in adolescent and young adult (AYA)-aged childhood cancer survivors when making clinical decisions. This study described reported menstrual patterns and the agreement between respondent-reported and biochemical premature ovarian insufficiency (POI) in this population. PROCEDURE: This was a cross-sectional study of survivors (or their parent proxy) aged 13-21.9 years who received gonadotoxic therapy and were enrolled in a longitudinal health survey. Participants reported menstrual regularity, hormone-replacement therapy (HRT) use, and ovarian dysfunction. Respondent-reported POI was defined as the survivor taking HRT for ovarian failure or having been told she had ovarian failure. Biochemical POI was defined as follicle-stimulating hormone (FSH) level ≥40 mIU/mL. The agreement between respondent-reported and biochemical POI was determined using Cohen's kappa coefficient (κ) and analyzed by demographic and clinical factors. RESULTS: Among 182 AYA-aged survivors (72.5% non-Hispanic White, 46.7% leukemia survivors), 14.8% reported requiring HRT to have menses but 55.5% reported regular menses without HRT use. Among survivors with FSH measurements (n = 130), 17.7% reported POI whereas 18.5% had FSH ≥40 mIU/mL (κ = 0.66, sensitivity 70.8%, specificity 94.3%). The highest agreement between respondent-reported and biochemical POI was with young adult self-report (κ = 0.78) and survivors with >5 survivor clinic (κ = 0.83) and/or >5 endocrinologist (κ = 1.00) visits. CONCLUSIONS: The majority of AYA-aged survivors reported having regular menses without HRT support. The accuracy of respondent-reported POI increased with repeated survivor clinic or endocrinologist visits, highlighting the importance of continued education. Survivors must be informed about their ovarian function to enable them to advocate for their reproductive health.

Guidelines for Treatment and Monitoring of Adult Survivors of Pediatric Brain Tumors.

OPINION STATEMENT: Pathologies of pediatric brain tumors are more varied than those diagnosed in adults and survival outcomes more optimistic. Therapies for pediatric brain tumors are also diverse and treatment options are expanding. The growing number of adult survivors of childhood brain tumors is quite diverse. Medical management of these adults requires understanding the tumor diagnosis and location, the modalities used to treat the tumor, the age of the survivor at the time of diagnosis and treatment, any complications of treatment, and, most importantly, the baseline medical condition and neurological function of each adult survivor. A network of medical, neurological, and mental health providers is critical in the care of a child with a brain tumor. A comparable network should be available to survivors of these tumors since they may transition to adulthood with medical and neurological deficits and can acquire additional late effects of treatments as they age. Optimally, each survivor will have an individualized survivor health plan (SHP) that concisely summarizes the tumor, treatments, potential late effects, and screening that may identify evolving late effects before they impact mental, social or physical functioning. This plan helps patients, families, and the medical team advocate for surveillance aiming to optimize the survivor's quality of life. Failure to support the health and function of these heroic cancer survivors renders the medical advances, the courage, and the struggle that permitted survival meaningless.

Perceptions of body mass index (BMI) in pediatric cancer survivors and their providers.

BACKGROUND: Abnormalities in BMI are well documented in childhood cancer survivors. Perceptions of BMI status in cancer survivors have been understudied. This study determines the accuracy of parent/survivor and provider reporting of BMI status in a cancer survivor program. PROCEDURE: This is a retrospective study. Parent/survivor assessment of BMI status was obtained from a baseline questionnaire of subjects enrolled in Children's Healthcare of Atlanta-Childhood, Adolescent, and Young Adult Cancer Survivor Study (CHOA-CAYACSS). Provider reporting of BMI was obtained from a clinic visit close in date to completion of the survey. Perceptions of BMI were compared to actual BMI status calculated from clinic visits and categorized based on the Centers for Disease Control and Prevention (CDC) BMI guidelines. RESULTS: Perceptions of BMI were collected from 290 survivors of pediatric cancer or their parents (range, 4.3-22.9 years). Nearly 5% of survivors were underweight, 19.7% overweight and 16.2% obese. High BMI was the BMI state least likely to be correctly identified by parents, survivors, and providers. Among survivors with high BMI, parents, survivors, and providers failed to identify the problem 49.4%, 66.7%, and 26.9% of the time, respectively. Providers were less likely to correctly identify overweight compared to obese status (P < 0.0001). Accuracy of BMI recognition was independent of gender of survivor, ethnicity, or primary cancer diagnosis. CONCLUSION: Abnormal BMI states, especially overweight, are frequently not correctly perceived by parents/survivors or providers. Assessment of BMI status and discussion about steps to normalize BMI is needed to prevent weight related morbidities in this population.

Guidance for Pediatric Pituitary MRI Ordering: A Single Institution Improvement Initiative.

We identified inconsistencies in the pituitary MRI ordering practices at our pediatric institution. We used an interdepartmental collaboration to develop a pituitary MRI ordering guide based on available evidence and local expertise. The initiative has led to an improvement in the appropriate use of intravenous gadolinium-based contrast agents for pediatric pituitary MRI studies.

A Mixed-Methods Exploration of the Experience of People With Aphasia Using Text-to-Speech Technology to Support Virtual Book Club Participation.

PURPOSE: This mixed-methods research sought to examine the experience of people with aphasia who used text-to-speech (TTS) support to read a novel for virtual book club participation. METHOD: Six people with chronic aphasia used a TTS system to review portions of a novel about which they conversed during eight virtual book club meetings occurring over 5 weeks. During one-on-one interactions prior to each meeting, participants answered comprehension questions and provided feedback about reading experiences. Then, during group meetings, they reviewed and discussed relevant book content and predicted upcoming content. During a structured individual interview, participants reflected on their supported reading and book club experience. RESULTS: Participants reported a range of reading confidence prior to study participation, mostly influenced by decreased comprehension or reading speed. After book club participation, four participants expressed increased confidence. Some reported searching for key words and skipping difficult words as strategies additional to TTS support. All reviewed at least some book sections more than once either with or without TTS support. Highly motivated participants expressed low frustration and high reading ease and enjoyment. Perceived comprehension was roughly consistent with actual comprehension across participants. Most believed TTS support promoted faster reading than otherwise possible. Participants liked adjustable features affecting speech output rate, word or sentence highlighting, and font size. Psychosocial benefits included decreased isolation and increased friendship. CONCLUSIONS: The findings extend previous evidence about perceived and actual benefits associated with TTS support. People with aphasia express positive experiences when given TTS support during book club participation.