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Behavioral and Electrophysiological Assessment of Central Auditory Processing in Individuals With Sickle Cell Disease 镰状细胞病患者中枢性听觉加工的行为和电生理评估。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-01-14 DOI: 10.1002/pbc.31539
Rhayane Vitória Lopes, Josefina Aparecida Pellegrini Braga, Andrea Angel, Daniela Gil
{"title":"Behavioral and Electrophysiological Assessment of Central Auditory Processing in Individuals With Sickle Cell Disease","authors":"Rhayane Vitória Lopes,&nbsp;Josefina Aparecida Pellegrini Braga,&nbsp;Andrea Angel,&nbsp;Daniela Gil","doi":"10.1002/pbc.31539","DOIUrl":"10.1002/pbc.31539","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Sickle cell anemia has a genetic origin characterized by an autosomal recessive inheritance pattern. The nervous system may be subject to vaso-occlusion and, consequently, affect the proper functioning of the central portion of hearing.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Objective</h3>\u0000 \u0000 <p>To assess central auditory skills and analyze short- and long-latency auditory evoked potentials in children with sickle cell disease.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Cross-sectional study. All children had normal hearing thresholds, and their central auditory processing underwent behavioral assessment with a battery of tests involving dichotic and monotic listening, binaural interaction, and temporal processing. The electrophysiological assessment used short- and long-latency auditory evoked potentials. Descriptive statistics were performed.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Of the 28 subjects evaluated (mean age of 9.46 years), 18 were females and 10 were males. Central auditory processing disorder was identified in 85.7% of the children. The auditory skills of figure-ground for verbal sounds, binaural interaction, and complex temporal ordering were the most affected. An increase in the absolute latencies of Waves III and V was observed in the short-latency potential.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Individuals with sickle cell disease have central auditory processing disorder, identified primarily by behavioral assessment.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 4","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-01-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142984559","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
Sensitivity and Specificity of a Signaling Question for Surveillance of Cognitive Functioning in Pediatric Sickle Cell Disease 儿童镰状细胞病认知功能监测信号问题的敏感性和特异性
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-01-12 DOI: 10.1002/pbc.31511
Steven J. Hardy, Megan E. Connolly, Sydney Forman, Robert S. Nickel
{"title":"Sensitivity and Specificity of a Signaling Question for Surveillance of Cognitive Functioning in Pediatric Sickle Cell Disease","authors":"Steven J. Hardy,&nbsp;Megan E. Connolly,&nbsp;Sydney Forman,&nbsp;Robert S. Nickel","doi":"10.1002/pbc.31511","DOIUrl":"10.1002/pbc.31511","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Sickle cell disease (SCD) confers neurological risks that contribute to cognitive and academic difficulties. Clinical guidelines state that cognition should be monitored using signaling questions. However, evidence is lacking regarding the extent to which signaling questions accurately identify children with cognitive issues.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Procedure</h3>\u0000 \u0000 <p>Caregivers of youth with SCD between ages 7 and 16 years (<i>n</i> = 89) responded to a signaling question about their child's academic performance: “How would you rate your child's academic performance relative to their peers?” Response choices included below grade level, on grade level, or above grade level. Youth with SCD completed the Wechsler Intelligence Scale of Intelligence, Fifth Edition to assess cognitive functioning.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Most children were described by caregivers as being on grade level (67%), followed by below grade level (21%), and above grade level (11%). Cognitive testing revealed that 36% of participants had a low IQ (≤85; i.e., ≥1 SD below the mean), 60% had an IQ within normal limits (86–114; i.e., &lt;1 SD below or above the mean), and 4% had a high IQ (≥115; i.e., ≥1 SD above the mean). Academic performance grouping was significantly associated with differences across cognitive domains (all <i>p </i>&lt; 0.001). Cognitive functioning varied by academic grouping for younger (ages 7–11; all <i>p </i>≤ 0.002) but not older children (ages 12–16; all <i>p </i>≥ 0.05). The signaling question's sensitivity for identifying those with cognitive difficulties was low (42%–54%); specificity, negative predictive value, and positive predictive value estimates were acceptable (87%–93%, 74%–84%, and 53%–79%, respectively).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>Asking caregivers of children with SCD to report on academic performance is a simple method of monitoring cognition. Children rated as below grade level should be referred for cognitive screening or evaluation. However, this question will likely result in a high rate of false negatives, and additional questions may be necessary to determine when to refer patients without obvious academic concerns.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 4","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-01-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142971838","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
Pediatric Brain Tumors in Western Kenya: Patient Outcomes and Healthcare Providers’ Perspectives 儿童脑肿瘤在肯尼亚西部:患者的结果和医疗保健提供者的观点。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-01-11 DOI: 10.1002/pbc.31544
Jesse Lemmen, Njie Albertine, Miyaada Abdi, Nilesh Mohan, Kibet Keitany, Marie Eliasson-Hofvander, Gertjan Kaspers, Festus Njuguna
{"title":"Pediatric Brain Tumors in Western Kenya: Patient Outcomes and Healthcare Providers’ Perspectives","authors":"Jesse Lemmen,&nbsp;Njie Albertine,&nbsp;Miyaada Abdi,&nbsp;Nilesh Mohan,&nbsp;Kibet Keitany,&nbsp;Marie Eliasson-Hofvander,&nbsp;Gertjan Kaspers,&nbsp;Festus Njuguna","doi":"10.1002/pbc.31544","DOIUrl":"10.1002/pbc.31544","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Pediatric brain tumors are understudied compared to other pediatric malignancies in low- and middle-income countries. Care delivery is inherently dependent on collaboration between multiple departments. This study aimed to present baseline data of pediatric neuro-oncology care in Western Kenya and illustrate barriers and facilitators of multidisciplinary care.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>We performed a mixed-methods study using medical records and interviews. Children below age 19 years, managed for a brain tumor at the neurosurgery or pediatric oncology departments between 2015 and 2022, were included. Various cadres (consultants, residents, medical officers, clinical officers, nurses, counselors) and teams (neurosurgery, pediatric oncology, radio-oncology, radiology, pathology) involved in pediatric brain tumor care participated.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Seventy-nine brain tumor patients were identified. The most prevalent confirmed diagnosis was medulloblastoma (<i>n</i> = 21). Most patients underwent surgery (<i>n</i> = 60; 76%). Event-free survival rate at 2 years was 13%. Abandonment was the most common (<i>n</i> = 36; 46%) treatment failure. Multidisciplinary consultation occurred more frequently between 2020 and 2022 than between 2015 and 2019 (OR 2.7 [95% CI: 1.0–6.9; <i>p</i> = 0.04]). Barriers and potential facilitators of multidisciplinary management were resources, diagnostic and therapeutic flow, standards, knowledge, information comprehension, and work relationships. Themes interacted at a governmental, facility, and community level.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>This baseline overview of pediatric neuro-oncology care in Western Kenya showed that survival of children with pediatric brain tumors was poor and treatment abandonment was common. Strengthening the capacity at different organizational levels will improve continuity of care and expand the knowledge to support holistic multidisciplinary care for children with brain tumors in Kenya.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 4","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-01-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31544","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142966148","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}
引用次数: 0
Major ABO Incompatibility in Non-Myeloablative Hematopoietic Stem Cell Transplant for Sickle Cell Disease—Not an Insurmountable Obstacle 非清髓性造血干细胞移植治疗镰状细胞病的ABO血型不合并非不可逾越的障碍。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-01-09 DOI: 10.1002/pbc.31515
Kimberly Ji Eun Chung, Donna A. Wall, Kuang-Yueh Chiang
{"title":"Major ABO Incompatibility in Non-Myeloablative Hematopoietic Stem Cell Transplant for Sickle Cell Disease—Not an Insurmountable Obstacle","authors":"Kimberly Ji Eun Chung,&nbsp;Donna A. Wall,&nbsp;Kuang-Yueh Chiang","doi":"10.1002/pbc.31515","DOIUrl":"10.1002/pbc.31515","url":null,"abstract":"<div>\u0000 \u0000 <p>With advances in conditioning strategies and graft-versus-host disease (GvHD) prevention, hematopoietic stem cell transplantation (HSCT) is a safe, curative treatment option for pediatric patients with sickle cell disease (SCD). However, donor options have been limited in non-myeloablative matched sibling donor (MSD) setting by excluding recipients with major ABO blood group incompatible donors due to concern of the risk of significant complications such as pure red cell aplasia (PRCA). We present three cases of successful HSCT with major ABO incompatibility with their donors, and discuss strategies to safely expand the donor pool to include these donors.</p>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 4","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-01-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142952845","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
Diagnosis of Leptomeningeal Disease in Diffuse Midline Gliomas by Detection of H3F3A K27M Mutation in Circulating Tumor DNA of Cerebrospinal Fluid 脑脊液循环肿瘤DNA H3F3A K27M突变检测对弥漫性中线胶质瘤轻脑膜病的诊断价值
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-01-09 DOI: 10.1002/pbc.31535
Satoshi Shibuma, Jotaro On, Manabu Natsumeda, Akihide Koyama, Haruhiko Takahashi, Jun Watanabe, Masaki Mitobe, Satoshi Nakata, Yuki Tanaka, Yoshihiro Tsukamoto, Masayasu Okada, Junichi Yoshimura, Mari Tada, Hiroshi Shimizu, Soichi Oya, Junko Murai, Kouichirou Okamoto, Hiroyuki Kawashima, Akiyoshi Kakita, Makoto Oishi
{"title":"Diagnosis of Leptomeningeal Disease in Diffuse Midline Gliomas by Detection of H3F3A K27M Mutation in Circulating Tumor DNA of Cerebrospinal Fluid","authors":"Satoshi Shibuma,&nbsp;Jotaro On,&nbsp;Manabu Natsumeda,&nbsp;Akihide Koyama,&nbsp;Haruhiko Takahashi,&nbsp;Jun Watanabe,&nbsp;Masaki Mitobe,&nbsp;Satoshi Nakata,&nbsp;Yuki Tanaka,&nbsp;Yoshihiro Tsukamoto,&nbsp;Masayasu Okada,&nbsp;Junichi Yoshimura,&nbsp;Mari Tada,&nbsp;Hiroshi Shimizu,&nbsp;Soichi Oya,&nbsp;Junko Murai,&nbsp;Kouichirou Okamoto,&nbsp;Hiroyuki Kawashima,&nbsp;Akiyoshi Kakita,&nbsp;Makoto Oishi","doi":"10.1002/pbc.31535","DOIUrl":"10.1002/pbc.31535","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Leptomeningeal disease (LMD) in diffuse midline gliomas (DMGs) can lead to devastating symptoms such as severe pain, urinary incontinence, and tetraparesis, with limited treatment options. We determined whether detecting <i>H3F3A</i> K27M-mutant droplets in cerebrospinal fluid (CSF) circulating tumor deoxyribonucleic acid (ctDNA) could be a biomarker for detecting LMD in DMGs.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Twenty-five CSF samples were obtained from 22 DMG patients. Histological confirmation of <i>H3F3A</i> K27M mutation was obtained in 10 (45.5%) cases. ctDNA was extracted from CSF, and <i>H3F3A</i> K27M-mutant and wildtype droplets were detected using digital droplet polymerase chain reaction (ddPCR). LMD was diagnosed by CSF cytology and pre- and post-contrast head and spine magnetic resonance (MR) imaging.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>The number of <i>H3F3A</i> K27M-mutant droplets (median 27 [range: 1–379] vs. median 0 [range: 0–1]; <i>p</i> &lt; 0.0001) and variant allele frequency (VAF) (median 48.9% [range: 7.5%–87.5%] vs. median 0.0% [range: 0.0%–50.0%]; <i>p</i> &lt; 0.0001) were significantly higher in the LMD/early-LMD group compared to no-LMD group. In two cases (Cases 4 and 11) without clinical evidence of LMD, multiple <i>H3F3A</i> K27M-mutant droplets were detected in CSF ctDNA. In those cases, extensive spinal dissemination was detected 6 months after the initial liquid biopsy. One case (Case 15) with high Schlafen11 (SLFN11) expression responded well to treatment for LMD and survived for 532 days after the diagnosis of LMD.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>This study provides evidence that detecting <i>H3F3A</i> K27M-mutant droplets in CSF ctDNA is diagnostic for LMD and is more sensitive than traditional methods such as CSF cytology and MR imaging.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 4","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-01-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142952843","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
Acute Liver Failure in a 17-Year-Old Boy Caused by a Blastic Plasmacytoid Dendritic Cell Neoplasm With a Novel MYB Rearrangement 伴MYB重排的17岁男孩母浆细胞样树突状细胞肿瘤引起的急性肝衰竭
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-01-08 DOI: 10.1002/pbc.31542
Atsushi Kohso, Hidemi Toyoda, Mari Morimoto, Kaori Niwa, Takahiro Ito, Ryo Hanaki, Tomonori Sakazaki, Yosuke Okumura, Takao Deguchi, Yuji Shimokariya, Satoru Muramatsu, Yoshifumi Hirokawa, Hideki Muramatsu, Masahiro Hirayama
{"title":"Acute Liver Failure in a 17-Year-Old Boy Caused by a Blastic Plasmacytoid Dendritic Cell Neoplasm With a Novel MYB Rearrangement","authors":"Atsushi Kohso,&nbsp;Hidemi Toyoda,&nbsp;Mari Morimoto,&nbsp;Kaori Niwa,&nbsp;Takahiro Ito,&nbsp;Ryo Hanaki,&nbsp;Tomonori Sakazaki,&nbsp;Yosuke Okumura,&nbsp;Takao Deguchi,&nbsp;Yuji Shimokariya,&nbsp;Satoru Muramatsu,&nbsp;Yoshifumi Hirokawa,&nbsp;Hideki Muramatsu,&nbsp;Masahiro Hirayama","doi":"10.1002/pbc.31542","DOIUrl":"10.1002/pbc.31542","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 3","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-01-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142952833","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
Prevention and Management of Infectious Complications in Pediatric Patients With Cancer: A Survey Assessment of Current Practices Across Children's Oncology Group Institutions 儿童癌症患者感染并发症的预防和管理:对儿童肿瘤组机构当前实践的调查评估。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-01-08 DOI: 10.1002/pbc.31532
Leonora R. Slatnick, David Hoogstra, Brian T. Fisher, Joshua Wolf, Etan Orgel, C. Nathan Nessle, Pratik A. Patel, Tamara P. Miller, Jennifer Wilkes, L. Lee Dupuis, Erin Goode, Kasey Jackson, Daniel N. Willis, Caitlin Elgarten, Catherine Aftandilian, Joel Thompson, Sarah Alexander, Melissa P. Beauchemin, Jennifer A. Belsky, Jennifer Hess, Zachary D. Prudowsky, Terri Guinipero, Jenna Rossoff, Jenna Demedis, Alexandra M. Walsh, Rebecca Richards, Daniel K. Choi, Christopher C. Dvorak, Adam J. Esbenshade
{"title":"Prevention and Management of Infectious Complications in Pediatric Patients With Cancer: A Survey Assessment of Current Practices Across Children's Oncology Group Institutions","authors":"Leonora R. Slatnick,&nbsp;David Hoogstra,&nbsp;Brian T. Fisher,&nbsp;Joshua Wolf,&nbsp;Etan Orgel,&nbsp;C. Nathan Nessle,&nbsp;Pratik A. Patel,&nbsp;Tamara P. Miller,&nbsp;Jennifer Wilkes,&nbsp;L. Lee Dupuis,&nbsp;Erin Goode,&nbsp;Kasey Jackson,&nbsp;Daniel N. Willis,&nbsp;Caitlin Elgarten,&nbsp;Catherine Aftandilian,&nbsp;Joel Thompson,&nbsp;Sarah Alexander,&nbsp;Melissa P. Beauchemin,&nbsp;Jennifer A. Belsky,&nbsp;Jennifer Hess,&nbsp;Zachary D. Prudowsky,&nbsp;Terri Guinipero,&nbsp;Jenna Rossoff,&nbsp;Jenna Demedis,&nbsp;Alexandra M. Walsh,&nbsp;Rebecca Richards,&nbsp;Daniel K. Choi,&nbsp;Christopher C. Dvorak,&nbsp;Adam J. Esbenshade","doi":"10.1002/pbc.31532","DOIUrl":"10.1002/pbc.31532","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>While clinical practice guidelines (CPGs) for pediatric oncology infection prophylaxis and management exist, few data describe actual management occurring at pediatric oncology centers.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>An electronic survey querying infection management practices in nontransplant pediatric oncology patients was iteratively created by the Children's Oncology Group (COG) Cancer Control and Supportive Care Infectious Diseases Subcommittee and sent to leaders at all COG institutions, limiting each site to one response to represent their institution.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>The response rate was 57% (129/227 institutions). Many sites reported utilizing COG-endorsed CPGs for antibacterial (76%) and antifungal prophylaxis (74%), and fever and neutropenia (FN, 64%). Most institutions reported using antimicrobial prophylaxis for patients with acute myeloid leukemia (88% antibacterial, 100% antifungal) and relapsed acute lymphoblastic leukemia (82% antibacterial, 95% antifungal). Definitions of fever, phagocyte recovery, and antibiotic duration in febrile patients varied. Most institutions administer empiric broad-spectrum antibiotics for nonneutropenic fever, although 14% reported withholding antibiotics based on initial clinical status or risk stratification tools. Most respondents reported (70%) admitting FN patients for at least 48 h, however 15% have low-risk FN protocols allowing outpatient management. FN patients remain admitted on antibiotics through count recovery in 50% of institutions, whereas the others employed various early discharge/early antibiotic discontinuation strategies.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>There is often consistency but also substantial variability in reported antimicrobial prophylaxis strategies and management of patients with fever and represents an opportunity for implementation studies to standardize application of CPG recommendations and randomized trials to advance evidence where knowledge gaps exist.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 3","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-01-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31532","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142952851","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}
引用次数: 0
Thrombopoietin Receptor Agonists for Thrombocytopenia in Pediatric Hematologic Malignancies 血小板生成素受体激动剂治疗儿童血液恶性肿瘤中的血小板减少症。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-01-08 DOI: 10.1002/pbc.31528
Amanda E. Marinoff, Allyson Thrall, Kathryn Aaronson, Benjamin S. Braun, Maria Castellanos, Julia Chu, Michelle Hermiston, Benjamin J. Huang, Anya Levinson, Erica Southworth, Beth Apsel Winger, Adam Olshen, Elliot Stieglitz
{"title":"Thrombopoietin Receptor Agonists for Thrombocytopenia in Pediatric Hematologic Malignancies","authors":"Amanda E. Marinoff,&nbsp;Allyson Thrall,&nbsp;Kathryn Aaronson,&nbsp;Benjamin S. Braun,&nbsp;Maria Castellanos,&nbsp;Julia Chu,&nbsp;Michelle Hermiston,&nbsp;Benjamin J. Huang,&nbsp;Anya Levinson,&nbsp;Erica Southworth,&nbsp;Beth Apsel Winger,&nbsp;Adam Olshen,&nbsp;Elliot Stieglitz","doi":"10.1002/pbc.31528","DOIUrl":"10.1002/pbc.31528","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Thrombopoietin receptor agonists (TPO-RAs) have demonstrated efficacy in treating clinically significant thrombocytopenia, including chemotherapy-induced thrombocytopenia in adults. However, data regarding their safety and efficacy in pediatric, adolescents, and young adult (AYA) patients with hematologic malignancies are limited.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>We retrospectively identified 15 pediatric and AYA patients aged 25 years or younger with hematologic malignancies treated with a TPO-RA at UCSF Benioff Children's Hospitals between 2015 and 2023. Platelet counts and transfusion requirements were compared before and after TPO-RA therapy.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>The median age at TPO-RA initiation was 16 years (range: 7–25 years). Nine patients (60%) had a history of bleeding or comorbidity that predisposed to severe bleeding risk. Eleven patients received romiplostim and four patients received eltrombopag. The median platelet count significantly increased from 24 × 10<sup>9</sup>/L at baseline to 54 × 10<sup>9</sup>/L after 3 weeks of any TPO-RA therapy (<i>p</i> = 0.029). Monthly platelet transfusion requirements significantly decreased from a median of 15 to two units after TPO-RA therapy (<i>p</i> = 0.007). Fourteen of the 15 patients (93%) achieved a sustained platelet count &gt;50,000/µL within 8 weeks, with a median time to response of 3 weeks. No TPO-RA-related adverse events were observed.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>TPO-RAs were effective in managing refractory thrombocytopenia in pediatric and young adult patients being treated for hematologic malignancies, with a favorable safety profile, even among patients with multiple comorbidities. These findings warrant further investigation through prospective clinical trials to confirm efficacy and establish clinical guidelines for this population.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 3","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-01-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142952857","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
Successful Treatment of Bone Marrow Necrosis and Fat Embolism Syndrome Without Long-Term Sequelae in a Young Adult with Sickle Cell Disease: A Case Report and Recommendations for Diagnosis and Management 成功治疗年轻成人镰状细胞病骨髓坏死和脂肪栓塞综合征无长期后遗症:一例报告及诊断和管理建议
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-01-08 DOI: 10.1002/pbc.31537
Chelsea L. Haynes, Ana Guimaraes, Shagun Vashisth, Evelisse Viamonte, Jennifer J. G. Welch, Bradley Denardo, Manpreet Kochhar, Lydia Musula, Diana O. Treaba, Sarah Welsh, Patrick T. McGann
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引用次数: 0
Pediatric Hematology–Oncology or Pediatric Hematology and Pediatric Oncology: A Need for Further Discussion 儿童血液学肿瘤学或儿童血液学和儿童肿瘤学:需要进一步讨论。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-01-08 DOI: 10.1002/pbc.31540
Daniel C. Moreira, Margit Mikkelsen, Jane S. Hankins, Tezer Kutluk
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引用次数: 0
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