Pediatric Blood & Cancer最新文献

{"title":"Treatment Experience Using a Micro-Induction Buprenorphine Protocol for Chronic Pain in Pediatric Sickle Cell Disease.","authors":"Ashwin Patel, Grace Kalmus, Carlton Dampier, Ifeyinwa Osunkwo, Tamara New, Beatrice Gee, Elna Saah","doi":"10.1002/pbc.31731","DOIUrl":"https://doi.org/10.1002/pbc.31731","url":null,"abstract":"<p><strong>Background: </strong>Patients with sickle cell disease (SCD) experience painful vaso-occlusive episodes that increase with age; a subset develops chronic pain (CP). CP is usually managed with acute pain management guidelines despite evidence of ineffectiveness. Buprenorphine (BUP), a partial opioid agonist, is a potent analgesic with less euphoric effect and a respiratory \"ceiling effect.\" BUP therefore provides an alternative \"harm reduction\" approach for CP management in pediatric SCD patients.</p><p><strong>Methods: </strong>This single urban center retrospective study assessed the feasibility of inpatient transition to BUP-containing analgesics in adolescents with SCD and CP. Patients aged 12-20 years who transitioned from full opioid agonists (FOA) to BUP between December 2020 and September 2022 were included. Acute care utilization, hospital length of stay, and FOA use in both inpatient and outpatient settings were compared pre- and post-BUP induction for up to a year.</p><p><strong>Results: </strong>Fourteen adolescents with SCD underwent inpatient BUP induction and maintenance therapy. Inpatient transition using a micro-induction approach was feasible and well tolerated in this population. There were low rates of adverse events, such as opioid withdrawal signs. Maintenance on BUP products was sustainable over the 1-year post-induction period. Three patients (21%) discontinued BUP during maintenance therapy. There was a significant reduction (p < 0.05) in acute care utilization, length of stay, and FOA use (both inpatient and outpatient).</p><p><strong>Conclusion: </strong>Inpatient micro-induction to BUP from FOA in adolescent SCD patients with CP is feasible with minimal signs of opioid withdrawal. This study suggests decreased acute care utilization with BUP.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31731"},"PeriodicalIF":2.4,"publicationDate":"2025-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144136205","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Wilms Tumor in Children with AMER1/WTX Germline Pathogenic Variants: A Multicenter Case Series.","authors":"Insiyah Campwala, Jaclyn Schienda, Andrew J Murphy, Basil Hashimi, Taylor Perry, Nicholas Cost, Junne Kamihara, Elizabeth A Mullen, Teresa Santiago, Marcus M Malek","doi":"10.1002/pbc.31798","DOIUrl":"https://doi.org/10.1002/pbc.31798","url":null,"abstract":"<p><strong>Background: </strong>10-15% of children with Wilms tumor (WT) have predisposing genetic syndromes. Somatic mutations are frequently identified; however, germline pathogenic variants in AMER1 are much less prevalent and are associated with osteopathia striata with cranial sclerosis (OSCS).</p><p><strong>Methods: </strong>A multicenter retrospective case series was conducted reviewing patients with AMER1 germline variants and WT from 2012 to 2023. Results were compared with published data from six other children.</p><p><strong>Results: </strong>Four female children were identified. Age at WT diagnosis ranged from 5 months to 8 years. One patient had familial AMER1 germline variant. Stage of disease ranged from I to IV, and three children required adjuvant therapy. Nephrogenic rests were noted in two patients. Two patients underwent open partial nephrectomy, and two underwent open radical nephrectomy. One patient had mild kidney disease post-resection, and no patients had recurrence or died from disease progression.</p><p><strong>Conclusion: </strong>Our cohort of four patients, combined with the six patients with WT and AMER1 pathogenic variants previously reported, with 20% (two out of 10) collective incidence of bilateral tumors, support AMER1 as a WT predisposition gene warranting surveillance. Collectively, age of WT diagnosis ranged from 5 months to 12 years, which demonstrates potential for prolonged risk. Pathogenic AMER1 germline variants were previously thought to have 100% penetrance; however, one of four current cases did not exhibit an OSCS phenotype. We report the first documented case of a familial AMER1 germline variant and WT. We conclude that nephron-sparing surgery and familial genetic testing should be considered for children with AMER1 germline variants and WT.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31798"},"PeriodicalIF":2.4,"publicationDate":"2025-05-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144120287","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Comparative Analysis of Clinical Utility of Cytogenetics and Copy-Number-Integrated Risk Stratification Scores in a Prospective Cohort of Pediatric B-Cell Acute Lymphoblastic Leukemia.","authors":"Ajmeera A Azeez, Prateek Bhatia, Sangeetha Kirubanandhan, Rozy Thakur, Minu Singh, Sandeep Rose, Meenakshi Malhotra, Sharun Garg, Sreejesh Sreedharanunni, Manupdesh S Sachdeva, Shelly Singla, Parminder Kaur, Richa Jain, Deepak Bansal, Amita Trehan","doi":"10.1002/pbc.31812","DOIUrl":"https://doi.org/10.1002/pbc.31812","url":null,"abstract":"<p><p>The present study compares and evaluates the clinical utility of three published copy-number alteration (CNA)- and cytogenetics-integrated risk group classifiers in a prospective cohort of pediatric B-cell acute lymphoblastic leukemia (B-ALL) patients. All cases underwent CNA testing via digital (n = 185) or conventional multiplex ligation probe amplification (MLPA; n = 22), and the data were integrated with genetics according to the three classifier systems. Out of 207 pediatric B-ALL patients enrolled, a primary genetic abnormality was noted in 87% (181/207) of the patients, with high hyperdiploidy being the most common (31%). The overall CNA frequency was 54% (112/207), with CDKN2A/2B deletion being most common (28.5%). Poor-risk CNAs in all three integrated risk classifiers correlated significantly with high TLC (>50 × 10⁹/L), NCI-HR, MRD>0.01%, ICiCLe-HR, and event. In addition, the two-year EFS and OS were worse for the UKALL-integrated poor-risk group (56.7%; p = <0.0001 and 68.3%; p = 0.012), whereas the digital MLPA-combined poor-risk group and the PersonALL IKZF1 high-risk group had poor two-year EFS (55.3%; p = 0.038) and (39.5%; p = 0.015), respectively. Multivariate analysis revealed the UKALL poor-risk integrated group (GEN-PR) to predict relapse (HR: 48.58; p = 0.014), thereby highlighting that the UKALL-CNA-integrated classifier is more likely to precisely identify a subset of intermediate-risk cytogenetic cases with intermediate- or poor-risk CNAs that require escalated treatment.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31812"},"PeriodicalIF":2.4,"publicationDate":"2025-05-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144120193","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Long-Term Projections of Childhood Cancer Incidence and Prevalence in Ontario, Canada Until 2040.","authors":"Alexandra Moskalewicz, Sumit Gupta, Petros Pechlivanoglou, Paul C Nathan","doi":"10.1002/pbc.31810","DOIUrl":"https://doi.org/10.1002/pbc.31810","url":null,"abstract":"<p><strong>Background: </strong>The prevalence of childhood cancer continues to rise due to increases in cancer incidence and advances in treatment, leading to better survival. We generated epidemiologic projections for childhood cancer, by cancer type, in Ontario, Canada, until 2040.</p><p><strong>Methods: </strong>We used the Pediatric Oncology Microsimulation Model for Prevalence (POSIM-Prev) to simulate incident and prevalent cases of childhood cancer across historical (1970-2019) and future (2020-2040) time periods. The model was utilized to estimate annual population-level projections of incidence (counts and crude rates per million children), overall survival rates, and limited-duration prevalence (counts and crude rates per 100,000 population) for 14 types of childhood cancer between 2020 and 2040.</p><p><strong>Results: </strong>Across future years, crude incidence rates are projected to increase for 10 cancer types in Ontario, with the largest growth expected for non-Hodgkin lymphomas. Crude prevalence rates are projected to rise between 2020 and 2040 for 13 cancer types and remain stable for bone tumors. While individuals diagnosed with lymphoid leukemia will continue to comprise the largest proportion of overall prevalence during this period, the largest relative increases in prevalence are estimated for those diagnosed with hepatic tumors and acute myeloid leukemia. By 2040, the percentage of prevalent individuals, by malignancy, who are expected to reach late adulthood (aged 60+) ranges from 4% (hepatic tumors) to 19% (bone tumors).</p><p><strong>Conclusion: </strong>Further increases in incidence and improvements in survival for several pediatric cancer types will contribute to substantially higher prevalence by 2040, with a projected shift toward older subpopulations.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31810"},"PeriodicalIF":2.4,"publicationDate":"2025-05-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144120280","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Prediction of B/T Subtype and ETV6-RUNX1 Translocation in Pediatric Acute Lymphoblastic Leukemia by Deep Learning Analysis of Giemsa-Stained Whole Slide Images of Bone Marrow Aspirates.","authors":"Arkadi Piven, Gil Shamai, Sarah Elitzur, Galit Pinto Berger, Yoav Binenbaum, Ron Kimmel, Ronit Elhasid","doi":"10.1002/pbc.31797","DOIUrl":"https://doi.org/10.1002/pbc.31797","url":null,"abstract":"<p><strong>Background: </strong>Accurate determination of B/T-cell lineage and the presence of the ETV6-RUNX1 translocation is critical for diagnosing acute lymphoblastic leukemia (ALL), as these factors influence treatment decisions and outcomes. However, these diagnostic processes often rely on advanced tools unavailable in low-resource settings, creating a need for alternative solutions.</p><p><strong>Procedure: </strong>We developed a deep learning pipeline to analyze Giemsa-stained bone marrow (BM) aspirate smears. The models were trained to distinguish between ALL, acute myeloid leukemia (AML), and non-leukemic BM samples, predict B- and T-cell lineage in ALL, and detect the presence of the ETV6-RUNX1 translocation. The performance was evaluated using cross-validation (CV) and an external validation cohort.</p><p><strong>Results: </strong>The models achieved a statistically significant area under the curve (AUC) of 0.99 in distinguishing ALL from AML and control samples. In cross-validation (CV), the models achieved a cross-validation AUC of 0.74 for predicting B/T subtypes. For predicting ETV6-RUNX1 translocation, the models achieved an AUC of 0.80. External cohort validation confirmed significant AUCs of 0.72 for B/T subtype classification and 0.69 for ETV6-RUNX1 translocation prediction.</p><p><strong>Conclusions: </strong>Convolutional neural networks (CNNs) demonstrate potential as a diagnostic tool for pediatric ALL, enabling the identification of B/T lineage and ETV6-RUNX1 translocation from Giemsa-stained smears. These results pave the way for future utilization of CNNs as a diagnostic modality for pediatric leukemia in low-resource settings, where access to advanced diagnostic techniques is limited.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31797"},"PeriodicalIF":2.4,"publicationDate":"2025-05-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144120282","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Treatment-Related Risk Factors for Diabetes Mellitus in Childhood Cancer Survivors-A Case-Cohort Study Within Adult Life After Childhood Cancer in Scandinavia (ALiCCS).","authors":"Line Kenborg, Peter H Asdahl, Smita Bhatia, Jane Christensen, Thorgerdur Gudmundsdottir, Lars Hjorth, Rebecca Howell, Anja Krøyer, Morten Jørgensen, Michael R T Laursen, Yasmin Lassen-Ramshad, Sofie de Fine Licht, Thomas Tjørnelund Nielsen, Catherine Rechnitzer, Susan A Smith, Thomas Wiebe, Henrik Hasle, Anna S Holmqvist","doi":"10.1002/pbc.31805","DOIUrl":"https://doi.org/10.1002/pbc.31805","url":null,"abstract":"<p><p>The aim was to identify treatment-related risk factors for diabetes in 5-year survivors of childhood cancer (CCS). This case-cohort study within Adult Life after Childhood Cancer in Scandinavia (ALiCCS) included 140 CCS with diabetes and a subcohort of 390 randomly selected CCS. Incidence rate ratios (IRRs) and 95% confidence intervals (CIs) were calculated to assess the association between treatment exposures and diabetes. Radiotherapy was the most important risk factor, with increased IRR after total body irradiation (TBI) (9.8, 95% CI = 2.9-33.7), abdominal radiation (4.8, 95% CI = 2.2-10.4), and cranial radiation (4.3, 95% CI = 2.1-8.8). Continued follow-up with regard to diabetes is indicated after TBI, abdominal radiation, and cranial radiation in CCS.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31805"},"PeriodicalIF":2.4,"publicationDate":"2025-05-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144120285","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Contemporary Biology, Management, and Outcomes of Renal Medullary Carcinoma in Children and Adults: A Pediatric Surgical Oncology Research Collaborative Study.","authors":"Catherine B Beckhorn, Marcus M Malek, Harold N Lovvorn, Harold J Leraas, Katlyn G McKay, Nelly-Ange T Kontchou, Zachary J Kastenberg, David W Hoyt, Jonathan P Roach, Emily K Myers, Nicholas G Cost, Bhargava Mullapudi, Charles R Marchese, Amanda R Jensen, Timothy B Lautz, Michela Carter, Roshni Dasgupta, John Lundstedt, Joseph G Brungardt, Lindsay J Talbot, Andrew M Davidoff, Andrew J Murphy, Jennifer H Aldrink, Sara A Mansfield, Nelson Piché, Dave R Lal, Brian T Craig, Jennifer M Schuh, Barrett P Cromeens, Sindhu V Mannava, Shannon L Castle, Adriana Lopez, Kelsey Mello, Joshua Short, Robin T Petroze, Shay Rajaval, Grace R Thompson, Peter Mattei, David H Rothstein, Elizabeth Fialkowski, Kathryn L Fowler, Nathan Martchenke, Barrie S Rich, Richard D Glick, Erin G Brown, Kathleen Doyle, Paige Abril, Hannah N Rinehardt, Natashia M Seemann, Jacob Davidson, Claire A Wilson, Hau D Le, Devashish Joshi, Michael Stellon, Tamer Ahmed, Alexandra M Dimmer, Erika A Newman, Maya Hammoud, Keyonna Williams, Christa N Grant, Merit Gorgy, Stephanie F Polites, Julia Debertin, Danielle B Cameron, Alyssa Stetson, Eugene S Kim, William G Lee, Aaron Barkhordar, Mary T Austin, Brian A Coakley, Anastasia Kahan, Joseph T Murphy, Michael Pitonak, Chloé Boehmer, Elisabeth T Tracy","doi":"10.1002/pbc.31774","DOIUrl":"https://doi.org/10.1002/pbc.31774","url":null,"abstract":"<p><strong>Background: </strong>Renal medullary carcinoma (RMC) is an aggressive tumor associated with sickle cell trait. Despite treatment advances for other rare renal tumors, RMC survival remains poor. We aimed to describe the contemporary management and survival of children and adults with RMC.</p><p><strong>Procedure: </strong>In this multicenter retrospective cohort study, Pediatric Surgical Oncology Research Collaborative sites searched their databases for patients diagnosed with RMC (2000-2022). Descriptive statistics were calculated and survival analyses performed using Kaplan-Meier and Cox regression.</p><p><strong>Results: </strong>Thirty-four patients with RMC were identified. Median age was 19 years (IQR: 15-28; range: 7-52). Most were male (24/34; 71%), Black (27/32; 84%), had sickle cell trait or disease (30/33; 91%), presented with metastatic disease (27/34; 79%), and were symptomatic at presentation (32/34; 94%). Median overall survival (OS) was 24 months from diagnosis (16 months for children, 28 months for adults, p = 0.6). Receipt of platinum-based chemotherapy (23/34; 68%) was associated with significantly higher OS than other regimens (35 vs. 5 months, p < 0.001). Nephrectomy (24/34; 71%) was associated with significantly improved OS compared with non-operative management (34 vs. 7 months, p = 0.001). Immunotherapy, targeted therapy, or radiation therapy were not associated with significant differences in OS, nor were age, sex, race, sickle cell status, SMARCB1/INI-1, stage, nephrectomy approach, retroperitoneal lymph node dissection, gross residual disease, margins, or tumor size.</p><p><strong>Conclusions: </strong>RMC survival remains poor despite newer therapies. Nephrectomy and platinum-based chemotherapy should be considered in locally advanced and metastatic disease. Coordinated international cooperative group studies are needed to meaningfully improve RMC survival.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31774"},"PeriodicalIF":2.4,"publicationDate":"2025-05-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144111524","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Therapeutic Response to Imatinib in Bilateral Infiltrative and Leukostatic Retinopathy in Chronic Myelogenous Leukaemia.","authors":"Anamika Patel, Ilaria Testi, David O'Connor, Ameenat Lola Solebo","doi":"10.1002/pbc.31801","DOIUrl":"https://doi.org/10.1002/pbc.31801","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31801"},"PeriodicalIF":2.4,"publicationDate":"2025-05-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144110112","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Malignant Peripheral Nerve Sheath Tumor in Children and Adolescents: Local Treatment in a Retrospective Single-Center Experience.","authors":"Andrea Ferrari, Luca Bergamaschi, Stefano Chiaravalli, Marco Fiore, Chiara Colombo, Emilia Pecori, Arianna Trovò, Carlo Morosi, Roberto Luksch, Monica Terenziani, Filippo Spreafico, Cristina Meazza, Marta Podda, Veronica Biassoni, Elisabetta Schiavello, Nadia Puma, Giovanna Gattuso, Giovanna Sironi, Olga Nigro, Valeria Colombo, Patrizia Gasparini, Sandro Pasquali, Maura Massimino, Michela Casanova, Sabina Vennarini","doi":"10.1002/pbc.31813","DOIUrl":"https://doi.org/10.1002/pbc.31813","url":null,"abstract":"<p><strong>Background: </strong>Malignant peripheral nerve sheath tumor (MPNST) is a rare and aggressive sarcoma often associated with neurofibromatosis type 1, whose clinical management remains complex and challenging. Few publications exist on pediatric MPNST, and limited data are available on the best treatment approach, in particular regarding local therapy.</p><p><strong>Methods: </strong>This retrospective analysis concerned 45 patients less than 18 years old with MPNST, treated at a referral center for pediatric sarcomas from 1983 to 2023. Patients were treated using a multimodal approach, based on the protocols adopted at the time of their diagnosis.</p><p><strong>Results: </strong>For the series as a whole, the median event-free survival (EFS) and overall survival (OS) were 16 and 26 months, respectively, and 5-year EFS and OS were 28.8% and 40.1%. The first event was local failure in 18 cases, local failure plus metastases in nine cases, and metastases-only in four cases. At univariable analysis, survival was better for males and patients younger than 15 years, and was influenced by tumor invasiveness and tumor size. With regard to treatment modalities, survival rates were significantly better for patients who responded to chemotherapy; EFS, local relapse-free survival (LRFS), and OS were better for patients who had a surgical resection; EFS and LRFS were better for patients who received radiotherapy combined with surgery, while OS was better for patients who had R0 resection.</p><p><strong>Conclusions: </strong>Our study confirmed the unsatisfactory outcome of MPNST pediatric patients. Our series would suggest that a combined local treatment that included both surgical resection and radiotherapy could improve local control.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31813"},"PeriodicalIF":2.4,"publicationDate":"2025-05-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144111645","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Describing Outpatient Physical Therapy Use Among Adolescents and Young Adults With Sickle Cell Disease: A Single-Center Retrospective Study.","authors":"Ben Reader, Jason Benedict, Alexander Rospert, Kathleen Lemanek, Susan Creary","doi":"10.1002/pbc.31809","DOIUrl":"https://doi.org/10.1002/pbc.31809","url":null,"abstract":"<p><strong>Background: </strong>Physical impairments and functional limitations are common for individuals with sickle cell disease (SCD), leading to poor health-related quality of life (HR-QoL). Physical therapists provide interventions that restore functional capacity and promote independence, but it is unclear if these services are utilized by youth with SCD. This study aimed to describe physical therapy (PT) referral patterns and attendance rates among adolescents and young adults (AYA) with SCD, while also exploring if referral and receipt of outpatient PT were associated with age or HR-QoL.</p><p><strong>Methods: </strong>This retrospective chart review assessed youth aged ≥13 years who attended one or more multidisciplinary SCD clinic appointment between Septmenber 2010 and September 2023 where they completed one or more HR-QoL assessments. Demographic, PT referral and attendance, and HR-QoL data were collected from the electronic health record.</p><p><strong>Results: </strong>A total of 150 AYA with SCD met inclusion criteria, and 40% (n = 60) were referred for outpatient PT. Primary reasons for referral included joint pain/issues (50%), deconditioning (20%), and unspecific chronic pain (12%). Of those referred for outpatient PT, 61% (n = 37) completed one or more PT visits. Age did not predict referral (p = 0.12) or attendance (p = 0.81). Having a lower physical functioning HR-QoL score was associated with being referred for PT (p < 0.001) but not with attending PT (p = 0.26).</p><p><strong>Conclusion: </strong>AYAs with SCD are frequently referred to outpatient PT for a range of functional impairments and physical limitations. Findings suggest that AYA with SCD may face significant barriers to attending outpatient PT, and future studies are needed to identify and mitigate these challenges.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31809"},"PeriodicalIF":2.4,"publicationDate":"2025-05-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144111642","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}