Adolescent Young Adults Research Articles

Experience with Ruxolitinib (Jakafi®) As a Salvage Therapy for Graft-Versus-Host Disease in Children and Young Adults

Ruxolitinib was recently approved as a treatment for steroid refractory acute GVHD (aGVHD) and is used in adults for the treatment of chronic GVHD (cGVHD). As a JAK-1/JAK-2 inhibitor, it reduces T-cell proliferation and trafficking and leads to a potent anti-inflammatory effect. There is limited data of its use in pediatric and adolescent/young adult (AYA) patients. We report our single center experience with ruxolitinib in this population. Data on patients treated with ruxolitinib at Lucile Packard Children's Hospital between 8/1/18 and 8/31/19 were retrospectively analyzed after institutional IRB approval. aGVHD was graded according to the Modified Glucksberg scale, while cGVHD was scored as limited or extensive based on Seattle criteria. Only patients on ruxolitinib for ≥14 days were evaluated for response, which was defined as a reduction by at least one grade in severity of aGVHD. Ability to reduce other GVHD medications, especially steroids, and/or symptomatic improvement was considered a response in cGVHD. 15 patients (median age 18, range: 11-29 years) received ruxolitinib: 5 for aGVHD and 10 for cGVHD. Standard starting dose was 5 mg twice a day. Patients in the aGVHD group were steroid refractory with ≥ Grade III GVHD and had received a median of 3 (range: 2-5) different agents prior to ruxolitinib. Patients received treatment for a median of 46 days (range: 23-332). 4 of 5 patients had a partial response (PR) with one non-responder, for an overall response rate (ORR) of 80%. All patients experienced at least one episode of infection- CMV reactivation (n=2), adenovirus (n=1), viral gastroenteritis (n=1), bacterial infections (n=3) and probable fungal infection (n=1). Dose-limiting cytopenia was the only toxicity observed in 3 patients (60%), requiring dose reduction or discontinuation. 2 patients had TRM, both related to GVHD and infections. 10 patients treated for cGVHD (6 limited, 4 extensive) had received a median of 3 (range: 2-6) different agents prior to ruxolitinib. One patient required early cessation of therapy due to a severe allergic reaction and in another patient ruxolitinib was stopped due to leukemia relapse. Of the eight patients evaluable for response, treatment duration was for a median of 195 days (range: 59-372) with an ORR of 88%. 5 (63%) patients developed infections: CMV reactivation (n=2), respiratory infections (n=4), bacterial infection (n=1). Only one patient required dose reduction due to GI side effects and cytopenias. Ruxolitinib is an effective salvage therapy for severe GVHD. It was well tolerated in our patient population, with myelosuppression as the dose limiting toxicity, especially in aGHVD patients. A high rate of infections was noted during ruxolitinib treatment and concomitant antimicrobial prophylaxis is strongly recommended.

Communicating and disseminating research findings to study participants: Formative assessment of participant and researcher expectations and preferences.

Translating research findings into practice requires understanding how to meet communication and dissemination needs and preferences of intended audiences including past research participants (PSPs) who want, but seldom receive, information on research findings during or after participating in research studies. Most researchers want to let others, including PSP, know about their findings but lack knowledge about how to effectively communicate findings to a lay audience. We designed a two-phase, mixed methods pilot study to understand experiences, expectations, concerns, preferences, and capacities of researchers and PSP in two age groups (adolescents/young adults (AYA) or older adults) and to test communication prototypes for sharing, receiving, and using information on research study findings. PSP and researchers agreed that sharing study findings should happen and that doing so could improve participant recruitment and enrollment, use of research findings to improve health and health-care delivery, and build community support for research. Some differences and similarities in communication preferences and message format were identified between PSP groups, reinforcing the best practice of customizing communication channel and messaging. Researchers wanted specific training and/or time and resources to help them prepare messages in formats to meet PSP needs and preferences but were unaware of resources to help them do so. Our findings offer insight into how to engage both PSP and researchers in the design and use of strategies to share research findings and highlight the need to develop services and support for researchers as they aim to bridge this translational barrier.

222. Using Reproductive Health Visits to Engage Adolescent and Young Adult Women in Primary Care.

Adolescent/Young Adult (AYA) primary care visits are an opportune time to address physical, psychological, and social influences on developing health behaviors. However, many AYAs do not receive primary care despite available guidelines for screening, immunizations, and annual physical exams. Frequent interactions with the healthcare system by AYA women for family planning and other reproductive health services may be used to improve engagement in care and promote wellness; however, less cumbersome methods may require less interaction with the healthcare system. The purpose of this research is to examine differences in clinic engagement, receipt of primary care, STI/HIV screening, and immunizations by contraceptive use.

124. Fyre (Foster Youth Risk Assessment) Investigation

Foster care youth have long been considered a high-risk patient population . Recent evidence suggests that institutional and sociodemographic factors (e.g. access to services, age, gender, race/ethnicity) play important roles in behavioral and health (B&H) outcomes in foster care-involved youth as compared to the general population. There is little data, however, comparing foster care-involved youth to an already high-risk patient population. This project aims to understand whether adolescent young adults (AYAs) with foster care involvement engage in high-risk behaviors more frequently than high-risk AYAs without foster care involvement.

237. Utilizing Community Engagement Methods and Partnerships to Reduce STDs and Improve Health Equity Among Adolescent Minorities and Young Adults: Results and Reflections From Year 1 of the Cars Project

237. Utilizing Community Engagement Methods and Partnerships to Reduce STDs and Improve Health Equity Among Adolescent Minorities and Young Adults: Results and Reflections From Year 1 of the Cars Project

A qualitative investigation assessing whether implementing a teenage young adult information evening, within a radiotherapy department, would improve the information support and treatment pathway for 18–24-year-old cancer patients

Key Words: Teenage Young Adult, Radiotherapy, Information Support, Open Evening. Introduction: Teenage Young Adult (TYA) patients within the 18 to 24 year age group form a unique cohort who often report high levels of unmet needs throughout their treatment pathway; lack of medical and practical information surrounding treatments and their short term effects, deficiency of long term side effect and fertility discussions and limited opportunities to meet other TYA patients stated commonly 1-3. Furthermore, despite guidelines already being in place that reinforce the unique nature of this cohort and highlight the need for specialised care 4, these patients still regularly have insufficient access to dedicated wards, specialised staff and age appropriate information and support networks 5,6. This study evaluated the current information services provided to TYA oncology patients, aged 18 to 24 years, within a single radiotherapy department through retrospective patient views and additionally assessed the merit of implementing a dedicated TYA information evening. Method: Focus group sessions and open-ended questionnaires were used as the primary data collection tool for this study; producing rich and informative data. The transcriptions from the focus groups, along with the questionnaire responses were then analysed using coding techniques 7, resulting in 6 distinct themes. Results: The participants felt they received adequate information about their radiotherapy treatment and its side effects. However, participants did report gaps within the service provision that need addressing to improve future treatment pathways. Furthermore, treatment information was always supplied in paper format which was often undesirable to the cohort with a more interactive format preferred. The participants disclosed very strong feelings of being different to “usual cancer patients” and displayed a desire for more opportunities to meet other TYA patients to be provided within their pathway. Conclusion: The findings of this study suggest that the implementation of a dedicated TYA open evening would improve the information support and treatment pathway for 18-24-year-old cancer patients. The evening could offer a wanted opportunity where TYA patients could meet each other and additionally, treatment information and age appropriate support information could be provided in a different and more suitable format than current paper-based literature. Numerical References [1] Cheung CK, Zebrack B. What do adolescents and young adults want from cancer resources? Insights from a Delphi panel of AYA patients. Supportive Care Cancer. 2017;25(1):119-126. [2] Olsson M, Jarfelt M, Pergert P, Enskär K. Experiences of teenagers and young adults treated for cancer in Sweden. European Journal of Oncology Nursing. 2015;19(5):575-581. [3] Skaczkowski G, White V, Thompson K, et al. Factors influencing the documentation of fertility-related discussions for adolescents and young adults with cancer. European Journal of Oncology Nursing. 2018; 34:42-48. [4] National Institute for Health and Clinical Excellence (NICE). Cancer services for children and young people. http://nice.org.uk/guidance/qs55. Updated 2014. Accessed 19.03.18. [5] Barr RD, Ferrari A, Ries L, Whelan J, Bleyer WA. Cancer in adolescents and young adults: A narrative review of the current status and a view of the future. JAMA Pediatrics. 2016;170(5):495-501. [6] Lopez G, Liu W, Madden K, Fellman B, Li Y, Bruera E. Adolescent-young adults (AYA) with cancer seeking integrative oncology consultations: Demographics, characteristics, and self-reported outcomes. Supportive Care Cancer. 2018;26(4):1161-1167. [7] Vaismoradi M, Turunen H, Bondas T. Content analysis and thematic analysis: Implications for conducting a qualitative descriptive study. Nursing Health Science. 2013;15(3):398-405.

Sex and age-related changes in L-arginine metabolism in peripheral blood leukocytes in young caucasians with type 1 diabetes mellitus

We found that hyperactivation of cytoplasmic (anti-inflammatory) and mitochondrial (pro-inflammatory) arginase isoforms in peripheral blood leukocytes (PBL) is more pronounced in women than in male patients with type 1 diabetes mellitus (T1DM) who received insulin for one year, especially in adolescents young adults 15 years old (12.0 - 25.0) compared with children/adolescents 9.3 years old (4.5-11.8). Long-term treatment with insulin up to 14 years (on average 5.3-5.9) reduces the activity of arginase, especially in puberty girls with a tendency to normalize mitochondrial arginase, while in prepubertal boys the activity of both arginase isoforms almost doubles and remains elevated in puberty boys and can be involved in inhibiting nitric oxide synthase (NOS) and decreasing the bioavailability of NO. This is confirmed by the concomitant continuous decrease in the levels of nitric oxide synthase (NOS) products, stable metabolites of NO (nitrite) and L-citrulline in the cytoplasm and mitochondria of PBL in prepubertal girls and boys, in the latter, regardless of age and insulin therapy, while in girls of puberty changes not found, apparently, due to the increased level of sex hormones that promote the expression and activity of NOS, which contribute to the inhibition of arginase. Further studies are needed to understand whether sex and age-related changes found in L-arginine metabolism in PBL can be useful in assessing the stage and progression of T1DM and the effectiveness of therapy.

Adolescent/Young Adult Perspectives of a Therapeutic Music Video Intervention to Improve Resilience During Hematopoietic Stem Cell Transplant for Cancer.

This empirical phenomenology study reports adolescents/young adults (AYA) experiences of the therapeutic music video (TMV) intervention arm of a randomized controlled clinical trial (Children's Oncology Group; COG-ANUR0631; R01 NR008583) during hospitalization for a hematopoietic stem cell transplant. A purposive subsample of 14 AYA were interviewed using a broad open-ended data-generating question about their TMV intervention experiences. At the end of each interview, we also asked AYA for suggestions on how to improve the TMV. Analysis of the narrative data resulted in four theme categories: (a) An Interwoven Experience of the Transplant and TMV Intervention; (b) TMV as a Guided Opportunity for Reflection, Self-Expression, and Meaning-Making; (c) Telling My Story: The Work of Deriving Meaning; and (d) A Way to Overcome the Bad Side of Cancer. AYA suggestions for improving the TMV are also summarized. Findings provide insight into ways the TMV supports AYA efforts to overcome distress and challenges by providing opportunities to reflect on what is meaningful, connect with others, and explore/identify personal strengths. Findings also inform our understanding about how the TMV may have functioned (i.e., mechanisms of action) to bring about significant change in AYA self-reported outcomes (i.e., positive coping, social support, and family function) for this trial.

A Randomized Phase 3 Trial of Blinatumomab Vs. Chemotherapy As Post-Reinduction Therapy in High and Intermediate Risk (HR/IR) First Relapse of B-Acute Lymphoblastic Leukemia (B-ALL) in Children and Adolescents/Young Adults (AYAs) Demonstrates Superior Efficacy and Tolerability of Blinatumomab: A Report from Children's Oncology Group Study AALL1331

A Randomized Phase 3 Trial of Blinatumomab Vs. Chemotherapy As Post-Reinduction Therapy in High and Intermediate Risk (HR/IR) First Relapse of B-Acute Lymphoblastic Leukemia (B-ALL) in Children and Adolescents/Young Adults (AYAs) Demonstrates Superior Efficacy and Tolerability of Blinatumomab: A Report from Children's Oncology Group Study AALL1331

Prognostic Significance of Cytogenetic Abnormalities for Patients with Acute Lymphoblastic Leukemia, a Region-Wide Retrospective Study

Background; Cytogenetic abnormalities (CA) have been reported as one of the independent prognostic factors for patients with acute lymphoblastic leukemia (ALL). The most common CA in ALL is the Philadelphia chromosome (Ph). Recently, by the introduction of tyrosine kinase inhibitors for Ph+ALL patients and pediatric-inspired protocol for adolescent/young-adult (AYA) patients, the management of ALL has progressed dramatically. We therefore retrospectively analyzed the impact of CA for patients with ALL in the era of TKIs and pediatric-inspired protocol for AYA. Methods; Clinical data for 512 patients who were diagnosed as having ALL between 2007 and 2017 were collected from 21 centers in Hokkaido, Japan. Patients with lymphoblastic lymphoma and Burkitt leukemia were excluded from this study. Results; The median age of the patients was 55 years (range: 15-84 years). Ninety-two of the patients were pediatric (0-14 years), 86 were AYA (15-35 years), 195 were adult (36-65 years) and 148 were elderly patients (66- years). Cytogenetic (G-banding) results were available in 486 patients. Three hundred forty-seven patients had abnormal karyotypes (AK), and 139 patients had normal karyotype (NK). BCR-ABL, including masked Ph, was positive in 193 patients, and 181 (93.8%) of them were more than 36 years. Abnormalities of -7/del (7), +8, +21, 11q32 (MLL), E2A/PBX1 or complex karyotype were seen in 38, 34, 46, 13, 10 and 94 of the patients, respectively. After a first remission induction therapy, 418 of 467 (89.5%) evaluable patients achieved complete remission (CR), and BCR-ABL+ patients showed better CR rate than those without BCR-ABL (93.7% vs. 87.4%, P=0.04). At the median follow-up of 1180 days (9-4049 days), overall survival (OS) was superior in patients with NK than those with AK (P=0.01), and BCR-ABL+ patients showed poorer OS than those without BCR-ABL (P=0.01). However, by subgroup analyses of the age groups, there were no difference of OS between NK and AK (P=0.56 for pediatric, P=0.19 for AYA, P=0.32 for adult and P=0.98 for elderly). In adult and elderly patients, OS was not different between BCR-ABL+ and BCR-ABL-, though in patients over 70 years, BCR-ABL positivity was associated with superior OS (Hazard ratio, 3.2; 95% confidence intervals, 1.04-4.78, P=0.02). The abnormalities of +8 or +21 showed excellent OS in pediatric or AYA patients, however, they showed poor OS in adult or elderly patients. In adult or elderly BCR-ABL- patients, OS of patients with complex karyotype was inferior to those without complex karyotype. Conclusion; BCR-ABL was not associated with poor outcome in the era of TKI. We need to evaluate the effect of CA on patients' outcome depending on age groups. Disclosures No relevant conflicts of interest to declare.

Benefits of a Pharmacist Led Oral Chemotherapy Monitoring Program for Patients with Chronic Myeloid Malignancies: A Patient Reported Outcome (PRO) Study

Benefits of a Pharmacist Led Oral Chemotherapy Monitoring Program for Patients with Chronic Myeloid Malignancies: A Patient Reported Outcome (PRO) Study

The VAC regimen for adult rhabdomyosarcoma: Differences between adolescent/young adult and older patients.

Compared to pediatric patients, adult rhabdomyosarcoma (RMS) patients have poor prognoses, but the differences in prognoses and treatment sensitivity between adolescent/young adult (AYA) patient and older RMS patients have not been established. To evaluate and compare the efficacy and safety of the vincristine/dactinomycin/cyclophosphamide (VAC) regimen, that is, the standard combination chemotherapy regimen for pediatric RMS, in AYA and older patients. We retrospectively reviewed the clinical records of adolescent and adult RMS patients treated at our institution and patients treated with the VAC were enrolled in the analyses. The differences in the efficacy and safety of VAC between the AYA patients (15-39 years old) and older patients (≥40 years old) were compared. Total of 23 patients were enrolled (14 AYA patients and nine older patients) were enrolled. With the median follow-up of 89.4weeks (range 17.7-263.1weeks), the median progression-free survival (PFS) and overall survival (OS) of the VAC regimen were 61.4weeks (95% CI, 32.6-90.3) and 104.0weeks (95% CI, 43.8-164.2), respectively, with no significant differences in the PFS or OS between the AYA and older patients. There were no differences in the response rate of the VAC between AYA (64.3%) and older (66.7%), either. Safety profiles were similar between the AYA and older groups, but exposure to antitumor drugs of the VAC tended to be lower in the older group; median number of total cycle of the VAC was 13 in AYA and 10 in older, cumulative cyclophosphamide dose was 16.8g/m2 in AYA and 12.8g/m2 in older, and number of vincristine skips was six in AYA and 12 in older, respectively. There were no significant differences in responses to the VAC among AYA and older patients with RMS. With the appropriate dose modification, the VAC regimen could be a feasible treatment option for RMS patients>40 years old.

Development and Content Validation of End of Treatment Questionnaires for Children With Cancer.

Purpose: To describe the development and content validation of measures to assess the psychoeducational needs of children, adolescents/young adults (AYAs), and their parents at the end of successful treatment for cancer. Method: Professional experts, which included pediatric oncology nurses and advanced practice registered nurses, conducted a systematic review of the literature to determine specific end of treatment (EOT) needs of children and AYAs with cancer and their parents and evaluate available tools to measure these needs. From this review, two EOT questionnaires were initially developed. Oncology Family Advisory Board (FAB) members served as experiential experts in refining and validating these questionnaires. FAB members participated in a content validation process, rating questionnaires online, and subsequently participating in a focus group to establish content validity (n = 6). Results: Three EOT questionnaires were ultimately developed. The Child/AYA questionnaire was divided into two separate measures for developmental and literacy considerations. The Parent/Caregiver and the AYA questionnaires each contain 38 items with a content validity index score of 100%. The Child questionnaire contains 37 items with a content validity index score of 100%. Conclusion: Content validity was established for three EOT questionnaires, each of which has the potential to elicit information regarding needs and potential gaps in services perceived by childhood cancer survivors and their parents. Further psychometric testing is needed to determine stability (test-retest reliability) and construct validity of the questionnaires.

P14.04 10 years of weekly web conference for brain tumor of Adolescent/Young Adult (AYA) on behalf of ANOCEF, GO-AJA and SFCE societies

Abstract BACKGROUND The skills of adult versus pediatric neuro-oncologists are not completely similar though additive. Because the tumors and their protocols are different and the tolerance and expected sequelae are specific. Multidisciplinary meetings including adult and pediatric neuro oncologists are warranted to share expertise. MATERIAL AND METHODS Since 2008, on behalf of ANOCEF, GO-AJA and SFCE societies, a weekly national web based conference was held in France. Any patient with the following criteria could be discussed: Adolescent and Young Adults aged between 15 and 25 years, and any adult with a pediatric type pathology, including medulloblastoma, germ cell tumors, embryonic tumors, ependymoma, pilocytic astrocytoma. RESULTS Number of cases discussed increased from 8 to 202/year, with a mean number of 3 cases (1 to 7) discussed at each meeting. In 2018, 36 meetings were held, with a mean of 8 participants (2 to 14) issued from various French centers. 168 cases were discussed in 2018. Mean age was 30 years old (7 to 67). 45% were discussed at diagnosis; The patients had mostly medulloblastomas (n=47), ependymomas (n=24), low (n=21) or high grade gliomas (n=11), pineal tumors (n=20), germ cell tumors (n=9) and embryonal tumors (n=6). The rate of inclusion in protocols was increased since the opening of this web conference, especially for the germ cell tumor GCT SIOP protocol that is opened without age restriction, and in RSMA standard risk or MEVITEM relapse adult medulloblastoma protocols. CONCLUSION Multidisciplinary Web conference for AYAs is feasible and fruitful. It shares knowledge and increases the inclusion rate in protocols. It should be developped further.

Abstract 5053: Epstein-Barr virus prevalence in classical Hodgkin lymphoma tumors is explained by histologic subtype, not race/ethnicity in a multiethnic US population

Abstract Epstein-Barr virus (EBV) is present in a varying proportion of classical Hodgkin lymphoma (cHL) tumors reportedly associated with age, sex, race/ethnicity and histologic subtype. As part of a study to examine the tumor microenvironment and survival in a multiethnic set of US cHL cases, we examined the distribution of EBV expression in tumor blocks from a subset of 269 of the available cases. We confirmed cHL diagnosis and histological subtype in H&E sections of FFPE tumor blocks of excisions and core biopsies from cases provided by Southern California Kaiser, Winship Cancer Center at Emory University, City of Hope National Medical Center, Grady Memorial Hosiptal, University of California Los Angeles and University of Southern California hospitals diagnosed from 1996-2016. Immunostain results were available for most cases. Tissue microarrays were constructed with two 2 mm cores from each case with 29 cases on each array. EBER was assessed using in situhybridization and scored as negative, positive in HRS cells or positive in the surrounding normal infiltrate. Demographic and clinical information, including the biopsy anatomic site, subtype, race/ethnicity (Hispanic, African American, Asian, non-Hispanic white), age at diagnosis and gender. Multiple logistic regression was conducted to assess the relationship between age, race/ethnicity, gender, subtype and EBV tumor status. Race/ethnicity data was available at this time for 237 cases. Of these, 186 were nodular sclerosis (NS), 53 were mixed cellularity (MC), 8 were lymphocyte depleted (LD), 3 were lymphocyte rich (LR), and 10 were not otherwise specified (NOS). Fifty-seven cases were African American, 48 were non-Hispanic white, 115 were Hispanic, 16 were Asian and one was other race. 106 were female (42%). Age at diagnosis ranged from 5-84 years, with 118 (50%) in the adolescent/young adult (AYA) range (15-35). EBV prevalence in HRS cells by subtype was 0% for LD, 47% for MC, 33% for LR, 23% for NS and 50% for NOS. When restricted to the two most common subtypes, NS and MC, histologic subtype alone was a statistically significant predictor of EBV tumor status (p=.0008) and when adjusting for age, sex, site and race/ethnicity (p=.0011). In NS cases, EBV HRS cell positivity was highest in non-Hispanic whites (31%), followed by Hispanics (23%) and African Americans (18%). Among MC cases, it was very high among African Americans (80%) compared to Hispanics and non-Hispanic whites (33-39%). The patterns were similar when restricted to the AYA age group. 6.5%, 4% and 20% of EBV-negative NS, MC and NOS cases, respectively, had EBV present in non-malignant lymphocytes but not in HRS cells. Histologic subtype was the strongest predictor of EBV HRS cell positivity in this set of multiethnic cHL patients. Unlike previous reports, EBV-positive tumors were not more common among non-whites, except for African American MC cases. Citation Format: Rachel Bolanos, Amie Hwang, Chun Chao, Christopher Flowers, Sheeja Pullarkat, Jose Aparicio, Sophia Wang, Karen Mann, Leon Bernal-Mizrachi, Joo Song, Christian Steidl, Christine Lee, Wendy Cozen, Iran Siddiqi. Epstein-Barr virus prevalence in classical Hodgkin lymphoma tumors is explained by histologic subtype, not race/ethnicity in a multiethnic US population [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2019; 2019 Mar 29-Apr 3; Atlanta, GA. Philadelphia (PA): AACR; Cancer Res 2019;79(13 Suppl):Abstract nr 5053.

Assessment of enrollment characteristics for Children’s Oncology Group (COG) upfront therapeutic clinical trials 2004-2015.

6564 Background: Improvements in pediatric cancer survival are attributed to cooperative clinical trials. Under representation of specific demographic groups has been described in adult and pediatric cancer and poses a threat to the generalizability of trial results. A comprehensive evaluation of data provided by the Children’s Oncology Group (COG) of upfront trial enrollment for US patients 0 to 29 years old between 2004 and 2015 was performed to assess for disparities in participation. Methods: Estimates of cancer cases were calculated using the Surveillance, Epidemiology, and End Results registry and the US Census and compared to observed COG cases. Percent enrollment and Standardized Ratios of enrollment were calculated across various demographic, disease, and socioeconomic groups. The COG website was utilized to quantify available upfront trials during the study period and assess age eligibility criteria. Results: 21.3% of estimated US cancer patients age 0 to 19 years enrolled on COG trials. Younger patients were consistently more represented across disease types and race/ethnicities. Patients with hematologic malignancies were more represented compared to solid and central nervous system (CNS) tumors. Conclusions: COG clinical trial enrollment rates are declining, which may be due to challenges in pediatric drug development, difficulty designing feasible trials for highly curable diseases, and issues in ensuring trial availability for the heterogeneous group of solid and CNS tumors. Though racial/ethnic groups and county-level socioeconomic factors were proportionally represented, under representation of the adolescent/young adult (AYA) population and younger patients with solid and CNS tumors remain significant concerns. Targeted enrollment efforts should focus on the identified subgroups and further research should evaluate AYA enrollment across all available trials to provide continued treatment advances for all patients.

The Role of Stigma in the Relationship Between Illness Intrusiveness and Adjustment in Adolescents and Young Adults: A Path Model.

Adolescents/Young Adults (AYAs) with a chronic illness display elevated risk for poor psychosocial outcomes, yet relatively little is known about factors that place these individuals at risk. Illness intrusiveness is a known predictor of negative psychosocial outcomes in AYAs. Illness-related stigma, an understudied concept in this population, may also be a key contributor to increased intrusiveness. The present study sought to determine if higher levels of illness-related stigma would be associated with higher levels of depressive and anxious symptoms in AYAs with a chronic illness, and whether this relationship would be mediated by illness intrusiveness. College students with a chronic illness completed measures of illness-related stigma, illness intrusiveness, and both depressive and anxious symptoms. A path model indicated that stigma was significantly related to illness intrusiveness, and illness intrusiveness was significantly related to depressive and anxious symptoms. Both indirect paths from stigma to depressive and anxious outcomes were significant. There were also significant direct effects of stigma on depressive and anxious outcomes. An additional path model was tested to assess anxious and depressive outcomes as multidimensional factors by evaluating the individual factors of both scales as outcomes. This model revealed similar results. Findings support previous research indicating relationships between stigma, illness intrusiveness, and negative psychosocial outcomes, with illness intrusiveness serving as a possible mediator between illness-related stigma and depressive and anxious symptoms.

Lasting effects of cancer and its treatment on employment and finances in adolescent and young adult cancer survivors.

The impact of cancer and its treatment on employment and financial burden in adolescents/young adults (AYAs) is not fully known. Eligibility for this cross-sectional study of AYA cancer survivors included the diagnosis of a malignancy between ages 18 and 39years and survey completion within 1 to 5years from diagnosis and ≥1year after therapy completion. Participants were selected randomly from the tumor registries of 7 participating sites and completed an online patient-reported outcomes survey to assess employment and financial concerns. Treatment data were abstracted from medical records. Data were analyzed across diagnoses and by tumor site using logistic regression and Wald-based 95% confidence intervals adjusting for age (categorized), sex, insurance status, education (categorized), and treatment exposures. Participants included 872 survivors (breast cancer, n=241; thyroid cancer, n=126; leukemia/lymphoma, n=163; other malignancies, n=342). Exposure to chemotherapy in breast cancer survivors was associated with an increase in self-reported mental impairment in work tasks (odds ratio [OR], 2.66) and taking unpaid time off (OR, 2.62); survivors of "other" malignancies reported an increase in mental impairment of work tasks (OR, 3.67) and borrowing >$10,000 (OR, 3.43). Radiation exposure was associated with an increase of mental impairment in work tasks (OR, 2.05) in breast cancer survivors, taking extended paid time off work in thyroid cancer survivors (OR, 5.05), and physical impairment in work tasks in survivors of "other" malignancies (OR, 3.11). Finally, in survivors of "other" malignancies, having undergone surgery was associated with an increase in physical (OR, 3.11) and mental impairment (OR, 2.31) of work tasks. Cancer treatment has a significant impact on AYA survivors' physical and mental work capacity and time off from work.

The development of the Adolescent/Young Adult Self-Management and Independence Scale II: Psychometric data.

Measuring self-management behaviors in adolescents and young adults with chronic health conditions has become a priority in health care, yet there is a paucity of instruments that capture these behaviors. The purpose of this psychometric study was to evaluate the reliability and validity of the 17-item generic Adolescent/Young Adult Self-Management and Independence Scale II (AMIS II). Data were collected from 201 adolescents/young adults (AYA) with spina bifida and 129 of their parents. Exploratory factor analysis, confirmatory factor analysis, Cronbach alpha, frequencies, Pearson correlations, and intraclass correlations were used to evaluate the data. The exploratory factor analysis of parent data supported two related self-management factors (Condition Self-Management and Independent Living Self-Management). Confirmatory factor analysis of AYA data confirmed these two factors and an overall scale with good fit statistics (GFI and CFI = 0.86-0.95; RMSEA = 0.057). Internal reliabilities ranged from α= 0.72-0.89. Intraclass correlation analysis supported the stability of the instrument (ICC parent report = 0.82, AYA report = 0.84). Concurrent validity was supported with low to moderate correlations to six related but distinct variables. Psychometric analysis supports this expanded measure of self-management for AYA with spina bifida. Evaluation of this instrument in AYA with other chronic health conditions is underway.

Reduced-Intensity Allogeneic HSCT for Children and Adolescents/Young Adults with CML: A Study from the Adult and Pediatric CML Working Group of the Japan Society for Hematopoietic Cell Transplantation

Background: The introduction of tyrosine kinase inhibitor (TKI) has reduced the indications for allogeneic hematopoietic stem cell transplants (HSCT) in patients with chronic myeloid leukemia (CML). In children and adolescents/young adults (AYA), however, the long-term side effects of TKI and financial burden could become a future issue. Thus, the role of HSCT with reduced intensity conditioning (RIC) as an alternative to TKI especially in the first chronic phase (CP1) should be defined for children and AYA with CML. However, less is well-known about the efficacy of RIC regimens on children and AYA with CML, in chronic phase (CP) as well as advanced phase.Aims: To describe the long-term outcomes of children and AYA with all phases of CML treated with a RIC HSCT and to define potential prognostic factors for outcome.Patients and Methods: We retrospectively analyzed 3796 patients with CML using data from the Transplant Registry Unified Management Program of the Japan Society of Hematopoietic Cell Transplantation and included children and AYA under 30 years old at HSCT who underwent allo-HSCT between 2001 and 2014 and received TKI before HSCT. The myeloablative conditioning regimen was defined as TBI ≥ 8 Gy, busulfan > 8mg/kg, or melphalan > 180 mg/m2.Result: The characteristics of patients are summarized in Table 1. RIC was selected preferentially in patients with better status. The median follow-up of survivors was 64 months (3 - 171 months). There was no significant difference in 5-year overall survival (OS) among CP (124 cases), accelerated phase (AP) (23 cases) and blastic phase (BP) (53 cases) at diagnosis, 83%, 71% and 73%, respectively. In CP at diagnosis, 5-year OS was significantly higher in CP1 (89 cases) at HSCT than in CP2≤/AP/BP (35 cases) (89% versus 66%, p = 0.0004), and in CP1 at HSCT, there was no difference in 5-year OS between myeloablative conditioning (MAC) (58 cases) and RIC (31 cases) (both 89%) (Figure 1). However, 5-year OS in RIC was significantly higher in children (0-14 years old, 23 cases) than in AYA (15-29 years old, 8 cases) (95% versus 75%, p = 0.0495). Two of eight AYA patients with CP1 died after RIC HSCT due to grade IV acute GVHD-related complications without disease relapse, suggesting that the indications for RIC in AYA with CP1 should be carefully judged. In AP/BP at diagnosis, 5-year OS was significantly higher in the second chronic phase (CP2) (48 cases) at HSCT than in the third chronic phase (CP3)≤/AP/BP (28 cases) (82% versus 56%, p = 0.0073). However, the rate of major cytogenetic response (MCyR) at HSCT was significantly higher in CP2 than in CP3≤/AP/BP, and when focusing only on MCyR at HSCT in AP/BP at diagnosis, there was no difference in 5-year OS between CP2 at HSCT (40 cases) and CP3≤/AP/BP (13 cases) (83% versus 78%). On condition of MCyR at HSCT in AP/BP at diagnosis, regardless of the phase at HSCT, there was no difference in 5-year OS between MAC (46 cases) and RIC (7 cases: 2 in children and 5 in AYA) (79% versus 100%) (Figure 2), suggesting that even in AP/BP at diagnosis, RIC could be indicated for the patients with good response to TKI. On multivariate analysis, disease phase at HSCT and time from diagnosis to HSCT were independent predictors of OS in CP at diagnosis (Table 2), and cytogenetic response at HSCT and stem cell source in AP/BP at diagnosis (Table 3). There was no significant difference of OS between MAC and RIC.Conclusion: In HSCT for CML after TKI administration, the indications for RIC in children under 15 years old with CP1 are appropriate. In RIC HSCT for AYA patients under 30 years old with CP1, caution should be exercised in transplant-related mortality rather than disease progression. Furthermore, even in AP/BP at diagnosis, RIC could be indicated for children and AYA patients with MCyR at HSCT. [Display omitted] DisclosuresIchinohe:Astellas Pharma: Research Funding; Chugai Pharmaceutical Co.: Research Funding; CSL Behring: Research Funding; Eisai Co.: Research Funding; Kyowa Hakko Kirin Co.: Research Funding; Ono Pharmaceutical Co.: Research Funding; Pfizer: Research Funding; Nippon Shinyaku Co.: Research Funding; MSD: Research Funding; Otsuka Pharmaceutical Co.: Research Funding; Repertoire Genesis Inc.: Research Funding; Sumitomo Dainippon Pharma Co.: Research Funding; Taiho Pharmaceutical Co.: Research Funding; Takeda Pharmaceutical Co.: Research Funding; Zenyaku Kogyo Co.: Research Funding; Alexion Pharmaceuticals: Honoraria; Bristol-Myers Squibb: Honoraria; Celgene: Honoraria; JCR Pharmaceuticals: Honoraria; Janssen Pharmaceutical K.K.: Honoraria; Mundipharma: Honoraria; Novartis.: Honoraria.