Pediatric Blood & Cancer最新文献

{"title":"Comment on: “Comment on: Bivalirudin During Thrombolysis With Catheter-Directed tPA in a Heparin-Refractory Patient: A Case Report”: The Promise of Dilute Thrombin Time and Drug-Calibrated Assays for Improved Bivalirudin Monitoring","authors":"Elissa R. Engel, Lori Luchtman-Jones","doi":"10.1002/pbc.31418","DOIUrl":"10.1002/pbc.31418","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-11-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142590938","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":"Impact of Neighborhood Disadvantage on Preventive and Acute Care Utilization in Sickle Cell Disease","authors":"Ashley Catanzarite,&nbsp;Jillian R. Bouck,&nbsp;Megan Matthes,&nbsp;Derrick L. Goubeaux,&nbsp;Allie Carter,&nbsp;Julia E. LaMotte,&nbsp;Seethal A. Jacob","doi":"10.1002/pbc.31422","DOIUrl":"10.1002/pbc.31422","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Individuals with sickle cell disease (SCD) experience significant healthcare disparities; however, there is little known on the impact of psychosocial stressors and neighborhood disadvantage on preventive and acute care utilization in this population.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Procedure</h3>\u0000 \u0000 <p>A retrospective data collection was performed for all patients cared for at a comprehensive pediatric sickle cell center in the Midwest who had also completed the Psychosocial Assessment Tool (PAT), a validated caregiver-reported measurement of family psychosocial risk, from September 2021 through December 2022. Patient age, payor, primary language, frequency of acute and missed preventive care visits, as well as Area Deprivation Index (ADI) and Childhood Opportunity Index (COI) scores were collected for 256 patients.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>The average state ADI was 6.3 (±2.9), and the majority of patients were in the Very Low or Low COI categories. Total PAT score (<i>p</i> = 0.003), state ADI (<i>p </i>&lt; 0.001), and state COI (<i>p</i> &lt; 0.001) were all significantly correlated with missed SCD clinic visits, with increased odds of a missed visit with increasing neighborhood disadvantage (OR 1.22, <i>p</i> &lt; 0.001). The odds of acute care were also increased in those with higher family psychosocial risk (OR 1.76, <i>p</i> &lt; 0.011), though this was not seen with ADI or COI, suggesting an additional effect of family stressors and resilience on acute care utilization.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>This highlights the importance of regular, universal social and psychological risk screening, as well as inclusion of psychosocial team members in SCD programs to identify and readily address risk factors that impact child outcomes in a minoritized chronic disease population.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-11-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31422","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142583731","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":"Aggressive Pediatric Primitive Round Cell Tumors with MN1::ZNF341 Fusion: A Mimic of Neuroblastoma","authors":"Serena Y. Tan,&nbsp;Mark G. Evans,&nbsp;Raya Saab,&nbsp;Ankur Gupta,&nbsp;Jack Reid,&nbsp;Tuan Dao,&nbsp;Elyssa Rubin,&nbsp;Anthony Crymes,&nbsp;Kapitolina Semenova,&nbsp;Hiroyuki Shimada,&nbsp;Chad Livasy,&nbsp;Ali Nael","doi":"10.1002/pbc.31425","DOIUrl":"10.1002/pbc.31425","url":null,"abstract":"<p>Neuroblastoma is one of the most common tumors in young children, arising from the adrenal medulla or paraspinal sympathetic ganglia. We describe primitive round cell tumors presenting in three patients less than 1.5 years old, with striking clinical and pathologic similarities to neuroblastoma. Unlike neuroblastoma, however, these primitive tumors did not show specific histologic or immunophenotypic evidence of neuroblastic differentiation, and harbored a <i>MN1::ZNF341</i> fusion. All patients progressed through neuroblastoma therapy and ultimately died of disease. These highly aggressive tumors mimicking neuroblastoma appear to be a novel and distinctive entity in need of further characterization.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-11-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31425","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142583609","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":"Neurocutaneous Melanocytosis-Associated Hydrocephalus: The Memorial Sloan Kettering Experience from 2001 to 2022","authors":"Scarlett Rodriguez,&nbsp;Francesca DeIeso-Frechette,&nbsp;Chidobe Umeasor,&nbsp;Christianah Alli,&nbsp;Caline Azzi,&nbsp;Sofia Haque,&nbsp;John A Pugh,&nbsp;Nathan Meeker,&nbsp;Yasmin Khakoo","doi":"10.1002/pbc.31434","DOIUrl":"10.1002/pbc.31434","url":null,"abstract":"<div>\u0000 \u0000 <p>We report two neurocutaneous melanocytosis (NCM) patients who required ventriculoperitoneal shunt placement and subsequently developed intraperitoneal melanoma. These patients with NCM are at an increased risk for developing <i>NRAS</i>-associated melanomas in the central nervous system, which in turn may lead to symptomatic hydrocephalus requiring cerebrospinal fluid diversion. Due to the rarity of NCM, current knowledge on disease progression and appropriate management is limited. Ongoing studies aiming to better understand this condition and inform its clinical management may help to identify risk factors for developing more severe complications.</p>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-11-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142583786","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":"Photobiomodulation Therapy in the Prevention of Chemotherapy Induced Oral Mucositis in Children with Acute Myeloid Leukemia: A Randomized, Double-blind, Clinical trial","authors":"Reem E. Elkady,&nbsp;Reham Khedr,&nbsp;Mohammed Hassan,&nbsp;Samy El Bayoumy,&nbsp;Sonia Mahmoud,&nbsp;Ebtisam Hanafy,&nbsp;Salam ElAraby,&nbsp;Alaa El Haddad","doi":"10.1002/pbc.31400","DOIUrl":"10.1002/pbc.31400","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Many preventive modalities have been advocated to reduce the incidence and severity of chemotherapy-induced Oral Mucositis (OM). Photobiomodulation, also known as Low-Level laser. Therapy, has shown promising results. This clinical trial was performed to evaluate the effectiveness of Photobiomodulation (PBM) for the prevention of chemotherapy-induced oral mucositis (OM) in children with Acute Myeloid Leukemia (AML).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Patients and methods</h3>\u0000 \u0000 <p>A double-blinded randomized clinical trial was performed, including forty-two children diagnosed with Acute Myeloid Leukemia (AML) admitted to receiving chemotherapy (CT) at Children's Cancer Hospital 57357, Cairo, Egypt. Patients were randomly assigned to either the control or test group. Both groups followed the standard preventive protocol for oral mucositis. In the control group I sham therapy, while in the Laser group I had Photobiomodulation sessions for five consecutive days once a day performed by Diode laser (Sirrolaser Blue ™, USA) 660nm, 15watt, 10 s. Laser sessions began on day 1 of chemotherapy and were repeated on days 2,3, 4, and 5. The response to both groups was evaluated according to the development of oral mucositis; graded using the classification criteria of the World Health Organization (WHO) and the National Cancer Institute scale (NCI – Common Terminology Criteria for Adverse Events, version 4.0). Incidence of oral mucositis was evaluated from baseline to 5, 12, 19, and 30 days after treatment.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>A clinical significant difference between the laser and control group was observed. The laser group showed a higher prevalence of no oral mucositis with decreased incidence of OM, while on day 12, the control group showed a higher prevalence of grades I and II (<i>P</i>-value &lt;0.001, effect size = 0.796). However, the laser group showed higher prevalence of mucosa free of OM; on day 19, the control group showed higher rates of grades II and III, (<i>P</i>-value &lt;0.001, effect size = 0.670). while on day 30 there was higher rates of grades I and II (<i>P</i>-value &lt;0.001, effect size = 0.576) with higher rates of healthy oral mucosa in the laser group.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Photobiomodulation effectively reduces the incidence and severity of chemotherapy-induced oral mucositis in pediatric patients diagnosed with AML.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-11-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142583794","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 and Laboratory Characteristics of Pediatric Patients With ACKR1/DARC-Associated Neutropenia","authors":"Lital Oz-Alcalay,&nbsp;Orna Steinberg-Shemer,&nbsp;Eyal Elron,&nbsp;Michal Dvori,&nbsp;Sarah Elitzur,&nbsp;Orly Dgany,&nbsp;Sharon Noy-Lotan,&nbsp;Tanya Krasnov,&nbsp;Hannah Tamary,&nbsp;Dafna Brik-Simon,&nbsp;Joanne Yacobovich,&nbsp;Oded Gilad","doi":"10.1002/pbc.31430","DOIUrl":"10.1002/pbc.31430","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>ACKR1/DARC-associated neutropenia (ADAN), resulting from homozygosity for a single nucleotide polymorphism (SNP) in the <i>ACKR1/DARC</i> gene (rs2814778), is a common cause of benign neutropenia that primarily affects individuals of African and Jewish Yemenite descent. We aimed to characterize ADAN in pediatric patients in Israel, given its ethnically diverse population.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Procedure</h3>\u0000 \u0000 <p>We assessed children with isolated neutropenia treated during 2018–2023 at one pediatric center, for the <i>ACKR1/DARC</i> polymorphism, using Sanger sequencing or targeted next-generation sequencing.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Of 115 patients evaluated, 49 (42.6%) were diagnosed with ADAN; of these, 29 (59%) had absolute neutrophil counts in the severe range (0–0.5 × 10⁹/L) at diagnosis. The allele distribution revealed 37% of Muslim Arab and 61% of Jewish origin. Yemenite, Ethiopian, Mediterranean, Asian, and European ancestry were included; 59% had a family history of neutropenia. The median age at the first neutropenia detection was 1.2 years; 91.8% were identified during routine blood counts. The median absolute neutrophil count at diagnosis was 0.5 × 10⁹/L (interquartile range: 0.3). An increased susceptibility to infections was not found either before or during the median follow-up period of 2.5 years (interquartile range: 1.54) after the diagnosis of ADAN. In 34 patients (72.3%), neutrophil counts were in the normal range during febrile illnesses.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>We identified ADAN in individuals of variable ethnicities, almost half with severe neutropenia. We recommend testing for ADAN in all children with isolated neutropenia without severe infections. Homozygosity for the <i>ACKR1/DARC</i> rs2814778 SNP may obviate the need for further investigation, follow-up, or treatment in specific clinical scenarios.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-11-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31430","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142590876","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":"Long-term renal outcomes of children with cancers treated with platinum-based chemotherapy: A retrospective chart analysis","authors":"Adrian Kan,&nbsp;Sarah Marokakis,&nbsp;James H. Ward,&nbsp;Siah Kim,&nbsp;Melissa Gabriel,&nbsp;Anne M. Durkan","doi":"10.1002/pbc.31404","DOIUrl":"10.1002/pbc.31404","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Purpose</h3>\u0000 \u0000 <p>Platinum-based chemotherapy is a mainstay of treatment for many childhood cancers but is associated with acute nephrotoxicity and long-term ototoxicity. There is emerging evidence of long-term renal complications. This study aimed to assess the prevalence of chronic kidney disease (CKD) in children treated with platinum chemotherapy (cisplatin and carboplatin) and identify potential risk factors for the development of CKD.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>We conducted a retrospective review of children diagnosed with hepatoblastoma, osteosarcoma, neuroblastoma, or medulloblastoma who received platinum chemotherapy over a 16 year timeframe. Patients were excluded if they did not have at least 3 years follow up data, died within 3 years of platinum chemotherapy, or if they relapsed. Clinical data were collected at baseline (first dose), 1 year, and at most recent follow up.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Of 328 treated patients, 147 met the inclusion criteria and were followed for a mean of 8.1 years (range 3–15.7 years). The median age at first dose was 3.7 years (IQR 1.7–9.6 years). CKD ≥grade 2 was present in 53(36%) at last follow up and 15(10%) had tubular dysfunction. A history of acute kidney injury at any time during treatment was associated with CKD (OR 3.12 CI 1.07–9.12, <i>p =</i>0.04). On multivariable analysis older age at platinum therapy (OR 1.2, CI 1.1–1.4, <i>p</i> = 0.004) and a high aminoglycoside or vancomycin trough level (OR 4.3, CI 1.9–9.7, <i>p</i> &lt; 0.001) were risk factors for CKD.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>The high rate of CKD in children treated with platinum chemotherapy warrants long-term follow-up and screening for progressive disease.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-11-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142583782","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 Relapse Versus Treatment Failure: The Case for Surveillance for Re-Appearance of Minimal Measurable Disease in Pediatric Patients with Higher Risk B-ALL","authors":"Paul S. Gaynon,&nbsp;Linwei Li","doi":"10.1002/pbc.31423","DOIUrl":"10.1002/pbc.31423","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-11-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142583623","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":"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":"https://doi.org/10.1002/pbc.31412","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31412"},"PeriodicalIF":2.4,"publicationDate":"2024-11-05","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}

{"title":"Sustained Remission of Relapsed Extranodal NK/T-Cell Lymphoma After Treatment With Camrelizumab Plus P-GEMOX and Autologous Stem Cell Transplantation: A Case Report","authors":"Kun Yang,&nbsp;Jian Xiao,&nbsp;Wenqiang Kong,&nbsp;Xiaodong Liu","doi":"10.1002/pbc.31424","DOIUrl":"10.1002/pbc.31424","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-11-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142568405","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}