Pediatric Blood & Cancer最新文献

{"title":"Neurocognitive, School-Based, and Quality of Life Outcomes for Youth With Sickle Cell Disease and Thalassemia: A Scoping Review.","authors":"Mary Ann Etling, Hakim Tokhi, Alexander A Boucher","doi":"10.1002/1545-5017.70776","DOIUrl":"https://doi.org/10.1002/1545-5017.70776","url":null,"abstract":"<p><p>The medical burdens of sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT) are chronic and cumulative, even in children. Yet the effects expand beyond medications and healthcare visits due to their impacts on school attendance, academic performance, and peer engagement. This scoping review explores the extent to which published research has explored the effects of SCD and TDT on educational success. The review synthesizes their effects on neurocognitive and academic performance alongside health literacy and any interventions that have been studied to mitigate their effects. In addition, the data offer insights into psychosocial impacts and health-related quality of life (HRQOL) for the two diseases, given that these factors can affect academic performance. For SCD in particular, neurocognitive deficits are evident compared to those without SCD, even without silent strokes, and their social and HRQOL are significantly impacted. Fewer data are available for TDT from an academic performance standpoint, but negative HRQOL effects are notable. This research is less commonly reported compared to traditional medical outcomes; however, because most medical outcomes are interwoven with social determinants of health, academic functioning cannot be ignored. After analyzing the data on school and HRQOL outcomes in SCD and TDT, the negative impacts on neurocognitive and psychosocial functions are significant, but data gaps demonstrate that more research toward evidence-based interventions must be undertaken.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70776"},"PeriodicalIF":2.4,"publicationDate":"2026-10-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149220528","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":"Multi-Omics Reveals a Cryptic RNF213::SLC26A11 Fusion and Targeted Therapy Dynamics in a Patient With Refractory Leukemia.","authors":"Jinlei Yin, Jia Fan, Wei Lin, Yuanyuan Zhang, Ying Wu, Peijing Qi, Mingying Zhang, Zhigang Li, Ruidong Zhang","doi":"10.1002/1545-5017.70777","DOIUrl":"https://doi.org/10.1002/1545-5017.70777","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70777"},"PeriodicalIF":2.4,"publicationDate":"2026-10-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149220555","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":"Cell-Sorted EBV PCR Identifies Lineage Shift and Guides Management After Hematopoietic Cell Transplantation for Pediatric EBV-Positive T/NK-Cell Lymphoid Neoplasia: A Case Series.","authors":"Ana M Quintero, Monica Bhatia, Diane George, Pawel Muranski, Prakash Satwani","doi":"10.1002/1545-5017.70770","DOIUrl":"https://doi.org/10.1002/1545-5017.70770","url":null,"abstract":"<p><p>Epstein-Barr virus (EBV)-positive T/natural killer (NK)-cell lymphoid neoplasms, including chronic active EBV disease and EBV-positive T/NK-cell leukemias and lymphomas, are rare, aggressive disorders for which allogeneic hematopoietic cell transplantation (alloHCT) is the only established curative therapy. After alloHCT, plasma or whole-blood EBV polymerase chain reaction (PCR) cannot distinguish relapse from EBV-driven donor B-cell proliferation. Cell-sorted EBV PCR resolves this ambiguity by quantifying EBV DNA across lymphocyte subsets to identify the cell lineage with the highest viral burden. We report four children in whom cell-sorted EBV PCR demonstrated a post-transplant shift in predominant EBV burden from T/NK cells to B cells, enabling de-escalation from cytotoxic therapy to immunosuppression reduction and B-cell-directed treatment.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70770"},"PeriodicalIF":2.4,"publicationDate":"2026-10-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149196004","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":"Successful Salvage Therapy With Quizartinib Plus Chemotherapy Followed by Quizartinib Monotherapy for Transplant Bridging and Post-Transplant Maintenance in a Child With Induction-Refractory FLT3-ITD-Positive AML.","authors":"Keiji Tasaka, Koji Kawaguchi, Taemi Ogura, Hiroyuki Kuroda, Ryo Akazawa, Takayuki Takachi, Shota Kato, Masahiro Sekiguchi, Motohiro Kato, Kenichiro Watanabe","doi":"10.1002/1545-5017.70768","DOIUrl":"https://doi.org/10.1002/1545-5017.70768","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70768"},"PeriodicalIF":2.4,"publicationDate":"2026-10-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149196012","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":"Conquering Infant and Young Children Medulloblastoma in Resource-Limited Settings: Challenges, Opportunities, and Treatment Outcomes.","authors":"Nisreen Amayiri, Eman Khattab, Mayada Abu Shanab, Maysa Al-Hussaini, Mouness Obeidat, Bayan Maraqa, Hasan Hashem, Ahmad Kh Ibrahimi, Rawad Rihani, Nasim Sarhan, Eric Bouffet","doi":"10.1002/1545-5017.70771","DOIUrl":"https://doi.org/10.1002/1545-5017.70771","url":null,"abstract":"<p><strong>Background: </strong>Infant and young children medulloblastoma poses significant treatment challenges due to high recurrence rates and the severe neurodevelopmental risks associated with craniospinal irradiation (CSI). Outcome data from low-middle-income countries (LMICs) remain scarce.</p><p><strong>Methods: </strong>A retrospective chart review was conducted for children (age <5 years) diagnosed with medulloblastoma at King Hussein Cancer Center, Jordan, between 2010 and 2023.</p><p><strong>Results: </strong>Twenty-seven patients were identified. The median age was 2 years (range, 0.5-4.3 years) with 78% males, and over half (52%) presented with metastatic disease. Twelve patients (44%) had ≤ 1.5 cm² residual tumor. Seventeen patients (63%) required ventriculoperitoneal shunt placement. Seven (26%) developed postoperative complications. Ten tumors were of classic histology, eight desmoplastic, six anaplastic, and three MBEN. Sixteen tumors (59%) were molecularly subgrouped: SHH (fourteen), Group-3 (two). Patients received various treatment protocols: HeadStart 1&2-like protocols (seventeen), SKK-92 (eight), and BBSFOP (two). Although 21 patients were planned for autologous stem cell transplantation (ASCT), only 13 received it (three received SKK-92 due to financial constraints, two prematurely stopped therapy, two developed early tumor progression, and one shifted to BBSFOP protocol due to chemotherapy toxicity). At a median follow-up of 7.4 years (range, 2.5-16 years), 17 patients (63%) are alive. Twelve patients (44%) are alive in CR1 without radiotherapy (five of whom also avoided ASCT), three in CR2 after CSI-36 Gy, one in CR3 after ASCT then CSI-23.4 Gy, and one patient lost to follow-up after salvage CSI-36 Gy. Two patients developed secondary tumors: one died from high-grade glioma and one developed thyroid carcinoma. Two survivors needed cataract surgery, and four had hearing loss. Ten survivors need hormonal supplements.</p><p><strong>Conclusions: </strong>Some infants and young children with medulloblastoma can achieve durable cure without ACST or radiotherapy, even in LMICs. While these therapies remain critical salvage options, lifelong comprehensive support is imperative to optimize survival and quality of life.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70771"},"PeriodicalIF":2.4,"publicationDate":"2026-10-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149196043","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":"Clinical Characteristics of Intracranial Hemorrhage in Pediatric Acute Leukemia.","authors":"Hajime Kikui, Suguru Uemura, Kana Tsuji, Sosuke Kakee, Natsumi Kikuchi, Aiko Kozaki, Atsuro Saito, Toshiaki Ishida, Takeshi Mori, Junji Koyama, Atsufumi Kawamura, Daiichiro Hasegawa","doi":"10.1002/1545-5017.70767","DOIUrl":"https://doi.org/10.1002/1545-5017.70767","url":null,"abstract":"<p><strong>Background: </strong>Intracranial hemorrhage (ICH) is a life-threatening complication of pediatric acute leukemia. However, whether early-onset ICH and late-onset ICH are distinct clinical entities remains unclear. This study investigated the characteristics, neuroimaging findings, and outcomes of ICH.</p><p><strong>Methods: </strong>Children with newly diagnosed acute leukemia were retrospectively reviewed. Clinical characteristics and OS were compared between patients with and without ICH, while neuroimaging findings and neurological outcomes were evaluated in patients with ICH. Early-onset ICH was defined as hemorrhage within 7 days after leukemia diagnosis, and late-onset ICH was defined as hemorrhage occurring thereafter.</p><p><strong>Results: </strong>Among 340 children with acute leukemia, 11 (3.2%) developed ICH. Patients with ICH were significantly younger and more frequently had KMT2A rearrangements, hyperleukocytosis, thrombocytopenia, and coagulation abnormalities than those without ICH. Patients with ICH had a significantly lower 5-year OS rate than those without ICH (55.4% vs. 90.4%, p < 0.001). Early-onset ICH predominantly occurred in patients with acute myeloid leukemia, and it was characterized by multifocal hemorrhages at presentation. Late-onset ICH occurred exclusively in patients with acute lymphoblastic leukemia (ALL). Three infants with KMT2A-rearranged B-cell ALL developed marked cerebral atrophy, followed by subdural hematoma. Five patients required neurosurgical intervention, and all survivors had persistent neurological sequelae.</p><p><strong>Conclusions: </strong>In pediatric acute leukemia, ICH comprises two clinically distinct patterns. Early-onset ICH is primarily associated with leukemia-related coagulopathy. Late-onset ICH is characterized by cerebral atrophy, followed by subdural hematoma, in infants with KMT2A-rearranged B-cell ALL. Recognizing these distinct patterns may lead to earlier diagnosis, neuroimaging evaluation, and timely neurosurgical intervention.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70767"},"PeriodicalIF":2.4,"publicationDate":"2026-10-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149196059","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":"Elevated Blood Lead Levels in Children With Sickle Cell Disease.","authors":"Bryce Demopoulos, Leah Seifu, Slavenka Sedlar, Jacqueline Ehrlich","doi":"10.1002/1545-5017.70679","DOIUrl":"https://doi.org/10.1002/1545-5017.70679","url":null,"abstract":"<p><strong>Background: </strong>Although prior reports suggest sickle cell disease (SCD) as a risk factor for lead poisoning, this association remains understudied. This paper assesses the relationship between SCD and elevated blood lead levels (BLLs) in New York City children.</p><p><strong>Methods: </strong>We conducted a retrospective matched cohort study of children born in NYC from 2004 to 2016 and tested for BLLs from 2004 to 2022. Children with SCD were matched 1:2 to children without SCD based on maternal race and country of birth. Firth penalized-likelihood logistic regression estimated associations between SCD and BLLs ≥3.5, ≥5, ≥10, and ≥15 µg/dL, adjusting for Medicaid status, housing age, and maternal race. Additional analyses evaluated effect modification by age and SCD genotype.</p><p><strong>Results: </strong>The cohort included 1463 children with SCD and 2982 without SCD. SCD was associated with lower odds of BLLs ≥3.5 µg/dL (odds ratio [OR] 0.81; 95% confidence interval [CI]: 0.66-0.99), no difference at ≥5 µg/dL (OR 0.86; 95% CI: 0.66-1.10), and higher odds at ≥10 µg/dL (OR 1.82; 95% CI: 1.06-3.13), with a similar estimate at ≥15 µg/dL (OR 2.23; 95% CI: 0.98-5.07). The SCD-by-age interaction was significant at ≥10 µg/dL (OR 4.47; 95% CI: 1.41-15.50), and children with HbSS had significantly higher odds of BLLs ≥10 µg/dL and BLLs ≥15 µg/dL than children without SCD (OR 2.25 and 2.98, respectively).</p><p><strong>Conclusion: </strong>Children with SCD have increased odds of elevated BLLs at higher thresholds, especially those aged ≥3 years and those with HbSS. Targeted rescreening of these children warrants consideration.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70679"},"PeriodicalIF":2.4,"publicationDate":"2026-10-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149196049","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":"Trametinib Plus Azacitidine as Bridging Therapy to Hematopoietic Stem Cell Transplantation in an Infant With PTPN11-Mutated Juvenile Myelomonocytic Leukemia.","authors":"Hisato Segoe, Jutaro Abe, Shinsuke Hirabayashi, Risa Nakashima, Kazumi Oura, Yukayo Terashita, Yuko Cho, Daichi Sajiki, Hideki Muramatsu, Atsushi Manabe","doi":"10.1002/1545-5017.70758","DOIUrl":"https://doi.org/10.1002/1545-5017.70758","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70758"},"PeriodicalIF":2.4,"publicationDate":"2026-10-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149183786","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":"Integrating Hereditary Hemorrhagic Telangiectasia into the Diagnostic Workup of Adolescent Heavy Menstrual Bleeding.","authors":"Maria Carter-Febres, Katie Wusik, Adrienne M Hammill","doi":"10.1002/1545-5017.70764","DOIUrl":"https://doi.org/10.1002/1545-5017.70764","url":null,"abstract":"<p><p>To understand the effect of hereditary hemorrhagic telangiectasis (HHT) on uterine bleeding is paramount to improving clinical care of menstruating individuals with HHT. Heavy menstrual bleeding (HMB) in adolescents and young adults (AYAs) with HHT is underrecognized and among individuals with HMB, HHT should be considered in the differential diagnosis, especially among those with suggestive clinical features or family history. Stigmatization associated with vaginal blood loss likely contributes to the paucity of evidence to guide HMB management in individuals with HHT, as previously reported in von Willebrand disease and other inherited bleeding disorders.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70764"},"PeriodicalIF":2.4,"publicationDate":"2026-10-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149183767","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":"CblC-Type Methylmalonic Acidemia Presenting as Macrocytic Anemia Mimicking Congenital Dyserythropoietic Anemia.","authors":"Xingqin Huang, Run Chen, Shuangnian Xu, Ting Li, Fang Long","doi":"10.1002/1545-5017.70729","DOIUrl":"https://doi.org/10.1002/1545-5017.70729","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70729"},"PeriodicalIF":2.4,"publicationDate":"2026-10-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149183776","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}