Laura Jess, Gustaf Glavå, Simon Skau, Hans-Georg Kuhn, Maria Bäck, Marianne Jarfelt
{"title":"Exercise Capacity, Physical Activity, Fatigue, and Neurocognitive Function in Adult Survivors of Childhood Acute Lymphoblastic Leukemia.","authors":"Laura Jess, Gustaf Glavå, Simon Skau, Hans-Georg Kuhn, Maria Bäck, Marianne Jarfelt","doi":"10.1002/1545-5017.70736","DOIUrl":"https://doi.org/10.1002/1545-5017.70736","url":null,"abstract":"<p><strong>Background: </strong>Adult survivors of childhood acute lymphoblastic leukemia (ALL) are at risk of reduced exercise capacity, decreased physical activity levels, persistent fatigue, and neurocognitive deficits. The aim was to investigate exercise capacity and physical activity levels of adult survivors of childhood ALL and their associations with neurocognitive outcomes, including processing speed and functional frontal cortical brain connectivity.</p><p><strong>Methods: </strong>This cross-sectional study included 47 adult survivors of childhood ALL; 29 (61.7%) were male, with a median age of 29 years (min-max 21-47). Participants completed questionnaires, a graded exercise test on a cycle ergometer, neurocognitive tests, and frontal cortical brain imaging with functional near-infrared spectroscopy. Associations between predicted peak exercise capacity and fatigue (Fatigue Severity Scale [FSS]), processing speed (Processing Speed Composite), and functional frontal cortical network connectivity (global efficiency and modularity) were analyzed using linear regression. Parallel analyses were performed with physical activity level as the independent variable.</p><p><strong>Results: </strong>Participants achieved a mean peak exercise capacity corresponding to 83.8% (standard deviation [SD] = 13.1) of the age- and sex-predicted value (p < 0.001), and 42.2% did not meet WHO-recommended levels of physical activity. Statistically significant associations were identified between higher predicted peak exercise capacity and lower fatigue (FSS total score β = -0.33, 95% CI [confidence interval] -0.66 to -0.05, p = 0.02) and between higher physical activity levels and lower fatigue (FSS total score β = -0.4, 95% CI -1.8 to -0.2, p = 0.015). No other significant associations were found.</p><p><strong>Conclusion: </strong>Adult survivors of childhood ALL had reduced exercise capacity, and a substantial proportion did not meet recommended physical activity levels. Exercise capacity and physical activity levels were associated with fatigue. No significant associations were observed with functional frontal cortical network connectivity or cognitive measures.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70736"},"PeriodicalIF":2.4,"publicationDate":"2026-10-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149128097","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}
Joliza Patricia Cañeba, Shweta Bansal, Tea Reljic, Allyne Montecillo-Aguelo, Hamidah Alias, Farrah Amaranto, Maria Stella Aquino-Calingasan, Bambang Ardianto, Eggi Arguni, Sally Blair, Thi Tam Dang, Maria Beatriz Gepte, Samanmalee Gunasekara, Andini Handayani, Miriam Kimpo, Catherine G Lam, Nawachai Lertvivatpong, Sergio Licona, Ana Loseo, Ratha Mlis, Vivian Moreno Berrio, Nguyen Hoai Anh, Anna Lisa Ong-Lim, Ha Thu Pham, Krishna Sagar Sharma, Bun Sereyleak, Sally Jane G Velasco-Aro, Su Yadana, Ambuj Kumar, Sheena Mukkada
{"title":"Evidence-Based Clinical Practice Guidelines for the Management of Pediatric Febrile Neutropenia: Resource-Sensitive Recommendations for the Asian Continent.","authors":"Joliza Patricia Cañeba, Shweta Bansal, Tea Reljic, Allyne Montecillo-Aguelo, Hamidah Alias, Farrah Amaranto, Maria Stella Aquino-Calingasan, Bambang Ardianto, Eggi Arguni, Sally Blair, Thi Tam Dang, Maria Beatriz Gepte, Samanmalee Gunasekara, Andini Handayani, Miriam Kimpo, Catherine G Lam, Nawachai Lertvivatpong, Sergio Licona, Ana Loseo, Ratha Mlis, Vivian Moreno Berrio, Nguyen Hoai Anh, Anna Lisa Ong-Lim, Ha Thu Pham, Krishna Sagar Sharma, Bun Sereyleak, Sally Jane G Velasco-Aro, Su Yadana, Ambuj Kumar, Sheena Mukkada","doi":"10.1002/1545-5017.70743","DOIUrl":"10.1002/1545-5017.70743","url":null,"abstract":"<p><p>Febrile neutropenia (FN) is a frequent and serious complication in children receiving cancer therapy. Standardizing FN management across Asia is challenging due to variable healthcare infrastructure, diagnostic capacity, and infection patterns; however, it could improve resource use and patient outcomes. A multidisciplinary panel, including patient advocates from 10 countries developed a clinical practice guideline (CPG) for FN using the Grading of Recommendations, Assessment, Development, and Evaluation (GRADE) methodology. The final CPG contains 25 recommendations addressing regional priorities in diagnosis, risk stratification, and management. This CPG creates a regional standard to be tailored and studied in local context.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70743"},"PeriodicalIF":2.4,"publicationDate":"2026-10-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149128075","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}
Emily M Pariseau, Kimberly Buff, Michele A Scialla, Kimberly S Canter, Lori Wiener, Victoria Sardi-Brown, Julia B Tager, Kamyar Arasteh, Anne E Kazak
{"title":"Parents of Youth With Cancer Report on Their Psychosocial Care: iSTEPPP Survey Finds Alignment and Gaps With Standards.","authors":"Emily M Pariseau, Kimberly Buff, Michele A Scialla, Kimberly S Canter, Lori Wiener, Victoria Sardi-Brown, Julia B Tager, Kamyar Arasteh, Anne E Kazak","doi":"10.1002/1545-5017.70732","DOIUrl":"https://doi.org/10.1002/1545-5017.70732","url":null,"abstract":"<p><strong>Background: </strong>The Standards for Psychosocial Care for Children with Cancer and Their Families guide the provision of comprehensive psychosocial care. Parallel to research characterizing implementation of the Standards from the perspective of providers, this study examines implementation of the Standards from the perspective of parents of youth with cancer.</p><p><strong>Procedure: </strong>A national sample of parents of youth with cancer (N = 306), diverse in demographics and cancer characteristics, was recruited with Momcology (a patient-family support and advocacy organization). Parents completed an online survey about the psychosocial care their family received.</p><p><strong>Results: </strong>Fewer than half of parents agreed their psychosocial care was comprehensive and state of the art, and more than half endorsed unmet psychosocial needs. More consistently received Standards (endorsed by 55-87% of parents) include those closely connected with medical treatment (i.e., psychoeducation, adherence, coping with procedures, finances), patients' needs during treatment (i.e., psychosocial assessment and support, neurocognitive monitoring, guidance about survivorship), and end of life/bereavement. Less consistently received Standards (endorsed by only 32-49% of parents) include those related to school, social interaction, psychosocial screening in survivorship, palliative care, and family-centered psychosocial support for parents and siblings.</p><p><strong>Conclusions: </strong>The Standards least commonly received are those that do not fit \"neatly\" within the healthcare system, either because they include services for family members or collaborations with other systems. However, family-centered care and supporting patients beyond active treatment are crucial to adaptive adjustment to cancer. Strategies are needed to support consistent delivery of the Standards that are not yet well implemented.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70732"},"PeriodicalIF":2.4,"publicationDate":"2026-10-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149128134","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}
Kevin Shanchen Pu, Nicole Fernández-Viña, Briana C Martin-Villa, Daniela Ramirez, Rebecca M Lewinsohn, Mary Smith, Heidi Boynton, Lidia Schapira, Stephanie M Smith
{"title":"\"We Cannot Talk as a Family\": Complexities of Survivorship Communication Among Hispanic/Latino Young Adult Survivors of Childhood Cancer, Parents, and Clinicians.","authors":"Kevin Shanchen Pu, Nicole Fernández-Viña, Briana C Martin-Villa, Daniela Ramirez, Rebecca M Lewinsohn, Mary Smith, Heidi Boynton, Lidia Schapira, Stephanie M Smith","doi":"10.1002/1545-5017.70739","DOIUrl":"10.1002/1545-5017.70739","url":null,"abstract":"<p><strong>Background: </strong>Young adult childhood cancer survivors (YA-CCS) have an increased risk of treatment-related health complications throughout their lives. Lifelong survivorship care is recommended, yet many YA-CCS experience disruptions in care during pediatric-to-adult transitions and do not receive recommended survivorship care. Effective communication among YA-CCS, parents, and clinicians is essential for YA-CCS to understand their cancer history and survivorship care needs. However, families who use languages other than English, including Hispanic/Latino (H/L) families, often face barriers to effective communication and experience lower quality of care. With the long-term goal of improving communication and health outcomes, this study aimed to characterize communication related to survivorship care among H/L YA-CCS, their parents, and clinicians.</p><p><strong>Procedure: </strong>We conducted individual and small-group interviews with H/L YA-CCS (age 18-25, ≥5 years post-cancer diagnosis) and parents of YA-CCS to describe their experiences with posttreatment cancer-related communication, inside and outside the clinic. We analyzed qualitative data with applied thematic analysis.</p><p><strong>Results: </strong>Twenty YA-CCS and 18 parents participated at a median of 10 years post-diagnosis. Our results identified factors that influence cancer-related communication including family dynamics, language preferences, past experiences, and enduring emotional responses, which in turn shape YA-CCS' attitudes and beliefs about health risks.</p><p><strong>Conclusions: </strong>In this study of H/L YA-CCS and parents, we observed a diverse range of communication preferences among participants. To promote more effective survivorship care, clinical communication should be tailored based on each family's informational needs, familial communication dynamics, language preferences, and attitudes toward survivorship health risks.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70739"},"PeriodicalIF":2.4,"publicationDate":"2026-10-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149128059","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":"Decentralized CAR T-Cell Manufacturing in Southeast Asia: A Hub-and-Spoke Model for Therapeutic Equity.","authors":"Prasad Iyer","doi":"10.1002/1545-5017.70749","DOIUrl":"10.1002/1545-5017.70749","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70749"},"PeriodicalIF":2.4,"publicationDate":"2026-10-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149128113","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}
Karen Schow Jensen, Mette Tiedemann Skipper, Niels Holtum Birkebæk, Kjeld Schmiegelow, Thomas Leth Frandsen, Bente Langdahl, Rikke Beck Jensen, Asger Roer Pedersen, Christian Mølgaard, Liv Andrés-Jensen, Birgitte Klug Albertsen
{"title":"Bone Mineral Density After Contemporary Treatment for Acute Lymphoblastic Leukaemia: A Matched Cross-Sectional Study.","authors":"Karen Schow Jensen, Mette Tiedemann Skipper, Niels Holtum Birkebæk, Kjeld Schmiegelow, Thomas Leth Frandsen, Bente Langdahl, Rikke Beck Jensen, Asger Roer Pedersen, Christian Mølgaard, Liv Andrés-Jensen, Birgitte Klug Albertsen","doi":"10.1002/1545-5017.70727","DOIUrl":"https://doi.org/10.1002/1545-5017.70727","url":null,"abstract":"<p><strong>Background: </strong>Reduced bone mineral density (BMD) has been reported in survivors of childhood and young adult acute lymphoblastic leukaemia (ALL); however, data from cohorts treated with contemporary protocols and including matched controls remain limited.</p><p><strong>Procedure: </strong>In this population-based matched cross-sectional study, we used data from the Danish ALL-STAR cohort, comprising ALL survivors diagnosed between 2008 and 2019 at 1-45 years of age and treated according to the NOPHO ALL2008 protocol, and age- and sex-frequency matched controls. Participants were evaluated at least 1 year after completion of therapy and underwent dual-energy x-ray absorptiometry between 2019 and 2024. BMD was adjusted for sex, age and height and compared with that of matched controls.</p><p><strong>Results: </strong>The study included 298 ALL survivors and 346 matched controls. Median age at follow-up was 14.1 years, and the median time since diagnosis was 6.9 years. The greatest deficits in adjusted BMD were observed at hip sites, particularly among high-risk-treated survivors, with adjusted mean values of 91.2% (95% CI 88.2-94.4) at the femoral neck, 90.1% (95% CI 86.9-93.3) at the total hip, and 90.3% (95% CI 87.0-93.7) at the trochanter. Increasing time since diagnosis was associated with partial normalisation of BMD relative to controls in the standard- and intermediate-risk groups, a pattern not observed among high-risk-treated survivors.</p><p><strong>Conclusions: </strong>Persistent BMD deficits among survivors treated with high-risk regimens, without evidence of the partial normalisation observed in the standard- and intermediate-risk groups, underscore the need for long-term monitoring of skeletal health in this subgroup.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70727"},"PeriodicalIF":2.4,"publicationDate":"2026-10-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149113389","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}
Amber M Brown, Leah Stallkamp, April L Rahrig, Courtney M Rowan
{"title":"Albumin-Bilirubin Score Is Associated With Veno-Occlusive Disease-Related Mortality in Children Post-Hematopoietic Stem Cell Transplant.","authors":"Amber M Brown, Leah Stallkamp, April L Rahrig, Courtney M Rowan","doi":"10.1002/1545-5017.70731","DOIUrl":"https://doi.org/10.1002/1545-5017.70731","url":null,"abstract":"<p><strong>Background: </strong>Veno-occlusive disease (VOD) is a common complication post-hematopoietic stem cell transplant (HSCT) and is associated with increased mortality. Factors predictive of outcomes are lacking. Albumin-bilirubin (ALBI) score is used to quantify liver function and prognosticate but has not been investigated in VOD.</p><p><strong>Methods: </strong>Single-center retrospective study investigating the ALBI score in children aged 0-26 years who underwent HSCT from 2019 to 2024 and developed VOD. Primary outcome was hospital mortality. ALBI score was calculated at time points chosen a priori spanning preconditioning to post-VOD diagnosis.</p><p><strong>Results: </strong>Forty-three patients developed VOD. Most were transplanted for cancer (n = 33, 77%). Seven (16%) died. Preconditioning median ALBI scores did not differ between survivors and non-survivors. Median ALBI scores significantly increased from preconditioning -3.00 (IQR: -3.10, -2.80) to VOD diagnosis -1.70 (IQR: -2.00, -1.50), p < 0.001. In the week after VOD diagnosis, ALBI scores improved in survivors but worsened in non-survivors. ALBI scores 7 days post-VOD diagnosis were higher in non-survivors (p = 0.008, AUC = 0.83). Using an established cutoff, severe ALBI on day 7 post-VOD diagnosis had greater mortality odds (OR = 8.3 [95% CI: 1.4, 48.5], p = 0.02). Those who died trended toward higher ALBI 3 days prior to VOD diagnosis than those who survived, -1.70 (IQR: -1.90, -1.30) versus -2.10 (IQR: -2.40, -1.80), p = 0.16.</p><p><strong>Conclusions: </strong>In this cohort, ALBI score worsened with VOD diagnosis and at 7 days post-diagnosis was associated with mortality. The ALBI score is easily obtained and may aid in VOD prognostication.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70731"},"PeriodicalIF":2.4,"publicationDate":"2026-10-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149113331","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}
Liliana Vasquez, Claudia Sampor, Cindy Martinez, Patricia Gallardo, Julia Palma, Daniela Zavando, Reyna Copana, Hector Ojeda-Casares, Omaira Roldán, Luis Fernando Alarcón, Juan Arturo Sabinés, Essy Maradiegue, Claudia Pascual, Lisette Marmol, Nilda Villacrés, Carmen Guerrero, Gisela Vargas, Edgar Rivera, César García, Yaneth Clavo, Patricia Loggetto, Arantxa Cayon, Maria Migliora, Soad Fuentes-Alabí, Luis Beingolea, Paola Friedrich, Mauricio Maza, Piedad Huerta, Silvana Luciani, Maria Del Carmen Calle Dávila, Fernando Araos
{"title":"Implementing a South-South Cooperation Model to Strengthen Early Diagnosis of Childhood Cancer in the Andean Subregion.","authors":"Liliana Vasquez, Claudia Sampor, Cindy Martinez, Patricia Gallardo, Julia Palma, Daniela Zavando, Reyna Copana, Hector Ojeda-Casares, Omaira Roldán, Luis Fernando Alarcón, Juan Arturo Sabinés, Essy Maradiegue, Claudia Pascual, Lisette Marmol, Nilda Villacrés, Carmen Guerrero, Gisela Vargas, Edgar Rivera, César García, Yaneth Clavo, Patricia Loggetto, Arantxa Cayon, Maria Migliora, Soad Fuentes-Alabí, Luis Beingolea, Paola Friedrich, Mauricio Maza, Piedad Huerta, Silvana Luciani, Maria Del Carmen Calle Dávila, Fernando Araos","doi":"10.1002/1545-5017.70713","DOIUrl":"https://doi.org/10.1002/1545-5017.70713","url":null,"abstract":"<p><strong>Background: </strong>This project aims to promote early diagnosis of childhood cancer in the Andean subregion through South-South cooperation to strengthen health-system capacity. It was implemented within the WHO Global Initiative for Childhood Cancer (GICC) and the PAHO Cooperation Among Countries for Health Development (CCHD) mechanism, led by the Pan American Health Organization (PAHO), the Andean Health Organization (ORAS-CONHU), and St. Jude Children's Research Hospital.</p><p><strong>Methods: </strong>Between April 2023 and July 2024, seed funding supported South-South cooperation activities to strengthen early diagnosis through education, communication, and collaboration. A monitoring and sustainability phase was conducted from August 2024 to December 2025. The assessment focused on implementation activities and programmatic outputs, while available country-level indicators of diagnostic timeliness were reviewed descriptively.</p><p><strong>Results: </strong>The initiative supported implementation of a regional South-South cooperation model across six Andean countries. Outputs included training 61,502 participants through virtual activities and 4,114 through in-person activities, development and adaptation of communication materials, community awareness campaigns, strengthened national and regional coordination mechanisms, and incorporation of early diagnosis activities into national policy and cancer control frameworks. Country-level data on diagnostic timeliness were available only in selected settings and were not sufficiently standardized for regional assessment of impact.</p><p><strong>Conclusions: </strong>This initiative demonstrates the feasibility of South-South cooperation to support regional capacity building, health-system strengthening, and policy development for childhood cancer early diagnosis. The experience highlights the value of shared knowledge, resource optimization, regional coordination, and adaptation to local contexts. Longer-term standardized monitoring is needed to determine whether these implementation outputs translate into measurable improvements in early diagnosis and other patient-level outcomes.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70713"},"PeriodicalIF":2.4,"publicationDate":"2026-10-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149113352","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}
Olga M Sergeenko, Segev Gabay, Jonathan Roth, Amit Ringel, Rina Dvir, Shlomi Constantini
{"title":"Chemotherapy for Spinal Cord Intramedullary Pediatric Low-Grade Glioma-A Systematic Literature Review.","authors":"Olga M Sergeenko, Segev Gabay, Jonathan Roth, Amit Ringel, Rina Dvir, Shlomi Constantini","doi":"10.1002/1545-5017.70734","DOIUrl":"https://doi.org/10.1002/1545-5017.70734","url":null,"abstract":"<p><p>Pediatric intramedullary low-grade gliomas (PIMLGGs) are rare neoplasms that present unique clinical and management challenges. Although surgery remains the primary tool for tissue diagnosis and decompression, complete gross-total resection is frequently unfeasible due to the infiltrative nature of these lesions. To define the role of chemotherapy, we conducted a Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA)-based systematic review of 21 studies published between 2000 and 2025, comprising 98 patients who started chemotherapy at a median age of 3.9 years (ranging from 3 months to 17.7 years). In half of the cases, chemotherapy was used as primary therapy in cases of incomplete resection or biopsy, and half of the patients received salvage chemotherapy in cases of progressive tumor or recurrence. A platinum-based regimen was used in most of the patients. Complete response was achieved in 9% of cases, 9% had an incomplete response, and 42% had stable disease. Disease progression occurred in 26% of patients, and 15% died, most commonly due to disease progression. Median follow-up was 5 years (ranging from 3 months to 18 years).</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70734"},"PeriodicalIF":2.4,"publicationDate":"2026-10-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149113377","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}
Sarah Hsieh, Susmita Sarangi, Caileigh Pudela, Catherine M Broome, Tara K Suntum
{"title":"Classical Complement Pathway Inhibition in a Pediatric Patient With Refractory Immune Thrombocytopenia.","authors":"Sarah Hsieh, Susmita Sarangi, Caileigh Pudela, Catherine M Broome, Tara K Suntum","doi":"10.1002/1545-5017.70742","DOIUrl":"https://doi.org/10.1002/1545-5017.70742","url":null,"abstract":"<p><p>Pediatric refractory immune thrombocytopenia (rITP) is generally characterized by persistent thrombocytopenia and failure to achieve a sustained response to multiple standard therapies. We report a 4-year-old female with congenital tufting enteropathy, post-intestinal transplant patient on immunosuppressants, who developed severe rITP unresponsive to intravenous immunoglobulin, corticosteroids, rituximab, thrombopoietin receptor agonists, and vincristine. Complement inhibition with Berinert led to a rapid response, and sutimlimab led to sustained platelet recovery. This case highlights complement inhibition as a potential therapeutic option for rITP, representing the first reported pediatric case utilizing this approach for treatment-resistant pediatric ITP.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70742"},"PeriodicalIF":2.4,"publicationDate":"2026-10-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149113387","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}