Pediatric Blood & Cancer最新文献

{"title":"Impact of Food Insecurity on Malnutrition Treatment Response in Nigerian Children With Sickle Cell Anemia and Severe Acute Malnutrition.","authors":"Gabriela Ramirez-Cuebas, Shehu Umar Abdullahi, Safiya Gambo, Hassan Adam Murtala, Halima Kabir, Khadija A Shamsu, Garba Gwarzo, Sari A Acra, Virginia A Stallings, Mark Rodeghier, Michael R DeBaun, Lauren J Klein","doi":"10.1002/pbc.31637","DOIUrl":"10.1002/pbc.31637","url":null,"abstract":"<p><strong>Background: </strong>In this planned ancillary analysis of our completed clinical trial, we hypothesized that among older children with sickle cell anemia (SCA) and severe acute malnutrition, those with higher levels of food insecurity would have lower end-of-trial body mass index (BMI) z-scores compared to their peers with SCA and lower levels of food insecurity.</p><p><strong>Procedure: </strong>Data from 108 children who completed the feasibility trial for managing severe acute malnutrition in older children with SCA in Nigeria were analyzed. Children aged 5-12 years old with severe acute malnutrition (BMI z-score of <-3.0) were randomly allocated to receive either supplemental ready-to-use therapeutic food (RUTF) alone or RUTF with moderate-dose hydroxyurea (20 mg/kg/day). Caregivers completed the United States Household Food Security Survey Module to measure food security. We focused on the childhood section for its accuracy in assessing food security in older children. Higher scores (0-8) indicate greater food insecurity. We constructed multivariable linear regression models to estimate the association between childhood food insecurity and BMI z-scores at baseline and endpoint.</p><p><strong>Results: </strong>Most participants were food insecure, with 55% (n = 59) and 34% (n = 37) having low and very low food security, respectively. Higher scores on the continuous food security measure, indicating lower food security, were associated with lower BMI z-scores at both study entry (β = -0.05, p = 0.047) and after malnutrition treatment (β = -0.07, p = 0.016).</p><p><strong>Conclusions: </strong>Among severely malnourished children with SCA, lower childhood food security scores are associated with an adverse treatment response, reflected by a lower BMI z-score at the trial's end.</p><p><strong>Url and trial identification number: </strong>NCT03634488, https://clinicaltrials.gov/study/NCT03634488.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31637"},"PeriodicalIF":2.4,"publicationDate":"2025-03-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143586719","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":"Venetoclax Combined With Chemotherapy in Pediatric and Adolescent/Young Adult Patients With Relapsed/Refractory Acute Lymphoblastic Leukemia.","authors":"Andrew E Place, Seth E Karol, Christopher J Forlenza, Todd M Cooper, Christopher Fraser, Gunnar Cario, Maureen M O'Brien, Nicolas U Gerber, Jean-Pierre Bourquin, Dirk Reinhardt, Jeffrey E Rubnitz, Joseph T Opferman, Gauri Sunkersett, Maika Onishi, Diana R Dunshee, Xin Chen, Kristina Unnebrink, Deeksha Vishwamitra, Fengjiao Dunbar, Mohamed Badawi, Jeremy A Ross, Mignon L Loh","doi":"10.1002/pbc.31630","DOIUrl":"https://doi.org/10.1002/pbc.31630","url":null,"abstract":"<p><strong>Background: </strong>Acute lymphoblastic leukemia (ALL) is the most common childhood cancer, and although many patients respond to induction therapy, those who relapse or have refractory disease face a poor prognosis. Venetoclax has promising preclinical and clinical activity in ALL. Here, we report the safety and preliminary efficacy of venetoclax combined with chemotherapy in pediatric and adolescent/young adult patients with relapsed/refractory ALL.</p><p><strong>Procedure: </strong>This phase 1, open-label, two-part, multicenter study evaluated venetoclax combined with chemotherapy in pediatric and adolescent/young adult patients (<25 years of age) with relapsed/refractory ALL. The study is registered with ClinicalTrials.gov, NCT03236857.</p><p><strong>Results: </strong>Thirty-one patients were treated and received venetoclax monotherapy (n = 1), venetoclax plus dexamethasone and/or vincristine and/or pegasparaginase (VXL; n = 20) or venetoclax plus cytarabine and/or etoposide and/or pegasparaginase (n = 10). Patients were heavily pretreated, with a median of 3 prior lines of therapy. The most common grade 3/4 treatment-emergent adverse event was febrile neutropenia (55%). One fatal adverse event possibly related to venetoclax occurred. The overall response rate of treated patients was 42%, with all responding patients achieving complete remission/complete remission with incomplete marrow recovery. In biomarker-evaluable patients, responses to venetoclax plus VXL-based or cytarabine-based chemotherapy were observed in patients harboring a range of genetic alterations and heterogeneous BH3 family member dependencies.</p><p><strong>Conclusions: </strong>Venetoclax plus VXL-based or cytarabine-based chemotherapy was overall well tolerated, with promising preliminary efficacy.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31630"},"PeriodicalIF":2.4,"publicationDate":"2025-03-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143586439","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":"Insights Into the Caregiver Experience for Pediatric Patients With Sickle Cell Disease in Saudi Arabia: Demographic Profiles, Care Recipient Characteristics, and Subjective Well-Being.","authors":"Emad Shdaifat","doi":"10.1002/pbc.31650","DOIUrl":"https://doi.org/10.1002/pbc.31650","url":null,"abstract":"<p><strong>Objective: </strong>This study aimed to examine the demographic characteristics of informal caregivers of pediatric patients with sickle cell disease. The background characteristics of both caregivers and care recipients were analyzed, and the informal care situation was examined. Additionally, this study aimed to quantify the economic impact of care.</p><p><strong>Methods: </strong>This cross-sectional study was conducted in the eastern region of Saudi Arabia, involving a sample of 27 caregivers of pediatric patients diagnosed with sickle cell disease. The survey instruments comprised the iMAT Valuation of Informal Care Questionnaire (IVICQ) to evaluate informal care costs, along with the Katz Index of Independence in activities of daily living (ADL) to assess the functional abilities of the care recipients.</p><p><strong>Results: </strong>Most participants were female caregivers (81.5%) and married (74.1%). The average age of the caregiver members was 38.0 years. Those in need of care, predominantly daughters or sons (85.2%), had a positive health outlook (average score: 6.85) and moderate independence (average ADL score: 4.70). The CarerQol-7D indicated variable perceived quality of life, with fulfillment in caregiving tasks (74.1%) and minimal relationship problems (74.1%). Economically, caregivers invest 245 h per week, which costs SAR 13,805 (3681 USD). The total opportunity cost was SAR 806 (215 USD) per caregiver per week.</p><p><strong>Conclusion: </strong>This study examined caregiver dynamics in pediatric patients with sickle cell disease. Female, predominantly married caregivers, showed a long-term commitment. Despite these challenges, the caregivers demonstrated confidence in the importance of their roles. This study highlights the need for targeted support to improve caregivers' well-being.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31650"},"PeriodicalIF":2.4,"publicationDate":"2025-03-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143586483","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":"Global nLPHL One Working Group (GLOW) Research Roadmap for Nodular Lymphocyte-Predominant Hodgkin Lymphoma.","authors":"Monica Palese, Ranjana H Advani, Dominic Biney-Amissah, Suzi Birz, Graham P Collins, Richard T Hoppe, Kara M Kelly, Caroline Page-Kirby, Michael S Binkley, Jamie E Flerlage","doi":"10.1002/pbc.31646","DOIUrl":"https://doi.org/10.1002/pbc.31646","url":null,"abstract":"<p><p>Nodular lymphocyte-predominant Hodgkin lymphoma (NLPHL) is a rare, indolent lymphoma lacking an evidence-based standard of care. NLPHL research has been challenging due to its classification, unique features, and rarity. The Global nLPHL One Working Group (GLOW) launched in 2020 to accelerate NLPHL research internationally across all ages and stages and to establish a global standard of care. GLOW identified six core aims and 19 activities in its strategic roadmap to overcome historical research challenges, establish a research pipeline to inform a global standard of care, and disseminate findings. Once its prospective trials launch, GLOW will leverage this roadmap to study other rare lymphomas.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31646"},"PeriodicalIF":2.4,"publicationDate":"2025-03-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143586716","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":"Nephron-Sparing Surgery for Patients With Wilms Tumor, a Surgical Delphi Study Consensus Statement.","authors":"Matthijs Fitski, Guus M J Bökkerink, Andrew M Davidoff, Andrew Jackson Murphy, Abdelhafeez H Abdelhafeez, Lucas Krauel, Joerg Fuchs, Maximilian Pachl, S de Campos Vieira Abib, Israel Fernandez-Pineda, Sabine Sarnacki, Cees P van de Ven, Marc H W A Wijnen, A J Klijn, J Godziński, Alida F W van der Steeg","doi":"10.1002/pbc.31636","DOIUrl":"https://doi.org/10.1002/pbc.31636","url":null,"abstract":"<p><strong>Background and aim: </strong>Current pediatric renal tumor treatment protocols allow for nephron-sparing surgery (NSS) for unilateral disease only under strict conditions. Oncological guidelines do not account for surgical feasibility, however, possibly reducing the utilization of NSS. To potentially change this, a definition of surgical feasibility is required. This study aimed to define surgical consensus statements for the assessment of patients with Wilms tumor (WT) for NSS.</p><p><strong>Methods: </strong>A Delphi study was performed inviting 34 potential experts. Surgeons were asked to answer the questionnaires without considering their current treatment protocol. The first questionnaire contained five open-ended questions regarding surgery, oncology, contraindications for NSS, technique, and organization. Follow-up questionnaires contained closed-ended statements based on previous answers.</p><p><strong>Results: </strong>Eleven surgeons were included in the expert panel and continued with three follow-up questionnaires containing 72 statements in total. A median of seven (3 minimum to 10 maximum) NSS procedures were performed per year in the hospitals of the experts. NSS for nonsyndromic unilateral WT patients can be surgically considered if they receive 4 weeks of neoadjuvant chemotherapy, have a preoperative tumor volume of less than 200 mL, and if partial nephrectomy with wide resection margin (>5 mm) can be performed. In keeping with organizational guidelines, among COG surgeons, NSS was not advocated for patients with nonsyndromic unilateral WT.</p><p><strong>Conclusions: </strong>Using a Delphi method, surgical experts defined consensus statements regarding NSS for patients with WT to define surgical feasibility in future treatment protocols and expand the utilization of oncologically appropriate NSS.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31636"},"PeriodicalIF":2.4,"publicationDate":"2025-03-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143586499","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":"Associations of Ferritin and Folate Status With Clinical Outcomes in Childhood Cancer Patients: A Prospective Cohort Study.","authors":"Kalum Withey, Mark F H Brougham, Ilenia Paciarotti, Jane M McKenzie, David C Wilson, Raquel Revuelta Iniesta","doi":"10.1002/pbc.31645","DOIUrl":"https://doi.org/10.1002/pbc.31645","url":null,"abstract":"<p><strong>Background: </strong>Given the limited research on folate and ferritin status in children with cancer undergoing treatment, we investigated the prevalence of abnormalities and their impact on clinical outcomes and treatment complications.</p><p><strong>Methods: </strong>This prospective cohort study enrolled children <18 years diagnosed with cancer between August 2010 and February 2014. Data collection occurred at diagnosis, 3, 6, 9, 12 and 18 months. Clinical outcomes were classified as event-free survival or events (relapse, death, the development of new metastasis, becoming palliative) and treatment complications. Micronutrient status was assessed through clinical and nutritional analyses. Binary logistic regression, multilevel model analysis explored relationships between micronutrient status and clinical outcomes.</p><p><strong>Results: </strong>Eighty-two patients (median [interquartile range] 3.9 (1.9-8.8) years, 56% males) were recruited. Excess ferritin (85%) and folate deficiency (25.5%) were prevalent micronutrient abnormalities throughout the study. Decreased ferritin levels reduced the odds of events by 83.9% (odd ratios = 0.161, 95% CI = 1.000-1.002, p = 0.032). Higher ferritin was associated with increased number of treatment-related complications (B = 7.3E-5, 95% CI = 1.5E-5-0.000, p = 0.013). Folate status showed significant association with body mass index category (χ²= 9.564, p = 0.008), indicating that overweight and obese patients were more prone to deficiency, and methotrexate (F(2.9); p = 0.06; -2LL (1381)). Haematological malignancies (F(2.8); p = 0.05; -2LL (4244)) and medium and high treatment intensity (F(2.4); p = 0.09; -2LL 4262)) were associated with higher ferritin levels over 18 months.</p><p><strong>Conclusions: </strong>Paediatric cancer patients undergoing treatment exhibit high ferritin and reduced folate levels. Elevated ferritin is linked to increased toxicity and negative clinical outcomes, highlighting the importance of regular assessment and monitoring of both folate and ferritin. Implementing routine monitoring for these biomarkers could help mitigate adverse effects associated with treatment. Large-scale population-based studies and clinical trials are now warranted.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31645"},"PeriodicalIF":2.4,"publicationDate":"2025-03-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143586712","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":"Disparities in Clinical Trial Participation in Children and Adolescent Patients With a Hematologic Malignancy.","authors":"Caitlin Monroe, Joshua P Muñiz, Traci Leong, Rebecca Williamson Lewis, Sharon M Castellino","doi":"10.1002/pbc.31641","DOIUrl":"https://doi.org/10.1002/pbc.31641","url":null,"abstract":"<p><strong>Background: </strong>Low socioeconomic groups and racial/ethnic minorities continue to experience pediatric cancer outcome disparities, and remain underrepresented in clinical trials. It is vital to understand why underrepresentation exists and to address it in order to generalize trial findings to all groups. This study examined institutional disparities in clinical trial offerings and enrollment for children and adolescents with hematologic malignancies.</p><p><strong>Procedure: </strong>We conducted a single-institution retrospective analysis of clinical trial participation in patients less than 18 years old with newly diagnosed hematologic malignancies between 2011 and 2017. Patient demographics (e.g., parental primary language, race) were abstracted, and patient address at diagnosis was geocoded to characterize neighborhood socioeconomic status. Endpoints were frontline therapeutic clinical trial offering and enrollment. Multivariable logistic regression was constructed to examine predictors of trial enrollment.</p><p><strong>Results: </strong>Among 464 trial-eligible patients, 90.1% were offered clinical trial participation, of which 85% enrolled. There was no significant difference in enrollment by age, sex, parental primary language, neighborhood socioeconomic status, or rurality. However, non-Hispanic Black patients [OR: 0.4 (95% CI: 0.20-0.8), p = 0.01] and patients with lymphoma [OR: 0.15 (95% CI: 0.04-0.6), p = 0.01] were less likely to enroll on a clinical trial in our adjusted analysis.</p><p><strong>Conclusions: </strong>Despite a high institutional clinical trial enrollment rate for eligible patients, we found racial and disease-type disparities. Further work is needed to more granularly determine reasons for not offering trial participation or for not enrolling. By better-defining barriers to clinical trial enrollment, targeted institution-level interventions can be created to improve trial enrollment and reduce outcome disparities.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31641"},"PeriodicalIF":2.4,"publicationDate":"2025-03-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143586713","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 Novel Mutation in the Adenosine Deaminase-2 Gene Presenting as Severe Chronic Neutropenia With Myeloid Hypoplasia.","authors":"Pronamee Borah, Preethi Jeyaraman, Nitin Dayal, Prasan Deep Rath, Rahul Naithani","doi":"10.1002/pbc.31654","DOIUrl":"https://doi.org/10.1002/pbc.31654","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31654"},"PeriodicalIF":2.4,"publicationDate":"2025-03-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143586711","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":"Higher Rates of Hypersensitivity Reactions to Calaspargase Compared With Pegaspargase: A Single Center Retrospective Review","authors":"Kyra McCarty,&nbsp;Caroline Smith,&nbsp;Song Zhang,&nbsp;Ang Gao,&nbsp;Bianca Patel,&nbsp;Jessica Cox,&nbsp;Naomi Winick,&nbsp;Tamra Slone","doi":"10.1002/pbc.31640","DOIUrl":"10.1002/pbc.31640","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Asparaginase is a critical component of curative therapy for the treatment of pediatric acute lymphoblastic leukemia (ALL) and acute lymphoblastic lymphoma (LLy). Pegaspargase (PEG) increased the duration of asparagine depletion and lowered the incidence of hypersensitivity reactions (HSRs) compared with native asparaginase. When a second pegylated product, Calasparagase pegol-mknl (Cal-PEG) replaced PEG, we observed an increased incidence of HSR, defined as anaphylaxis or silent hypersensitivity. This led to a single center, retrospective review to determine the incidence of HSR in patients with ALL or LLy who received Cal-PEG or PEG.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Procedure</h3>\u0000 \u0000 <p>Electronic medical records of all patients who received at least two doses of Cal-PEG (December 2022–December 2023) or PEG (December 2019–December 2022) were reviewed.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Result</h3>\u0000 \u0000 <p>Overall, patients who received Cal-PEG had a significantly increased rate of asparaginase-related HSR compared with patients who received PEG. Twenty-one of 44 (48%) patients receiving Cal-PEG and 35 out of 154 (23%) of patients receiving PEG experienced Grade 3 or higher anaphylaxis, or silent hypersensitivity (<i>p</i> = 0.001). After 2:1 matching based on propensity scoring for confounding variables, patients who received Cal-PEG continued to have a statistically significantly higher rates of HSR, with HSR occurring in 21 out of 44 (48%) and 26 out of 88 (30%) patients receiving Cal-PEG and PEG, respectively (<i>p</i> = 0.04).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>The current administration of Cal-PEG in large cooperative group trials will allow for a postmarketing exploration of the true risk of HSR and associated patient-specific risk factors.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 5","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31640","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143573604","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Engaging Parents of Children With Sickle Cell Disease in Shared Decision-Making for Hydroxyurea: The ENGAGE-HU Study","authors":"Aimee K. Hildenbrand,&nbsp;Constance A. Mara,&nbsp;Bridget Murphy,&nbsp;Anna M. Hood,&nbsp;Yolanda Johnson,&nbsp;Lisa M. Shook,&nbsp;Francis J. Real,&nbsp;Cara Nwankwo,&nbsp;Rogelle Hackworth,&nbsp;Sherif M. Badawy,&nbsp;Alexis A. Thompson,&nbsp;Jean L. Raphael,&nbsp;Kim Smith-Whitley,&nbsp;Allison A. King,&nbsp;Cecelia Calhoun,&nbsp;Susan E. Creary,&nbsp;Steven K. Reader,&nbsp;Neha Bhasin,&nbsp;Amy E. Sobota,&nbsp;Patricia Houston,&nbsp;Cynthia Gipson,&nbsp;Michael R. DeBaun,&nbsp;Kay L. Saving,&nbsp;Marsha Treadwell,&nbsp;Charles T. Quinn,&nbsp;Lori E. Crosby","doi":"10.1002/pbc.31639","DOIUrl":"10.1002/pbc.31639","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Guidelines recommend that hydroxyurea be offered to children with sickle cell disease (SCD) as early as 9 months of age using shared decision-making. To help clinicians implement shared decision-making with parents, we developed the Hydroxyurea Shared Decision-Making (H-SDM) toolkit. We evaluated its effectiveness on parent decisional uncertainty, perceptions of shared decision-making, hydroxyurea knowledge, and the likelihood of being offered and prescribed hydroxyurea.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Procedure</h3>\u0000 \u0000 <p>Sites began in the usual care condition (clinician pocket guide) before crossing over to the H-SDM toolkit condition between 2018 and 2022. Caregivers of children with SCD (birth to 5 years) eligible for hydroxyurea completed assessments at baseline, immediately after discussing hydroxyurea with their clinician, and 3–7 months later.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Participants included 176 caregivers (93.2% female, 89% Black); most toolkit participants were enrolled during the pandemic (<i>n</i> = 81). There were no statistically significant differences between conditions on parent decisional uncertainty, perceptions of shared decision-making, or hydroxyurea knowledge (<i>p</i>-values &gt;0.05). However, there was a clinically important difference in certainty, with higher decisional uncertainty in the usual care group. A greater proportion of participants enrolled during usual care were offered (80.7%) and prescribed hydroxyurea (48.2%), compared to 58.7% offered and 39.7% prescribed during the toolkit condition (<i>p</i>-values ≤0.01).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>Findings suggest the toolkit may help parents feel more confident in deciding about hydroxyurea. Given the significant impacts of the COVID-19 pandemic on study implementation, the impact on hydroxyurea uptake requires additional exploration. Ultimately, the H-SDM toolkit may be most beneficial for clinics that do not routinely use a shared decision-making process for those considering hydroxyurea.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 5","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31639","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143573603","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}