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Article ; Online: Relapse of large B-cell lymphoma with IRF4 rearrangement associated with SLAM-associated protein deficiency.

Elhodaky, Mostafa / Gunderman, Lauren M / Bachula, Mateusz / Liu, Guorong / Reilly, Paige / Schmidt, Mary L / Frederiksen, John Karl / Lu, Xinyan / Jennings, Lawrence / Rossoff, Jenna / Khanolkar, Aaruni / Gong, Shunyou

Pediatric blood & cancer

2023 , Page(s) e30478

Language	English
Publishing date	2023-05-30
Publishing country	United States
Document type	Editorial
ZDB-ID	2131448-2
ISSN	1545-5017 ; 1545-5009
ISSN (online)	1545-5017
ISSN	1545-5009
DOI	10.1002/pbc.30478
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Article ; Online: Practice Preferences for Consolidative Hematopoietic Stem Cell Transplantation Following Tisagenlecleucel in Children and Young Adults with B Cell Acute Lymphoblastic Leukemia.

McNerney, Kevin O / Moskop, Amy / Winestone, Lena E / Baggott, Christina / Talano, Julie-An / Schiff, Deborah / Rossoff, Jenna / Modi, Arunkumar / Verneris, Michael R / Laetsch, Theodore W / Schultz, Liora

Transplantation and cellular therapy

2023 Volume 30, Issue 1, Page(s) 75.e1–75.e11

Abstract: Treatment with tisagenlecleucel (tisa-cel) achieves excellent complete remission rates in children and young adults with relapsed or refractory B cell acute lymphoblastic leukemia (B-ALL), but approximately 50% maintain long-term remission. Consolidative ...

Abstract	Treatment with tisagenlecleucel (tisa-cel) achieves excellent complete remission rates in children and young adults with relapsed or refractory B cell acute lymphoblastic leukemia (B-ALL), but approximately 50% maintain long-term remission. Consolidative hematopoietic stem cell transplantation (cHSCT) is a potential strategy to reduce relapse risk, but it carries substantial short- and long-term toxicities. Additionally, several strategies for management of B cell recovery (BCR) and next-generation sequencing (NGS) positivity post-tisa-cel exist, without an accepted standard. We hypothesized that practice preferences surrounding cHSCT, as well as management of BCR and NGS positivity, varies across tisa-cel-prescribing physicians and sought to characterize current practice preferences. A survey focusing on preferences regarding the use of cHSCT, management of BCR, and NGS positivity was distributed to physicians who prescribe tisa-cel for children and young adults with B-ALL. Responses were collected from August 2022 to April 2023. Fifty-nine unique responses were collected across 43 institutions. All respondents prescribed tisa-cel for children and young adults. The clinical focus of respondents was HSCT in 71%, followed by leukemia/lymphoma in 24%. For HSCT-naive patients receiving tisa-cel, 57% of respondents indicated they made individualized decisions for cHSCT based on patient factors, whereas 22% indicated they would avoid cHSCT and 21% indicated they would pursue cHSCT when feasible. Certain factors influenced >50% of respondents towards recommending cHSCT (either an increased likelihood of recommending or always recommending), including preinfusion disease burden >25%, primary refractory B-ALL, M3 bone marrow following reinduction for relapse, KMT2A-rearranged B-ALL, history of blinatumomab nonresponse, and HSCT-naive status. Most respondents indicated they would pursue HSCT for HSCT-naive, total body irradiation (TBI) recipients with BCR before 6 months post-tisa-cel or with NGS positivity at 1 or 3 months post-tisa-cel, although there was variability in responses regarding whether to proceed to HSCT directly or provide intervening therapy prior to HSCT. Fewer respondents recommended HSCT for BCR or NGS positivity in patients with a history of HSCT, in noncandidates for TBI, and in patients with trisomy 21. The results of this survey indicate there exists significant practice variability regarding the use of cHSCT, as well as interventions for post-tisa-cel BCR or NGS positivity. These results highlight areas in which ongoing clinical trials could inform more standardized practice.
MeSH term(s)	Child ; Humans ; Young Adult ; Hematopoietic Stem Cell Transplantation/methods ; Receptors, Antigen, T-Cell/therapeutic use ; Precursor B-Cell Lymphoblastic Leukemia-Lymphoma/therapy ; Precursor Cell Lymphoblastic Leukemia-Lymphoma/drug therapy ; Recurrence ; Burkitt Lymphoma
Chemical Substances	tisagenlecleucel (Q6C9WHR03O) ; Receptors, Antigen, T-Cell
Language	English
Publishing date	2023-10-08
Publishing country	United States
Document type	Journal Article
ZDB-ID	3062231-1
ISSN	2666-6367
ISSN (online)	2666-6367
DOI	10.1016/j.jtct.2023.10.004
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Zs.A 5082: Show issues

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Article ; Online: INSPIRED Symposium Part 3: Prevention and Management of Pediatric Chimeric Antigen Receptor T Cell-Associated Emergent Toxicities.

McNerney, Kevin O / Hsieh, Emily M / Shalabi, Haneen / Epperly, Rebecca / Wolters, Pamela L / Hill, Joshua A / Gardner, Rebecca / Talleur, Aimee C / Shah, Nirali N / Rossoff, Jenna

Transplantation and cellular therapy

2023 Volume 30, Issue 1, Page(s) 38–55

Abstract: Chimeric antigen receptor (CAR) T cell (CAR-T) therapy has emerged as a revolutionary cancer treatment modality, particularly in children and young adults with B cell malignancies. Through clinical trials and real-world experience, much has been learned ... ...

Abstract	Chimeric antigen receptor (CAR) T cell (CAR-T) therapy has emerged as a revolutionary cancer treatment modality, particularly in children and young adults with B cell malignancies. Through clinical trials and real-world experience, much has been learned about the unique toxicity profile of CAR-T therapy. The past decade brought advances in identifying risk factors for severe inflammatory toxicities, investigating preventive measures to mitigate these toxicities, and exploring novel strategies to manage refractory and newly described toxicities, infectious risks, and delayed effects, such as cytopenias. Although much progress has been made, areas needing further improvements remain. Limited guidance exists regarding initial administration of tocilizumab with or without steroids and the management of inflammatory toxicities refractory to these treatments. There has not been widespread adoption of preventive strategies to mitigate inflammation in patients at high risk of severe toxicities, particularly children. Additionally, the majority of research related to CAR-T toxicity prevention and management has focused on adult populations, with only a few pediatric-specific studies published to date. Given that children and young adults undergoing CAR-T therapy represent a unique population with different underlying disease processes, physiology, and tolerance of toxicities than adults, it is important that studies be conducted to evaluate acute, delayed, and long-term toxicities following CAR-T therapy in this younger age group. In this pediatric-focused review, we summarize key findings on CAR-T therapy-related toxicities over the past decade, highlight emergent CAR-T toxicities, and identify areas of greatest need for ongoing research.
MeSH term(s)	Humans ; Child ; Receptors, Chimeric Antigen/therapeutic use ; Receptors, Antigen, T-Cell ; T-Lymphocytes ; Immunotherapy, Adoptive/adverse effects ; Risk Factors
Chemical Substances	Receptors, Chimeric Antigen ; Receptors, Antigen, T-Cell
Language	English
Publishing date	2023-10-10
Publishing country	United States
Document type	Journal Article ; Review
ZDB-ID	3062231-1
ISSN	2666-6367
ISSN (online)	2666-6367
DOI	10.1016/j.jtct.2023.10.006
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Article ; Online: A Population-Based Study of the Long-Term Risk of Infections Associated With Hospitalization in Childhood Cancer Survivors.

Chehab, Leena / Doody, David R / Esbenshade, Adam J / Guilcher, Gregory M T / Dvorak, Christopher C / Fisher, Brian T / Mueller, Beth A / Chow, Eric J / Rossoff, Jenna

Journal of clinical oncology : official journal of the American Society of Clinical Oncology

2022 Volume 41, Issue 2, Page(s) 364–372

Abstract: Purpose: Infections pose a significant risk during therapy for childhood cancer. However, little is known about the risk of infection in long-term survivors of childhood cancer.: Methods: We performed a retrospective observational study of children ... ...

Abstract	Purpose: Infections pose a significant risk during therapy for childhood cancer. However, little is known about the risk of infection in long-term survivors of childhood cancer. Methods: We performed a retrospective observational study of children and adolescents born in Washington State diagnosed with cancer before age 20 years and who survived at least 5 years after diagnosis. Survivors were categorized as having a hematologic or nonhematologic malignancy and were matched to individuals without cancer in the state birth records by birth year and sex with a comparator:survivor ratio of 10:1. The primary outcome was incidence of any infection associated with a hospitalization using diagnostic codes from state hospital discharge records. Incidence was reported as a rate (IR) per 1,000 person-years. Multivariate Poisson regression was used to calculate incidence rate ratios (IRR) for cancer survivors versus comparators. Results: On the basis of 382 infection events among 3,152 survivors and 771 events among 31,519 comparators, the IR of all hospitalized infections starting 5 years after cancer diagnosis was 12.6 (95% CI, 11.4 to 13.9) and 2.4 (95% CI, 2.3 to 2.6), respectively, with an IRR 5.1 (95% CI, 4.5 to 5.8). The survivor IR during the 5- to 10-year (18.1, 95% CI, 15.9 to 20.5) and > 10-year postcancer diagnosis (8.3, 95% CI, 7.0 to 9.7) periods remained greater than comparison group IRs for the same time periods (2.3, 95% CI, 2.1 to 2.6 and 2.5, 95% CI, 2.3 to 2.8, respectively). When potentially vaccine-preventable infections were evaluated, survivors had a greater risk of infection relative to comparators (IRR, 13.1; 95% CI, 7.2 to 23.9). Conclusion: Infectious complications continue to affect survivors of childhood cancer many years after initial diagnosis. Future studies are needed to better understand immune reconstitution to determine specific factors that may mitigate this risk.
MeSH term(s)	Adolescent ; Humans ; Child ; Young Adult ; Adult ; Cancer Survivors ; Neoplasms/epidemiology ; Neoplasms/therapy ; Survivors ; Hospitalization ; Retrospective Studies ; Risk Factors
Language	English
Publishing date	2022-07-25
Publishing country	United States
Document type	Observational Study ; Journal Article ; Research Support, U.S. Gov't, P.H.S. ; Research Support, Non-U.S. Gov't
ZDB-ID	604914-x
ISSN	1527-7755 ; 0732-183X
ISSN (online)	1527-7755
ISSN	0732-183X
DOI	10.1200/JCO.22.00230
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Zs.A 1847: Show issues			Location: Je nach Verfügbarkeit (siehe Angabe bei Bestand) bis Jg. 1994: Bestellungen von Artikeln über das Online-Bestellformular Jg. 1995 - 2021: Lesesall (1.OG) ab Jg. 2022: Lesesaal (EG)
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Article ; Online: Access to Technology and Preferences for an mHealth Intervention to Promote Medication Adherence in Pediatric Acute Lymphoblastic Leukemia: Approach Leveraging Behavior Change Techniques.

Heneghan, Mallorie B / Hussain, Tasmeen / Barrera, Leonardo / Cai, Stephanie W / Haugen, Maureen / Morgan, Elaine / Rossoff, Jenna / Weinstein, Joanna / Hijiya, Nobuko / Cella, David / Badawy, Sherif M

Journal of medical Internet research

2021 Volume 23, Issue 2, Page(s) e24893

Abstract: Background: Suboptimal adherence to 6-mercaptopurine (6-MP) is prevalent in pediatric acute lymphoblastic leukemia (ALL) and associated with increased risk of relapse. Rapid uptake of personal technology makes mobile health (mHealth) an attractive ... ...

Abstract	Background: Suboptimal adherence to 6-mercaptopurine (6-MP) is prevalent in pediatric acute lymphoblastic leukemia (ALL) and associated with increased risk of relapse. Rapid uptake of personal technology makes mobile health (mHealth) an attractive platform to promote adherence. Objective: Study objectives were to examine access to mobile technology and preferences for an mHealth intervention to improve medication adherence in pediatric ALL. Methods: A cross-sectional survey was administered in oncology clinic to parents of children with ALL as well as adolescents and young adults (AYAs) with ALL receiving maintenance chemotherapy. Results: A total of 49 parents (median age [IQR] 39 [33-42] years; female 76% [37/49]) and 15 patients (median age [IQR] 17 [16-19]; male 80% [12/15]) participated. All parents and AYAs owned electronic tablets, smartphones, or both. Parents' most endorsed mHealth app features included a list of medications (71%, 35/49), information about 6-MP (71%, 35/49), refill reminders (71%, 35/49), and reminders to take 6-MP (71%, 35/49). AYAs' most endorsed features included refill reminders (73%, 11/15), reminders to take 6-MP (73%, 11/15), and tracking 6-MP (73%, 11/15). Conclusions: Parents and AYAs reported ubiquitous access to mobile technology and strong interest in multiple adherence-specific mHealth app features. Parents and AYAs provided valuable insight into preferred features for a multifunctional behavioral intervention (mHealth app) to promote medication adherence in pediatric ALL.
MeSH term(s)	Adolescent ; Adult ; Behavior Therapy/methods ; Child ; Child, Preschool ; Cross-Sectional Studies ; Female ; Humans ; Male ; Medication Adherence/statistics & numerical data ; Mobile Applications/statistics & numerical data ; Precursor Cell Lymphoblastic Leukemia-Lymphoma/therapy ; Smartphone ; Surveys and Questionnaires ; Technology/methods ; Telemedicine/methods ; Young Adult
Language	English
Publishing date	2021-02-18
Publishing country	Canada
Document type	Journal Article ; Research Support, N.I.H., Extramural ; Research Support, U.S. Gov't, P.H.S.
ZDB-ID	2028830-X
ISSN	1438-8871 ; 1439-4456
ISSN (online)	1438-8871
ISSN	1439-4456
DOI	10.2196/24893
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Article ; Online: HLH-like toxicities predict poor survival after the use of tisagenlecleucel in children and young adults with B-ALL.

McNerney, Kevin O / Si Lim, Stephanie J / Ishikawa, Kyle / Dreyzin, Alexandra / Vatsayan, Anant / Chen, John J / Baggott, Christina / Prabhu, Snehit / Pacenta, Holly L / Philips, Christine / Rossoff, Jenna / Stefanski, Heather E / Talano, Julie-An / Moskop, Amy / Verneris, Michael / Myers, Doug / Karras, Nicole A / Brown, Patrick / Bonifant, Challice L /

Qayed, Muna / Hermiston, Michelle / Satwani, Prakash / Krupski, Christa / Keating, Amy K / Baumeister, Susanne H C / Fabrizio, Vanessa A / Chinnabhandar, Vasant / Egeler, Emily / Mavroukakis, Sharon / Curran, Kevin J / Mackall, Crystal L / Laetsch, Theodore W / Schultz, Liora M

Blood advances

2023 Volume 7, Issue 12, Page(s) 2758–2771

Abstract: Chimeric antigen receptor-associated hemophagocytic lymphohistiocytosis (HLH)-like toxicities (LTs) involving hyperferritinemia, multiorgan dysfunction, coagulopathy, and/or hemophagocytosis are described as occurring in a subset of patients with ... ...

Abstract	Chimeric antigen receptor-associated hemophagocytic lymphohistiocytosis (HLH)-like toxicities (LTs) involving hyperferritinemia, multiorgan dysfunction, coagulopathy, and/or hemophagocytosis are described as occurring in a subset of patients with cytokine release syndrome (CRS). Case series report poor outcomes for those with B-cell acute lymphoblastic leukemia (B-ALL) who develop HLH-LTs, although larger outcomes analyses of children and young adults (CAYAs) with B-ALL who develop these toxicities after the administration of commercially available tisagenlecleucel are not described. Using a multi-institutional database of 185 CAYAs with B-ALL, we conducted a retrospective cohort study including groups that developed HLH-LTs, high-grade (HG) CRS without HLH-LTs, or no to low-grade (NLG) CRS without HLH-LTs. Primary objectives included characterizing the incidence, outcomes, and preinfusion factors associated with HLH-LTs. Among 185 CAYAs infused with tisagenlecleucel, 26 (14.1%) met the criteria for HLH-LTs. One-year overall survival and relapse-free survival were 25.7% and 4.7%, respectively, in those with HLH-LTs compared with 80.1% and 57.6%, respectively, in those without. In multivariable analysis for death, meeting criteria for HLH-LTs carried a hazard ratio of 4.61 (95% confidence interval, 2.41-8.83), controlling for disease burden, age, and sex. Patients who developed HLH-LTs had higher pretisagenlecleucel disease burden, ferritin, and C-reactive protein levels and lower platelet and absolute neutrophil counts than patients with HG- or NLG-CRS without HLH-LTs. Overall, CAYAs with B-ALL who developed HLH-LTs after tisagenlecleucel experienced high rates of relapse and nonrelapse mortality, indicating the urgent need for further investigations into prevention and optimal management of patients who develop HLH-LTs after tisagenlecleucel.
MeSH term(s)	Humans ; Child ; Young Adult ; Lymphohistiocytosis, Hemophagocytic/etiology ; Retrospective Studies ; Receptors, Antigen, T-Cell ; Receptors, Chimeric Antigen ; Precursor B-Cell Lymphoblastic Leukemia-Lymphoma/complications ; Burkitt Lymphoma/complications ; Chronic Disease
Chemical Substances	tisagenlecleucel (Q6C9WHR03O) ; Receptors, Antigen, T-Cell ; Receptors, Chimeric Antigen
Language	English
Publishing date	2023-01-09
Publishing country	United States
Document type	Journal Article ; Research Support, N.I.H., Extramural ; Research Support, U.S. Gov't, Non-P.H.S.
ZDB-ID	2915908-8
ISSN	2473-9537 ; 2473-9529
ISSN (online)	2473-9537
ISSN	2473-9529
DOI	10.1182/bloodadvances.2022008893
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Zs.A 7153: Show issues

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Article ; Online: Tisagenlecleucel utilisation and outcomes across refractory, first relapse and multiply relapsed B-cell acute lymphoblastic leukemia: a retrospective analysis of real-world patterns.

Barsan, Valentin / Li, Yimei / Prabhu, Snehit / Baggott, Christina / Nguyen, Khanh / Pacenta, Holly / Phillips, Christine L / Rossoff, Jenna / Stefanski, Heather / Talano, Julie-An / Moskop, Amy / Baumeister, Susanne / Verneris, Michael R / Myers, Gary Douglas / Karras, Nicole A / Cooper, Stacy / Qayed, Muna / Hermiston, Michelle / Satwani, Prakash /

Krupski, Christa / Keating, Amy / Fabrizio, Vanessa / Chinnabhandar, Vasant / Kunicki, Michael / Curran, Kevin J / Mackall, Crystal L / Laetsch, Theodore W / Schultz, Liora M

EClinicalMedicine

2023 Volume 65, Page(s) 102268

Abstract: Background: Tisagenlecleucel was approved by the Food and Drug Administration (FDA) in 2017 for refractory B-cell acute lymphoblastic leukemia (B-ALL) and B-ALL in ≥2nd relapse. Outcomes of patients receiving commercial tisagenlecleucel upon 1st relapse ...

Abstract	Background: Tisagenlecleucel was approved by the Food and Drug Administration (FDA) in 2017 for refractory B-cell acute lymphoblastic leukemia (B-ALL) and B-ALL in ≥2nd relapse. Outcomes of patients receiving commercial tisagenlecleucel upon 1st relapse have yet to be established. We aimed to report real-world tisagenlecleucel utilisation patterns and outcomes across indications, specifically including patients treated in 1st relapse, an indication omitted from formal FDA approval. Methods: We conducted a retrospective analysis of real-world tisagenlecleucel utilisation patterns across 185 children and young adults treated between August 30, 2017 and March 6, 2020 from centres participating in the Pediatric Real-World CAR Consortium (PRWCC), within the United States. We described definitions of refractory B-ALL used in the real-world setting and categorised patients by reported Chimeric Antigen Receptor (CAR) T-cell indication, including refractory, 1st relapse and ≥2nd relapse B-ALL. We analysed baseline patient characteristics and post-tisagenlecleucel outcomes across defined cohorts. Findings: Thirty-six percent (n = 67) of our cohort received tisagenlecleucel following 1st relapse. Of 66 evaluable patients, 56 (85%, 95% CI 74-92%) achieved morphologic complete response. Overall-survival (OS) and event-free survival (EFS) at 1-year were 69%, (95% CI 58-82%) and 49%, (95% CI 37-64%), respectively, with survival outcomes statistically comparable to remaining patients (OS; Interpretation: Our findings suggested that tisagenlecleucel response and survival rates overlap across patients treated with upfront refractory B-ALL, B-ALL ≥2nd relapse and B-ALL in 1st relapse. We additionally highlighted that definitions of refractory B-ALL are evolving beyond morphologic measures of residual disease. Funding: St. Baldrick's/Stand Up 2 Cancer, Parker Institute for Cancer Immunotherapy, Virginia and D.K. Ludwig Fund for Cancer Research.
Language	English
Publishing date	2023-10-26
Publishing country	England
Document type	Journal Article
ISSN	2589-5370
ISSN (online)	2589-5370
DOI	10.1016/j.eclinm.2023.102268
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Article: The diagnosis, classification, and management of asthma according to severity.

Rossoff, L J

The American journal of managed care

1997 Volume 3, Issue 2, Page(s) 309–15; quiz 318, 320

Abstract: Unlabelled: This activity is designed for primary care and specialist physicians.: Goal: To provide prompt and appropriate treatment for asthma of all levels of severity resulting in improved level of activity and decreased need for urgent care and ... ...

Abstract	Unlabelled: This activity is designed for primary care and specialist physicians. Goal: To provide prompt and appropriate treatment for asthma of all levels of severity resulting in improved level of activity and decreased need for urgent care and hospitalization with a possible reduction in the annual decline of lung function, degree of permanent airway damage, and mortality. Objectives: 1. To provide a framework on the basis of history, physical findings, and laboratory results for the diagnosis of asthma. 2. To improve the ability to classify asthma by degree of severity. 3. To describe an incremental therapeutic approach to asthma by degree of severity. 4. To provide a systematic approach with regard to periodic reevaluation of asthma severity and modification of the treatment plan.
MeSH term(s)	Anti-Inflammatory Agents/therapeutic use ; Asthma/classification ; Asthma/diagnosis ; Asthma/epidemiology ; Asthma/therapy ; Bronchodilator Agents/therapeutic use ; Clinical Laboratory Techniques ; Education, Continuing ; Humans ; Immunotherapy ; Managed Care Programs ; Physical Examination ; Practice Guidelines as Topic ; Risk Factors ; Severity of Illness Index ; Steroids ; United States/epidemiology
Chemical Substances	Anti-Inflammatory Agents ; Bronchodilator Agents ; Steroids
Language	English
Publishing date	1997-02
Publishing country	United States
Document type	Journal Article
ZDB-ID	2035781-3
ISSN	1936-2692 ; 1088-0224 ; 1096-1860
ISSN (online)	1936-2692
ISSN	1088-0224 ; 1096-1860
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Zs.A 5353: Show issues			Location: Je nach Verfügbarkeit (siehe Angabe bei Bestand) bis Jg. 1994: Bestellungen von Artikeln über das Online-Bestellformular Jg. 1995 - 2021: Lesesall (2.OG) ab Jg. 2022: Lesesaal (EG)
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Article ; Online: Applying the COM-B model to patient-reported barriers to medication adherence in pediatric acute lymphoblastic leukemia.

Heneghan, Mallorie B / Hussain, Tasmeen / Barrera, Leonardo / Cai, Stephanie W / Haugen, Maureen / Duff, Ashley / Shoop, Jenny / Morgan, Elaine / Rossoff, Jenna / Weinstein, Joanna / Hijiya, Nobuko / Cella, David / Badawy, Sherif M

Pediatric blood & cancer

2020 Volume 67, Issue 5, Page(s) e28216

Abstract: Background: Adherence to oral chemotherapy, including 6-mercaptopurine (6-MP), is suboptimal in pediatric acute lymphoblastic leukemia (ALL), which is associated with increased risk of relapse. Study objectives were to examine self-reported adherence to ...

Abstract	Background: Adherence to oral chemotherapy, including 6-mercaptopurine (6-MP), is suboptimal in pediatric acute lymphoblastic leukemia (ALL), which is associated with increased risk of relapse. Study objectives were to examine self-reported adherence to 6-MP and related barriers to adherence, mapped to the capability, opportunity, motivation, and behavior (COM-B) model for behavior change. Procedure: Forty-nine parents (median, 39 years old; 76% females) and 15 patients (median, 17 years old, 20% females) completed the study survey. Results: Suboptimal adherence was reported in 43% of parents and 73% of patients. Most parents and patients (80% and 90%, respectively) reported ≥1 adherence barrier. Parents reported difficulty helping their child meet others with ALL (43%), contacting community organizations (39%), and meeting other parents (37%). Patients reported difficulty finding out what their medications are (40%), finding out what 6-MP does (47%), and meeting other patients (40%). Using the COM-B, we found that parents and patients endorsed barriers in multiple components, especially physical (55%, 67%) and social opportunity (56%, 47%), highlighting that barriers to adherence may be multifaceted. Conclusions: Our results suggest that parents and patients with ALL face various prevalent barriers to medication adherence and provide insight into the development of behavioral interventions focused on promoting adherence, which is essential to prevent relapse and optimize health outcomes in ALL.
MeSH term(s)	Adolescent ; Child ; Child, Preschool ; Cross-Sectional Studies ; Female ; Humans ; Infant ; Male ; Medication Adherence/psychology ; Models, Psychological ; Precursor Cell Lymphoblastic Leukemia-Lymphoma/psychology ; Precursor Cell Lymphoblastic Leukemia-Lymphoma/therapy
Language	English
Publishing date	2020-02-18
Publishing country	United States
Document type	Journal Article ; Research Support, N.I.H., Extramural ; Research Support, U.S. Gov't, P.H.S.
ZDB-ID	2131448-2
ISSN	1545-5017 ; 1545-5009
ISSN (online)	1545-5017
ISSN	1545-5009
DOI	10.1002/pbc.28216
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Article ; Online: Experience with ponatinib in paediatric patients with leukaemia.

Rossoff, Jenna / Huynh, Van / Rau, Rachel E / Macy, Margaret E / Sulis, Maria L / Schultz, Kirk R / Burke, Michael J / Athale, Uma / O'Brien, Maureen M / Gregory, John J / van der Sluis, Inge M / Keller, Frank G / Zwaan, Christian M / Suttorp, Meinolf / Hijiya, Nobuko

British journal of haematology

2020 Volume 189, Issue 2, Page(s) 363–368

Abstract: Ponatinib has proven to be effective in adults with Philadelphia chromosome-positive leukaemias, but data in paediatrics are scarce. Among paediatric patients with chronic myeloid leukaemia (n = 9) or acute lymphoblastic leukaemia (n = 12) treated with ... ...

Abstract	Ponatinib has proven to be effective in adults with Philadelphia chromosome-positive leukaemias, but data in paediatrics are scarce. Among paediatric patients with chronic myeloid leukaemia (n = 9) or acute lymphoblastic leukaemia (n = 12) treated with varying doses of ponatinib in 13 centres, 71% showed a decrease in disease burden after a median of three months. Ponatinib was well tolerated, with grade 3 toxicities occurring in 29% of patients. Toxicities were similar to those reported in adults, with the exception of arterial thrombotic events, which were not observed. Ponatinib has a favourable safety profile in this paediatric cohort, but dose-finding studies are needed.
MeSH term(s)	Adolescent ; Adult ; Antineoplastic Agents/pharmacology ; Antineoplastic Agents/therapeutic use ; Child ; Female ; Humans ; Imidazoles/pharmacology ; Imidazoles/therapeutic use ; Male ; Precursor Cell Lymphoblastic Leukemia-Lymphoma/drug therapy ; Pyridazines/pharmacology ; Pyridazines/therapeutic use ; Young Adult
Chemical Substances	Antineoplastic Agents ; Imidazoles ; Pyridazines ; ponatinib (4340891KFS)
Language	English
Publishing date	2020-01-24
Publishing country	England
Document type	Journal Article
ZDB-ID	80077-6
ISSN	1365-2141 ; 0007-1048
ISSN (online)	1365-2141
ISSN	0007-1048
DOI	10.1111/bjh.16338
Database	MEDical Literature Analysis and Retrieval System OnLINE

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