Significant improvement of child physical and emotional functioning after familial haploidentical stem cell transplant.

Allogeneic stem cell transplantation (AlloSCT) represents the only curative therapy for sickle cell disease (SCD). However, limited availability of matched related donors and suboptimal outcomes following AlloSCT with unrelated donors has led to investigation of alternative donors. Among children with high-risk SCD, we evaluated health-related quality of life (HRQoL) impact in the two years following familial haploidentical SCT. HRQoL was collected from parent and child raters, using the Child Health Ratings Inventories Generic measure and haploidentical SCT-specific module. Repeated measures models were fit to assess HRQoL changes over time and by rater. Nineteen children (mean age 12.9 yrs [standard deviation, 5.3]; 63% male) and their parents were included. There were no differences in the 2-yr trajectories of child physical or emotional functioning (EF) by rater. Child physical functioning and EF scores were significantly lower at day +45 than baseline, but scores recovered by day +180. There was significant improvement in EF (p = 0.03) at 2 yrs vs baseline. A similar pattern of scores over time was seen for parent ratings of child's global HRQoL. Despite treatment intensity in the initial months following AlloSCT, patient scores recovered or exceeded baseline scores at two years. This trial is registered at clinicaltrials.gov (NCT01461837).

Perceptions of Role in Treatment Decision Making and Understanding of Late Effects Among Hodgkin Lymphoma Survivors: Results From a National Survey HL Decision Making and Late Effects.

BACKGROUND: Hodgkin Lymphoma (HL) survivors are at risk of treatment-related late effects (LEs). With these potential risks and increasing numbers of treatment options for newly diagnosed patients, communication and shared decision making are essential to supporting patients throughout the cancer care continuum. We aimed to gather perspectives of HL survivors about their actual role in treatment decision making and their understanding of LEs. MATERIALS AND METHODS: After initial pilot testing at a cancer survivor conference, we disseminated a 23-question survey in a single-wave e-mail through the Leukemia & Lymphoma Society's national listserv. We focused on 4 constructs: (1) patient's understanding of HL at diagnosis; (2) initial discussions with an oncologist; (3) factors in decision making of treatment, and (4) current health status. RESULTS: A total of 135 participants responded to the survey. While 73% of survey respondents perceived some involvement in decision making, one-half of respondents felt the treatment plan was a shared decision with their provider. Among patient-level factors, side effects/LEs were most frequently endorsed as important to treatment decisions. Eighty-four percent of respondents had been educated about risk for potential LEs. Thirty-six percent had been diagnosed with a LE at the time of survey completion with 3% reporting a second cancer diagnosis. CONCLUSION: Survey respondents described their role in treatment decision making for newly diagnosed HL. Nearly half of patients did not endorse participating in shared decision making. A substantial number had experienced LEs. Future work should focus on improving patient-provider communication in decision processes for newly diagnosed HL.

Assessment of patients' understanding of and adherence to oral anticancer medication (OAM): Results of a cross-sectional institutional pilot study.

BACKGROUND: Although oral anticancer medications (OAM) provide opportunity for treatment at home, challenges include prescription filling, monitoring side effects, safe handling, and adherence. We assessed understanding of and adherence to OAM in vulnerable patients. METHODS: This 2018 pilot study defined vulnerable patients based on Chinese language, older age (≥65 years), and subsidized insurance. All participants had a cancer diagnosis and were taking an OAM filled through the hospital's specialty pharmacy. Participants reported on OAM taking (days per week, times per day, special instructions) and handling (handling, storage, disposal). The specialty pharmacist classified patient-reported responses about OAM taking and handling as adequate or inadequate. OAM regimens were classified by complexity. RESULTS: Of 61 eligible patients, 55 participated. Mean age was 68 years (standard deviation [SD] = 12) and 53% were female. Patient subgroups were: 27% Chinese, 64% ≥65 years, and 9% subsidized insurance. Forty-nine percent were on frontline therapy and median time on OAM was 1 year (Quartile 1 = 0.4, Quartile 3 = 1.7). Adequacy of OAM taking (30%) and handling (15%) were low; 15% had adequacy in both. Adequacy of OAM taking and handling did not vary by patient subgroup or regimen complexity. Mean patient-reported adherence was high (5.4, SD = 1, possible range 1-6) and did not vary by adequacy of OAM taking or handling. CONCLUSIONS: Understanding of OAM taking and handling in this group of vulnerable patients was low and did not align with patient-reported adherence. Future interventions should ensure that patients understand how to safely take and handle OAM, thereby optimizing their therapeutic potential.

Enhancing patients' understanding of and adherence to oral anticancer medication: Results of a longitudinal pilot intervention.

BACKGROUND: Oral anticancer medications (OAM) make administration more convenient for patients, but shifts the responsibility of care from clinical providers to the patients themselves. Following an institutional pilot study showing inadequate understanding and adherence among vulnerable patients taking OAM, a longitudinal intervention was developed using an oncology specialty pharmacist and medication navigators to enhance OAM understanding and adherence. METHODS: Patients initiating OAM were approached for four formalized teaching and check-in sessions, supplemented with medication information sheets and individualized calendars. At each session, participants were assessed on their OAM understanding and adherence using teach-back and validated measures. A study evaluation elicited feedback from participants on the usefulness of the intervention. RESULTS: Of 80 eligible patients, 58 (72.5%) received formal OAM teaching from the specialty pharmacist. Of those, 54 (93.1%) enrolled in the study with 39 (72%) completing the intervention for final analysis. At study completion, all participants adequately understood OAM taking, but 41.0% had inadequate understanding of OAM handling. Throughout the study, participants reported issues that were addressed by the intervention team (28.2% to 31.6%) as well as those requiring additional assistance from the treatment team (26.3% to 38.5%), Most participants found the intervention to be very beneficial (initial evaluation, 86.5%; final evaluation, 76.9%). CONCLUSIONS: This pilot intervention addressed gaps identified by our institutional assessment through formalized OAM teaching and follow-up. Improved understanding of taking and handling OAM through this subsequent study illustrated the enhanced effect of a multidisciplinary and multicomponent intervention to better educate and support patients on OAM.

The Creation of a Comprehensive Adolescent and Young Adult Cancer Survivorship Program: "Lost in Transition" No More.

Purpose: The Reid R. Sacco AYA Cancer Program set out to improve survivorship care for AYA-aged patients (15-39 years) of pediatric or AYA cancer. This article discusses the steps in establishing the clinic, including the creation of a database on cancer history, exposures, and attendant risks of late effects. Results from the database tell the broader story of AYAs who seek care within a dedicated survivorship clinic. Methods: The database was created with REDCap® (Research Electronic Data Capture), a secure web-based, HIPAA compliant application for research and clinical study data. Data were abstracted and analyzed by trained members of the program team. Results: A total of 144 patients were seen for their initial survivorship visit between January 2013 and September 2019. Regarding physical health, two-thirds of the patients presented with an established late effect, one third with an established medical comorbidity, and 11% (n = 16) with secondary cancer related to their oncologic treatment. In assessing mental health, a significant cohort reported a known affective disorder (32%, n = 46) with one quarter already taking a psychotropic medication. Despite the transient nature of AYAs, 85% of patients remained in care within the long-term follow-up clinical model. Conclusions: Data presented illustrate how multilayered and complex survivorship care needs can be, as patients enter the clinic with complicated pre-existing psychosocial issues, significant late effects, and comorbidities. This study reinforces the value of a clinical database to better understand AYA survivors with the ultimate goal of optimizing and coordinating care.

Assessment of health-related quality of life among adults hospitalized with sickle cell disease vaso-occlusive crisis.

Sickle cell disease (SCD) is characterized by painful vaso-occlusive crises (VOCs). Self-reported pain intensity is often assessed with the Numeric Rating Scale (NRS), whereas newer patient-reported outcome measures (PROMs) assess multidimensional pain in SCD. We describe pain experiences among hospitalized adults with VOCs, using 2 PROMs: the Patient-Reported Outcomes Measurement Information System (PROMIS) Global Health and the Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me). Adults with SCD hospitalized with VOCs at 2 academic centers in Boston, Massachusetts, from April 2016 to October 2017 were eligible. Participants completed the NRS and PROMs at admission and 7 days postdischarge. PROM scores were described and compared with population norms. Length of stay (LOS) and 30-day readmission rates were assessed. Forty-two (96%) of 44 eligible patients consented and completed admission assessments. Mean age was 30.2 years (standard deviation, 9.1), 60% were women, 76% were non-Hispanic black, and 64% had hemoglobin SS. Twenty-seven participants (64%) completed postdischarge assessments. Sixty percent had ≥4 VOCs in the last year. Nearly all PROMIS Global Health and ASCQ-Me scores were worse than population norms. NRS and PROMIS Global Physical Health scores improved after discharge, the latter driven principally by improvements in pain. Overall median LOS was 7 days, and 30-day readmission rate was 40.5%. Administration of PROMs among adults with SCD hospitalized for VOCs is feasible and demonstrates participants experienced recurrent, prolonged, and severe VOCs. PROMIS Global and ASCQ-Me scores indicated substantial suffering, and the striking 30-day readmission rate highlights the vulnerability of these patients.

Timeliness of Treatment Initiation in Newly Diagnosed Patients With Breast Cancer.

BACKGROUND: Evidence-based timeliness benchmarks have been established to assess quality of breast cancer care, as delays in treatment are associated with poor clinical outcomes. However, few studies have evaluated how current breast cancer care meets these benchmarks and what factors may delay the timely initiation of treatment. PATIENTS AND METHODS: Demographic and disease characteristics of 377 newly diagnosed patients with breast cancer who initiated treatment at Tufts Medical Center (2009-2015) were extracted from electronic medical records. Time from diagnosis to initial surgery and time from diagnosis to initiation of hormone therapy were estimated with Kaplan-Meier curves. Multivariable regression analysis was used to identify factors associated with treatment delays. Thematic analysis was performed to categorize reasons for delay. RESULTS: Of 319 patients who had surgery recommended as the first treatment, 248 (78%) met the 45-day benchmark (median, 28 days; 25th-75th %, 19-43). After adjusting for potential confounders, multivariable regression analysis revealed that negative hormone receptor status (odds ratio, 3.48; 95% confidence interval, 1.44-8.43) and mastectomy (odds ratio, 4.07; 95% confidence interval, 2.10-8.06) were significantly associated with delays in surgery. Delays were mostly owing to clinical complexity or logistical/financial reasons. Of 241 patients eligible for hormone therapy initiation, 232 (96%) met the 1-year benchmark (median, 147 days; 25th-75th %, 79-217). CONCLUSION: Most patients met timeliness guidelines for surgery and initiation of hormone therapy, although risk factors for delay were identified. Knowledge of reasons for breast cancer treatment delay, including clinical complexity and logistical/financial issues, may allow targeting interventions for patients at greatest risk of care delays.

Pediatric Specialty Care Model for Management of Chronic Respiratory Failure: Cost and Savings Implications and Misalignment With Payment Models.

OBJECTIVE: To describe program design, costs, and savings implications of a critical care-based care coordination model for medically complex children with chronic respiratory failure. DESIGN: All program activities and resultant clinical outcomes were tracked over 4 years using an adapted version of the Care Coordination Measurement Tool. Patient characteristics, program activity, and acute care resource utilization were prospectively documented in the adapted version of the Care Coordination Measurement Tool and retrospectively cross-validated with hospital billing data. Impact on total costs of care was then estimated based on program outcomes and nationally representative administrative data. SETTING: Tertiary children's hospital. SUBJECTS: Critical Care, Anesthesia, Perioperative Extension and Home Ventilation Program enrollees. INTERVENTIONS: None. MEASUREMENTS AND MAIN RESULTS: The program provided care for 346 patients and families over the study period. Median age at enrollment was 6 years with more than half deriving secondary respiratory failure from a primary neuromuscular disease. There were 11,960 encounters over the study period, including 1,202 home visits, 673 clinic visits, and 4,970 telephone or telemedicine encounters. Half (n = 5,853) of all encounters involved a physician and 45% included at least one care coordination activity. Overall, we estimated that program interventions were responsible for averting 556 emergency department visits and 107 hospitalizations. Conservative monetization of these alone accounted for annual savings of $1.2-2 million or $407/pt/mo net of program costs. CONCLUSIONS: Innovative models, such as extension of critical care services, for high-risk, high-cost patients can result in immediate cost savings. Evaluation of financial implications of comprehensive care for high-risk patients is necessary to complement clinical and patient-centered outcomes for alternative care models. When year-to-year cost variability is high and cost persistence is low, these savings can be estimated from documentation within care coordination management tools. Means of financial sustainability, scalability, and equal access of such care models need to be established.

Chronic respiratory failure: Utilization of a pediatric specialty integrated care program.

OBJECTIVE: Describe utilization and satisfaction in a specialty integrated care program for children with severe, chronic respiratory insufficiency (CRI). SUBJECTS: Enrollees of the Critical Care, Anesthesia, Perioperative Extension (CAPE) and Home Ventilation Program. METHODS: Children with CRI received home visits, care coordination, and "on-demand" 24/7 access to physicians. Program activity and outcomes were recorded for 3 years using an adapted Care Coordination Measurement Tool© version. Parents completed the Consumer Assessment of Healthcare Providers and Systems (CAHPS). Patient characteristics, program activity, clinical outcomes, utilization, and satisfaction were summarized using descriptive statistics. RESULTS: CAPE provided care for 320 patients from 2012 to 2014 with a median of 7 encounters per year. Neuromuscular (n=132, 41%), chronic lung disease (n=37, 12%), and congenital heart disease (n=13, 4%) represented the majority of underlying conditions. Services included 905 home, 504 clinic, and 3633 telephone encounters, of which 43.6% included a care coordination activity. CAHPS (n=102) revealed that 92.1% (n=93) of children had at least one non-urgent (i.e., routine) visit and nearly two-thirds (64.7%, n=66) reported the need for urgent or emergency care. Overall, parents were highly satisfied with CAPE, with a mean satisfaction rating of 9.3 (±1.3) out of 10. Most parents reported that the CAPE team understood the child's (96.0%, n=95) and family's day-to-day life (86.9%, n=86). CONCLUSIONS: When given open access to an intregated care program, children in our highly complex population required a median of 7 encounters per year. We believe that this experience is scalable and may inform other organizations contemplating similar services.

Predicting Health Care Utilization for Children With Respiratory Insufficiency Using Parent-Proxy Ratings of Children's Health-Related Quality of Life.

INTRODUCTION: Children with chronic respiratory insufficiency and mechanical ventilation often experience acute illnesses requiring unscheduled hospitalizations. Health-related quality of life (HRQL) may predict future health care utilization. METHODS: Participants were 30 days to 22 years old with chronic respiratory insufficiency (N = 120). Parent-proxies completed global HRQL and general health measures. Outcomes were total health care (emergency department, outpatient, inpatient) and inpatient days over 6 months. Adjusted negative binomial regression estimated the effects of global HRQL and general health on utilization. RESULTS: Three quarters of children had any utilization; 32% had hospitalizations. Children with poor/fair global HRQL had 3.7 times more health care days than those with very good/excellent global HRQL. Children with poor/fair global HRQL had 6.3 times more inpatient days than those with very good/excellent global HRQL. Similar relationships existed between general health and utilization. DISCUSSION: HRQL was associated with health care and inpatient days. Clinical teams can use HRQL as a marker for utilization risk, enabling potentially earlier intervention, better outcomes, and lower costs.

Leveraging pediatric PROMIS item banks to assess physical functioning in children at risk for severe functional loss.

BACKGROUND: Pediatric neuromuscular illnesses often result in decreased health-related quality of life (HRQL), notably in physical functioning. Generic HRQL measures have been developed for use in general populations, but may not adequately assess patients with severe functional loss. To address this measurement gap, we created two custom parent-proxy physical functioning short forms for use among children at risk for low levels of functioning, using pediatric Patient Reported Outcomes Measurement Information System (PROMIS) item banks for Upper Extremity and Mobility. METHODS: Two custom short forms from PROMIS Upper Extremity (13 items) and Mobility (13 items) parent-proxy item banks were created and administered to parents of children (ages 5 - 22 years) enrolled in an integrated care program for management of chronic respiratory insufficiency, largely due to neuromuscular illnesses. Standardized PROMIS T-scores have a mean of 50 (SD = 10); higher scores indicate better functioning. Physicians rated clinical severity. Single proxy-rated items on mental and physical health from the Child Health Rating Inventories (CHRIs) global health scale were completed by parents. Psychometric properties, including known groups comparisons, were explored. RESULTS: Fifty-seven parents completed the parent-proxy custom PROMIS short forms. The mean Upper Extremity T-score was 21 (SD = 13); the mean Mobility T-score was 22 (SD = 11). Some participants scored at the measurement floor; two items on assistive devices did not perform well in this sample and were excluded from the Mobility T-score. Known groups comparisons showed that those with lower clinical severity had better median Upper Extremity (22 vs. 14, p < 0.001) and Mobility (28 vs. 16, p = 0.004) function than those with worse clinical severity. Both Upper Extremity and Mobility T-scores were higher in the subgroups defined by better physical and mental health, as measured by the CHRIs. CONCLUSIONS: Upper Extremity and Mobility T-scores were nearly three standard deviations below the PROMIS pediatric calibration population mean. Preliminary psychometrics demonstrated the potential to more accurately measure lower physical functioning using items from PROMIS item banks. However, some participants scored at the measurement floor despite targeting items at the lower end of the scale. Further short form refinement, enrichment of the item banks, and larger-scale field testing are needed.

The Impact on Family of Pediatric Chronic Respiratory Failure in the Home.

OBJECTIVE: To assess the family impact of managing severe, chronic respiratory failure (CRF) at home. Better understanding will inform parental counseling and serve as a point of reference for interventions. STUDY DESIGN: Families of children with CRF completed the Impact on Family Scale (IFS) and Consumer Assessment of Healthcare Providers and Systems. Using multivariable linear regression, we assessed the relationship between IFS and family, clinical, and utilization characteristics. RESULTS: A total of 118 parents (60%) completed the IFS; 114 parents (58%) completed all measures. The 15-item IFS mean total score was 40 (SD = 10) with a possible range of 15-60 (greater scores indicate more impact). Modeling identified a negative association with parent emotional functioning, parent-rated child health, and private insurance only (compared with both private/public), and other family characteristics (eg, parental education, marital status, and income) were not associated with IFS scores. CONCLUSION: Families of children with CRF are greatly impacted by their child's health. In contrast to other children with special health care needs, family characteristics were not associated with IFS scores, excluding insurance type. These results may reflect more uniform demands and stressors related to CRF. Future research should identify interventions to attenuate the impact of CRF.