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Screening and Impact of Neuropathic Pain in Young Individuals With Sickle Cell Disease. 镰状细胞病年轻患者神经性疼痛的筛查及其影响
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-03-01 Epub Date: 2024-12-17 DOI: 10.1002/pbc.31493
Amira Adly, Maha Mohammed, Ahmed Aboata, Ghada Wassif, Marwa Nassef, Sara Makkeyah
{"title":"Screening and Impact of Neuropathic Pain in Young Individuals With Sickle Cell Disease.","authors":"Amira Adly, Maha Mohammed, Ahmed Aboata, Ghada Wassif, Marwa Nassef, Sara Makkeyah","doi":"10.1002/pbc.31493","DOIUrl":"10.1002/pbc.31493","url":null,"abstract":"<p><strong>Objective: </strong>To assess the frequency of neuropathic pain (NP) and its impact in young patients with sickle cell disease (SCD).</p><p><strong>Methods: </strong>We used the ID-Pain (ID-P) questionnaire and a bedside clinical sensory testing (CST) as screening tools for NP and performed sensory nerve conduction study (SNCS) for all the participants. The impact of pain was assessed using Patient-Reported Outcomes Measurement Information System (PROMIS) questionnaires and Pediatric Quality of Life Inventory (PedsQL) SCD module.</p><p><strong>Results: </strong>The mean age was 12.4 years. The ID-P questionnaire revealed that 20.5% of patients likely had NP, while 10 (34%) of the participants had abnormal CST, and only two patients (4.5%) had abnormal SNCS. On the PROMIS pain scores, the mean pain intensity score was 7.9 ± 2.0. Pain interference was the most affected domain in both patients and parent proxy reports (mean ± SD T-score 63.4 ± 6.6 and 65.2 ± 6.6, respectively) which was worse than other domains including pain quality-sensory and affective, and pain behavior (median T-scores of 47.8, 63.0, and 56.2, respectively). The mean global health-related quality of life (HRQoL) score of the children was 50.3 ± 12.1 indicating poor quality of life.</p><p><strong>Conclusion: </strong>We report the utility of simple tools like ID-P questionnaire and CST for screening of NP in young individuals with SCD.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31493"},"PeriodicalIF":2.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142847337","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}
引用次数: 0
Genomic and Clinicopathological Characterization of CRLF2-Rearranged Mixed Phenotype Acute Leukemia. crlf2重排混合表型急性白血病的基因组和临床病理特征。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-03-01 Epub Date: 2025-01-04 DOI: 10.1002/pbc.31507
Lina Han, Ruifang Zheng, Franklin Fuda, Miguel D Cantu, Prasad Koduru, Jesse M Jaso, Olga Weinberg, Sharon Germans, Mingyi Chen, Jing Xu, Weina Chen
{"title":"Genomic and Clinicopathological Characterization of CRLF2-Rearranged Mixed Phenotype Acute Leukemia.","authors":"Lina Han, Ruifang Zheng, Franklin Fuda, Miguel D Cantu, Prasad Koduru, Jesse M Jaso, Olga Weinberg, Sharon Germans, Mingyi Chen, Jing Xu, Weina Chen","doi":"10.1002/pbc.31507","DOIUrl":"10.1002/pbc.31507","url":null,"abstract":"<p><p>Rearrangements of cytokine receptor-like factor 2 gene (CRLF2) are present in ∼50% of B-lymphoblastic leukemia/lymphoma (B-ALL) with BCR::ABL1-like features. Herein, we report three patients with CRLF2-rearranged mixed phenotype acute leukemia (MPAL). All three cases were B/myeloid MPAL in young patients harboring P2RY8::CRLF2 or IGH::CRLF2 with additional genomic alterations in signaling (JAK and RAS) and cell cycle (CDKN2A/B) pathways, a genomic profile similar to that in BCR::ABL1-like B-ALL. Our study is the first case series to demonstrate clinicopathological and genomic features of this underrecognized entity. We recommend upfront genetic/molecular testing to timely diagnose and further characterize MPAL with BCR::ABL1-like features.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31507"},"PeriodicalIF":2.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142927666","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}
引用次数: 0
The Voxelotor Effect: Decreased Affinity for New Drugs for Sickle Cell Disease? 伏西洛效应：镰状细胞病新药亲和力降低？
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-03-01 Epub Date: 2024-12-10 DOI: 10.1002/pbc.31475
Charles T Quinn, Russell E Ware
{"title":"The Voxelotor Effect: Decreased Affinity for New Drugs for Sickle Cell Disease?","authors":"Charles T Quinn, Russell E Ware","doi":"10.1002/pbc.31475","DOIUrl":"10.1002/pbc.31475","url":null,"abstract":"<p><p>Voxelotor (OXBRYTA) was abruptly withdrawn from the global market in September 2024. Clinicians and patients were not prepared for this, and the sudden discontinuation has caused much consternation, uncertainty, and loss of trust.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31475"},"PeriodicalIF":2.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142807734","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}
引用次数: 0
Utility of Daratumumab as Bridging Therapy in De Novo T-Cell Acute Lymphoblastic Lymphoma.
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-03-01 Epub Date: 2025-01-06 DOI: 10.1002/pbc.31526
Jonathan J Holzmann, Rishi S Kotecha
{"title":"Utility of Daratumumab as Bridging Therapy in De Novo T-Cell Acute Lymphoblastic Lymphoma.","authors":"Jonathan J Holzmann, Rishi S Kotecha","doi":"10.1002/pbc.31526","DOIUrl":"https://doi.org/10.1002/pbc.31526","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 3","pages":"e31526"},"PeriodicalIF":2.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143029331","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}
引用次数: 0
Imlifidase Utilization in Glanzmann Thrombasthenia With Anti-GPIIb/IIIa and Anti-HLA Alloimmunization and Severe Platelet Refractoriness Following Hematopoietic Stem Cell Transplant. 伊立菲酶在伴有抗 GPIIb/IIIa 和抗 HLA 同种免疫以及造血干细胞移植后严重血小板折返的格兰茨曼血栓形成症中的应用。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-03-01 Epub Date: 2024-11-05 DOI: 10.1002/pbc.31412
Mohammad AlNajjar, Ryan Rochat, Amanda B Grimes, Amir Navaei, Todd Eagar, Caridad Martinez, Khalid Yassine, Robert Krance, Saleh Bhar
{"title":"Imlifidase Utilization in Glanzmann Thrombasthenia With Anti-GPIIb/IIIa and Anti-HLA Alloimmunization and Severe Platelet Refractoriness Following Hematopoietic Stem Cell Transplant.","authors":"Mohammad AlNajjar, Ryan Rochat, Amanda B Grimes, Amir Navaei, Todd Eagar, Caridad Martinez, Khalid Yassine, Robert Krance, Saleh Bhar","doi":"10.1002/pbc.31412","DOIUrl":"10.1002/pbc.31412","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31412"},"PeriodicalIF":2.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142583637","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}
引用次数: 0
Managing High-Risk PTPN11-Mutated Juvenile Myelomonocytic Leukemia in a Patient with SETBP1 Mosaicism. 处理一名 SETBP1 嵌合型患者的高风险 PTPN11 突变幼年骨髓单核细胞白血病。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-03-01 Epub Date: 2024-12-17 DOI: 10.1002/pbc.31491
Ursula Tanriver, Mario Geppert, Isabel Filges, Thomas Menter, Markus Schmugge, Tayfun Güngör, Carole Gengler, Brigitte Strahm, Miriam Erlacher, Tamara Diesch-Furlanetto, Nicolas von der Weid, Alexandra Schifferli
{"title":"Managing High-Risk PTPN11-Mutated Juvenile Myelomonocytic Leukemia in a Patient with SETBP1 Mosaicism.","authors":"Ursula Tanriver, Mario Geppert, Isabel Filges, Thomas Menter, Markus Schmugge, Tayfun Güngör, Carole Gengler, Brigitte Strahm, Miriam Erlacher, Tamara Diesch-Furlanetto, Nicolas von der Weid, Alexandra Schifferli","doi":"10.1002/pbc.31491","DOIUrl":"10.1002/pbc.31491","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31491"},"PeriodicalIF":2.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142847330","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}
引用次数: 0
Transient Severe Thrombocytopenia During Iron Repletion in an Adolescent Female. 青春期女性铁补充期间的短暂性严重血小板减少症。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-03-01 Epub Date: 2025-01-04 DOI: 10.1002/pbc.31509
Thomas Pincez, Mylène St-Onge Bégin, Rochelle Winikoff
{"title":"Transient Severe Thrombocytopenia During Iron Repletion in an Adolescent Female.","authors":"Thomas Pincez, Mylène St-Onge Bégin, Rochelle Winikoff","doi":"10.1002/pbc.31509","DOIUrl":"10.1002/pbc.31509","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31509"},"PeriodicalIF":2.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142927682","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}
引用次数: 0
Hemostatic Dynamics Mainly Based on Hypofibrinolysis During Early-Phase Interfant-Type Chemotherapy in Three Infants with Acute Lymphoblastic Leukemia: Results from Japan Children's Cancer Group ThrombALL-MLL-17. 3例急性淋巴细胞白血病患儿早期间型化疗期间主要基于低纤溶的止血动力学：来自日本儿童癌症组的结果
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-03-01 Epub Date: 2025-01-05 DOI: 10.1002/pbc.31524
Takashi Ishihara, Tomoko Onishi, Shinya Osone, Toshihiko Imamura, Naoya Matsumoto, Koji Suzuki, Yuki Arakawa, Takako Miyamura, Daisuke Tomizawa, Keiji Nogami
{"title":"Hemostatic Dynamics Mainly Based on Hypofibrinolysis During Early-Phase Interfant-Type Chemotherapy in Three Infants with Acute Lymphoblastic Leukemia: Results from Japan Children's Cancer Group ThrombALL-MLL-17.","authors":"Takashi Ishihara, Tomoko Onishi, Shinya Osone, Toshihiko Imamura, Naoya Matsumoto, Koji Suzuki, Yuki Arakawa, Takako Miyamura, Daisuke Tomizawa, Keiji Nogami","doi":"10.1002/pbc.31524","DOIUrl":"10.1002/pbc.31524","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31524"},"PeriodicalIF":2.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142932404","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}
引用次数: 0
A Decade of Achievements: International Society of Paediatric Oncology Baseline Nursing Standards. 十年成就：国际儿科肿瘤学会护理基线标准。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-03-01 Epub Date: 2024-12-15 DOI: 10.1002/pbc.31483
Linda Z Abramovitz, Courtney E Sullivan, Rehana Punjwani, Julia Challinor, Shenila Anwarali, Rachel Hollis, Lisa Morrissey, Glenn Mbah Afungchwi, Sara W Day
{"title":"A Decade of Achievements: International Society of Paediatric Oncology Baseline Nursing Standards.","authors":"Linda Z Abramovitz, Courtney E Sullivan, Rehana Punjwani, Julia Challinor, Shenila Anwarali, Rachel Hollis, Lisa Morrissey, Glenn Mbah Afungchwi, Sara W Day","doi":"10.1002/pbc.31483","DOIUrl":"10.1002/pbc.31483","url":null,"abstract":"<p><p>The six International Society of Paediatric Oncology (SIOP) Baseline Nursing Standards provide the first evidence-based framework for assessing and improving pediatric oncology nursing practice settings in low-, middle-, and high-income countries. Over the past 10 years, the standards have been translated to six languages, endorsed by 13 organizations, surveyed at 101 hospitals in 54 countries, and initiatives have been documented at over 63 institutions in 31 countries. Going forward, the SIOP Nursing Network's Baseline Standards Special Interest Group has established a 5-year strategic plan to guide advocacy, research, and standards implementation through global multidisciplinary collaboration.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31483"},"PeriodicalIF":2.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142829628","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}
引用次数: 0
Onset and Outcome of Ocular Lesions in Neonatal Severe Protein C Deficiency: Case Report and Literature Review. 新生儿严重蛋白C缺乏症眼部病变的发病和预后：病例报告和文献回顾。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-03-01 Epub Date: 2025-01-05 DOI: 10.1002/pbc.31518
Yuta Miyauchi, Naoki Egami, Hirosuke Inoue, Motoshi Sonoda, Katsuhide Eguchi, Masataka Ishimura, Masayuki Ochiai, Shoko Tsukamoto, Shinya Matsumoto, Takeshi Uchiumi, Koh-Hei Sonoda, Shouichi Ohga
{"title":"Onset and Outcome of Ocular Lesions in Neonatal Severe Protein C Deficiency: Case Report and Literature Review.","authors":"Yuta Miyauchi, Naoki Egami, Hirosuke Inoue, Motoshi Sonoda, Katsuhide Eguchi, Masataka Ishimura, Masayuki Ochiai, Shoko Tsukamoto, Shinya Matsumoto, Takeshi Uchiumi, Koh-Hei Sonoda, Shouichi Ohga","doi":"10.1002/pbc.31518","DOIUrl":"10.1002/pbc.31518","url":null,"abstract":"<p><p>Neonatal protein C (PC) deficiency, which presents with purpura fulminans or hemorrhagic infarction, has a poor prognosis. In a sporadic case of prenatal-onset purpura fulminans, preemptive intervention saved the patient but not her vision. Among 38 survivors from 47 cases with ocular lesions and biallelic PROC variants, 23 had total blindness and eight had unilateral blindness. Only one had intact vision. Prenatal-onset ocular lesions occurred in 11 cases (29% [corneal opacities, n = 7; bilateral persistent hyperplastic primary vitreous, n = 1; both, n = 3]). The eyes are the first organ affected by heritable severe PC deficiency. Prenatal intervention is needed to improve visual outcomes.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31518"},"PeriodicalIF":2.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142932406","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}
引用次数: 0
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