Pediatric Blood & Cancer最新文献

{"title":"Dental Adverse Effects Following Multimodality Treatment for Head and Neck Rhabdomyosarcoma: Results of a Trans-Atlantic Multicentre Study","authors":"Koen B. Krommenhoek,&nbsp;Marinka L. F. Hol,&nbsp;Olga Slater,&nbsp;Mark N. Gaze,&nbsp;Willem M. M. Fennis,&nbsp;Frederic J. Kolb,&nbsp;Ludwig Smeele,&nbsp;Daniel J. Indelicato,&nbsp;Reinier C. Hoogeveen,&nbsp;Alfred G. Becking,&nbsp;Johannes H. M. Merks","doi":"10.1002/pbc.32004","DOIUrl":"10.1002/pbc.32004","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>As survival of paediatric cancer improves, more emphasis is placed on late adverse effects. However, the dental and periodontal adverse effects of cancer treatments in children remain an underinvestigated complication. This study aims to investigate the prevalence of late clinical dental adverse effects (cDAE) in survivors of paediatric head and neck rhabdomyosarcoma (HNRMS).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Between 2017 and 2019, data were collected as part of a collaborative study between four international centres. During a ‘late adverse effect clinic’, dentists examined HNMRS survivors who were treated using multimodality treatments for dental and periodontal adverse effects using the following validated index scores ‘Simplified Oral Hygiene Index’ (SOHI), the ‘Decayed, Missing and Filled Teeth’ (DMFT) score and the ‘Dutch Periodontal Screening Index’ (DPSI).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Forty-six survivors, with a median follow-up of 9.9 years (range: 2–29.7 years), were included. Of these survivors of HNRMS, 89.1% had significant plaque accumulation. The median DMFT score was 3.00 (range: 0–26), which is considered normal. However, approximately a quarter of the survivors had exceptionally high DFMT scores, indicating many teeth were missing or filled. Affected gingivae, unhealthy gums, were seen in 67.4% of survivors of HNRMS.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Survivors of HNRMS have higher plaque and gingivitis rates, possibly due to cancer treatment or poor oral hygiene and diet. Next to regular care, additional monitoring of oral health and support of oral hygiene are indicated. Survivors, parents and physicians should be aware of the dental and periodontal risks and ensure proper care is available.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 11","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-09-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.32004","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144992879","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":"A Rare Case of Pediatric Translocation Renal Cell Carcinoma Following Medulloblastoma","authors":"Francesca Parisi,&nbsp;Valerio Gaetano Vellone,&nbsp;Gabriele Gaggero,&nbsp;Filippo Spreafico,&nbsp;Paola Collini,&nbsp;Virginia Livellara,&nbsp;Gianluca Piccolo,&nbsp;Marta Molteni,&nbsp;Elena Arkhangelskaya,&nbsp;Maria Beatrice Damasio,&nbsp;Stefano Avanzini,&nbsp;Federico Palo,&nbsp;Stefania Sorrentino","doi":"10.1002/pbc.32020","DOIUrl":"10.1002/pbc.32020","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 11","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-09-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144964289","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":"Romiplostim for Chemotherapy-Induced Thrombocytopenia: A Case Cohort Assessment of Safety, Efficacy, and Proposed Future Study in Pediatric Patients With Solid Tumors","authors":"Lev Gorfinkel,&nbsp;Emily M. Harris,&nbsp;Catherine B. Wall,&nbsp;Allison F. O'Neill","doi":"10.1002/pbc.32011","DOIUrl":"10.1002/pbc.32011","url":null,"abstract":"<div>\u0000 \u0000 <p>Romiplostim for chemotherapy-induced thrombocytopenia (CIT) is not well-studied in children. This single-center retrospective cohort study describes 24 pediatric patients with solid tumors who initiated romiplostim a median of 5 months into chemotherapy (range: 0–14 months) at a median maximum dose of 6.5 µg/kg (range: 1–10). A minority of patients achieved a decrease in cycle delays (30%) or dose reductions (13%). A comparative cohort, which did not receive romiplostim, had no difference in cycle delays (<i>p</i> = 0.42) or dose reductions (<i>p</i> &gt; 0.99). There were no adverse events. Romiplostim was safe in children with solid tumors, but requires further prospective study of efficacy.</p>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 11","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-09-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144964444","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":"Prevalence of Financial Hardship and Health-Related Social Needs in Pediatric Oncology: A Practice-Level Analysis of Systematic Screening.","authors":"Melissa P Beauchemin, Kathryn Henschel, David DeStephano, Mike Nicolaro, Rhea K Khurana, Kathryn McHenry, Dawn L Hershman, Justine M Kahn","doi":"10.1002/pbc.31988","DOIUrl":"10.1002/pbc.31988","url":null,"abstract":"<p><strong>Background: </strong>Screening for financial hardship and health-related social needs (HRSN) in pediatric oncology is recommended, yet practices vary widely, and standardized implementation remains limited. We implemented systematic screening in a pediatric oncology clinic in New York City.</p><p><strong>Procedure: </strong>We evaluated the implementation of financial hardship (two items) and HRSN (difficulty affording food, housing, transportation, eight items) screening in outpatient pediatric oncology clinic. Screening strategies included in-person screening by social work teams during inpatient or emergency department visits, and direct-to-patient screening via the electronic health record (EHR) patient portal for patients or caregivers who utilized the portal to check in for upcoming encounters. Data were extracted from the EHR of patients ≤18 years treated in the outpatient oncology clinic over a 2-year period. We analyzed response rates and calculated the prevalence of financial hardship and HRSN.</p><p><strong>Results: </strong>Among 621 pediatric oncology patients, 76% responded to screening, with Hispanic and non-Hispanic Black patients more likely to respond than non-Hispanic White patients. Among those who responded, 21% reported food insecurity, with significantly higher rates among Hispanic (35%) and non-Hispanic Black (24%) patients compared with non-Hispanic White (6%) and Asian (7%) patients. Financial hardship was reported by 76%, with higher rates among patients who were Hispanic (87%), non-Hispanic Black (87%), and Spanish-speaking (97%).</p><p><strong>Conclusions: </strong>Systematic screening for financial hardship and HRSN in pediatric oncology is feasible using a multipronged approach. Screening is crucial to early identification of at-risk families in order to implement timely interventions to prevent catastrophic financial situations and, potentially, to improve patient and household outcomes.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31988"},"PeriodicalIF":2.3,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144964378","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":"Targeted Therapy in Pediatric Langerhans Cell Histiocytosis: Describing a Novel Strategy to Minimize Long-Term Exposure While Maintaining Efficacy","authors":"Natalia Wojciechowska,&nbsp;Alexandra E. Richards,&nbsp;Aly Anthony,&nbsp;Maranda Diaz,&nbsp;Kelly Vallance,&nbsp;Chelsee Greer,&nbsp;Anish Ray","doi":"10.1002/pbc.32016","DOIUrl":"10.1002/pbc.32016","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Langerhans cell histiocytosis (LCH) is a rare malignancy driven by MAPK pathway activation and often involves <i>BRAF</i> V600E mutations. Targeted therapy with trametinib, a MEK inhibitor, is a promising alternative to conventional chemotherapy.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Procedure</h3>\u0000 \u0000 <p>We retrospectively analyzed the records of patients treated with trametinib either at relapse or as front-line therapy between 2020 and 2024.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Fifteen pediatric patients received trametinib either at diagnosis (<i>n</i> = 6), relapse (<i>n</i> = 7), or due to chemotherapy intolerance (<i>n</i> = 2). Molecular testing identified MAPK pathway mutations in 11 patients. The median age at treatment initiation was 5 years (range: 0.1–16.5). Notably, five of these patients started trametinib before the age of 1. The median treatment duration was 2.2 years (range: 0.1–4.7 years). All 15 patients achieved favorable responses without concerns regarding growth and development. The adverse effects included rash (40%) and diarrhea (13%), which were all mild and were managed with temporary dose adjustments. A self-weaning dosing strategy minimized long-term exposure while maintaining disease control.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Our data suggest that trametinib is a safe and effective therapy for pediatric LCH with broad efficacy and tolerability. However, prospective studies are needed to confirm these findings and refine targeted treatment protocols.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 11","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-08-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144964416","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":"Correction to “Feasibility Study of Micronutrient Status and Body Mass Index of Newly Diagnosed Pediatric Oncology Patients: Research Commentary”","authors":"","doi":"10.1002/pbc.31993","DOIUrl":"10.1002/pbc.31993","url":null,"abstract":"<p>Rogers PC, Cheng J, Lim A, Potts JE. Feasibility study of micronutrient status and body mass index of newly diagnosed pediatric oncology patients: Research commentary. <i>Pediatr Blood Cancer</i>. 2024;71(6):e30936. doi:10.1002/pbc.30936.</p><p>Description of error: Addition of co-author Lisi Dredze</p><p>The new author list is as follows:</p><p>Paul C. Rogers, Jeffrey Cheng, Alecia Lim, Lisi Dredze, James E. Potts.</p><p>We apologize for this late addition to the authorship.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 11","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-08-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31993","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144964410","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":"Examining Sociodemographic Differences in Response to Resilience Coaching for Adolescents and Young Adults Receiving Hematopoietic Cell Transplantation—A Post Hoc Analysis of the PRISM Intervention","authors":"Adam René P. Rosenbaum,&nbsp;Molly Ream,&nbsp;Kaitlyn M. Fladeboe,&nbsp;Nancy Lau,&nbsp;Joyce P. Yi-Frazier,&nbsp;Abby R. Rosenberg","doi":"10.1002/pbc.31998","DOIUrl":"10.1002/pbc.31998","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Adolescents and young adults (AYAs) receiving hematopoietic cell transplantation (HCT) for malignancy are at risk for poor psychological outcomes. Promoting Resilience in Stress Management (PRISM) is an early palliative care intervention that has previously been evaluated in a randomized trial among AYAs receiving HCT; results suggested the intervention's effect was limited to AYAs with baseline psychological symptoms.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Objective</h3>\u0000 \u0000 <p>We performed a post hoc analysis of these same data to explore differences in response based on sociodemographic factors.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>AYA participants were eligible for the trial if they were 12–24 years old, English-speaking, cognitively able, and within 4 weeks of HCT for malignancy or bone marrow failure. Enrolled AYAs reported their symptoms of anxiety, depression, hope, cancer-related quality of life, and resilience using standardized instruments at baseline and 6 months. We explored intervention effect sizes (Cohen's <i>d</i>) among groups stratified by gender, age, race, zip-code-based community distress (Distressed Community Index, with scores 3–5 indicating distressed communities), and baseline psychological symptoms.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Of 94 participants (50 PRISM, 44 Usual Care), 46% were female, 44% were 18–24 years old, 60% identified as non-Latino White, and 49% lived within distressed communities. Among AYAs 18–24 years old, those with baseline psychological distress tended toward gaining resilience, while those without baseline psychological symptoms seemed to gain hope. AYAs of all ages from distressed communities and with high baseline psychological burden seemed to gain both resilience and hope.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Discussion</h3>\u0000 \u0000 <p>AYAs with different ages, backgrounds, and psychological symptoms may have different responses to programs like PRISM.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Clinical Trial Registration Number</h3>\u0000 \u0000 <p>NCT03668223.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 11","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-08-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144964366","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":"Highlighting Healthcare Disparities in Sickle Cell Disease Management in Resource-Constrained Settings: A Cross-Sectional Study in Lebanon","authors":"Morgan Bou Zerdan,&nbsp;Adele Watfa,&nbsp;Bruna Kfoury,&nbsp;Jawad Hoballah,&nbsp;Rita El-Hachem,&nbsp;Lucia Ruggieri,&nbsp;Fedele Bonifazi,&nbsp;Miguel Raul Abboud,&nbsp;Soha Yazbeck","doi":"10.1002/pbc.31994","DOIUrl":"10.1002/pbc.31994","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Sickle cell disease (SCD) is a genetic disorder characterized by crescent-shaped red blood cells, which can result in a number of complications. SCD prevalence is higher in countries with limited resources and, in certain regions such as Lebanon, may be compounded by high consanguinity rates, posing significant challenges for both healthcare systems and patients. This study aimed to assess patient needs and the organization of SCD management centers, to identify service gaps, and enhance care.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>SCD patients, caregivers, or legal representatives completed questionnaires about their experiences from diagnosis to present. Data were safely preserved for analysis. Additionally, electronic surveys regarding services, equipment, and staffing were filled out by reference staff from healthcare facilities in Lebanon, a low-resource country with a large refugee population. Qualitative thematic analysis and SPSS were used to analyze data.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Among 124 participants, those living in rural areas reported significantly greater obstacles to quality care compared to urban residents (<i>p</i> &lt; 0.001). Facilities unequipped for SCD care reported significantly fewer trained staff (<i>p</i> = 0.001), fewer beds dedicated to SCD (<i>p</i> = 0.003), and reduced use of infection screening tools (<i>p</i> = 0.033) compared to equipped centers. Patients at unequipped centers also reported lower satisfaction with communication, pain control, and psychological support (all <i>p</i> &lt; 0.001), and showed less awareness of genetic counseling and SCD inheritance (<i>p</i> &lt; 0.001).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>This study reveals substantial deficiencies in SCD management in the surveyed setting. Addressing gaps in staffing, training, and resource availability is urgently required. Collaborative efforts to strengthen healthcare systems could greatly improve outcomes and ensure people with SCD obtain suitable, high-quality care.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 11","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-08-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144964370","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":"Expansion of the Phenotype of Lymphatic Anomalies Caused by Somatic Activating BRAF Variant","authors":"Michael D. Fox,&nbsp;Sumukh Kumar,&nbsp;Allison D. Britt,&nbsp;Abhay S. Srinivasan,&nbsp;Lea F. Surrey,&nbsp;Seth E. Vatsky,&nbsp;Alexandra J. Borst,&nbsp;Hakon Hakonarson,&nbsp;Dong Li,&nbsp;Sarah E. Sheppard,&nbsp;Kristen M. Snyder,&nbsp;Denise M. Adams","doi":"10.1002/pbc.32015","DOIUrl":"10.1002/pbc.32015","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>The somatic activating variant in <i>BRAF</i> (p.V600E) was recently described as a novel cause of macrocystic head and neck lymphatic malformations in three individuals. Other recent studies profiling the genetic causes of more complex lymphatic anomalies identified this same pathogenic <i>BRAF</i> variant. Our aim was to expand the phenotypic description of the somatic <i>BRAF</i> p.V600E variant in individuals with vascular anomalies.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Procedure</h3>\u0000 \u0000 <p>We searched the database of individuals with vascular anomalies at our institution for those identified as having complex lymphatic anomalies and somatic <i>BRAF</i> p.V600E variants. A comprehensive retrospective review of identified individuals’ electronic health records was conducted.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Six individuals with complex lymphatic anomalies had the <i>BRAF</i> p.V600E variant. All individuals had diffuse lymphatic malformations and abnormal lymphatic conduction. The conduction abnormalities were observed only after surgical interventions in five of the patients, in some cases, manifesting years later. There was immense phenotypic heterogeneity within the cohort.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>Complex lymphatic anomalies are an important new phenotype associated with the pathogenic BRAF p.V600E variant. Even in initially asymptomatic individuals within this patient population, longitudinal follow-up is necessary, and surgical intervention should be pursued with caution. Further investigation is needed to better understand the etiology of the conduction problems associated with this pathogenic variant to inform the multidisciplinary approach to treatment.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 11","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-08-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.32015","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144964408","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":"Efficacy and Toxicity of Inotuzumab Ozogamicin for Treatment of Relapsed/Refractory B-Cell Acute Lymphoblastic Leukemia in Pediatric and Young Adult Patients After CD19-Chimeric Antigen Receptor T-Cell Therapy","authors":"Paula Ogrodnik,&nbsp;Eric Anderson,&nbsp;Nicholas Gloude,&nbsp;Deborah Schiff","doi":"10.1002/pbc.32013","DOIUrl":"10.1002/pbc.32013","url":null,"abstract":"<p>We retrospectively analyzed outcomes for nine children and young adults who were treated with inotuzumab ozogamicin (InO) for relapsed/refractory (R/R) B-acute lymphoblastic leukemia (ALL) after CD19-chimeric antigen receptor T-cell therapy (CART). After InO cycle 1, overall response rate was 77.8%; 66.7% achieved measurable residual disease (MRD)-negative remission. One-year event-free survival (EFS) was 37.5%; 1-year overall survival (OS) was 50%; 2-year EFS was 37.5%; 2-year OS was 37.5%. Median survivor follow-up is 2.9 years (0.5–4.7). Significant adverse events included prolonged cytopenias, hepatotoxicity, and post-transplant sinusoidal obstructive sydrome. InO showed promising efficacy as treatment for children and young adults with R/R B-ALL after CD19-CART. Extramedullary disease prior to InO and positive MRD after InO cycle 1 were associated with poor outcomes.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 11","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.32013","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144964390","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}