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Journal of Pediatric Hematology/Oncology最新文献

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Clinical and Epidemiological Characteristics of Infants <6 Months With Leukemia, Neuroblastoma, and Retinoblastoma in a Single Center in Iran. 伊朗单一中心6个月以下婴儿白血病、神经母细胞瘤和视网膜母细胞瘤的临床和流行病学特征
IF 0.9 4区 医学
Journal of Pediatric Hematology/Oncology Pub Date : 2026-10-01 Epub Date: 2026-08-28 DOI: 10.1097/MPH.0000000000003268
Mohammad Faranoush, Ibrahim Qaddoumi, Azim Mehrvar, Hadi Musakhani, Morteza Ashrafi, Narjes Mehrvar, Mithra Ghalibafian, Mahyar Nourian
{"title":"Clinical and Epidemiological Characteristics of Infants <6 Months With Leukemia, Neuroblastoma, and Retinoblastoma in a Single Center in Iran.","authors":"Mohammad Faranoush, Ibrahim Qaddoumi, Azim Mehrvar, Hadi Musakhani, Morteza Ashrafi, Narjes Mehrvar, Mithra Ghalibafian, Mahyar Nourian","doi":"10.1097/MPH.0000000000003268","DOIUrl":"10.1097/MPH.0000000000003268","url":null,"abstract":"<p><strong>Background: </strong>Malignancies in infants younger than 6 months differ from those in older children in incidence, clinical presentation, treatment response, and outcomes. Limited data exist on these infants' survival, particularly in low- and middle-income countries.</p><p><strong>Procedure: </strong>We retrospectively analyzed the data of 153 infants (<6 mo) with diagnosed retinoblastoma (n=116), neuroblastoma (n=20), or leukemia (n=17) at MAHAK Pediatric Cancer Treatment and Research Center (2007 to 2021). Demographics, disease characteristics, family history, treatment, and survival outcomes were collected. Overall survival (OS) was estimated by using the Kaplan-Meier method, and prognostic factors were evaluated using the Mantel-Cox log-rank testing.</p><p><strong>Results: </strong>Retinoblastoma was the most common diagnosis, with 67.8% of retinoblastoma cases being bilateral. Various stages and subtypes of neuroblastoma and leukemia were present. Family history of cancer was positive in 53.8% of patients. Most patients with neuroblastoma or retinoblastoma experienced remission; however, those with leukemia experienced high mortality (14/17). Diagnosis significantly predicted survival (RR for leukemia: 4, 95% CI: 0.43-4.19, P <0.001). Thus, the 5-year OS rate was 79.1% overall but was 14.7% for leukemia, 86% for retinoblastoma, and 100% for neuroblastoma.</p><p><strong>Conclusions: </strong>Infants younger than 6 months with leukemia, retinoblastoma, or neuroblastoma show distinct clinical patterns and have differing survival rates. Neuroblastoma and retinoblastoma have favorable outcomes, whereas leukemia carries a poor prognosis. Early recognition, tailored therapy, and follow-up are essential.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e339-e344"},"PeriodicalIF":0.9,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148840536","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Unmanipulated Haploidentical Stem Cell Transplantation Outcome in a Pediatric Patient With CD27 Deficiency and EBV-Induced Diffuse Large B-Cell Lymphoma With Primary CNS Involvement. 未经处理的单倍体干细胞移植在CD27缺乏和ebv诱导的弥漫性大b细胞淋巴瘤伴原发性中枢神经系统的儿童患者中的疗效
IF 0.9 4区 医学
Journal of Pediatric Hematology/Oncology Pub Date : 2026-10-01 Epub Date: 2026-06-23 DOI: 10.1097/MPH.0000000000003242
Mohammad Jahanpanah, Maryam Behfar, Saeed Yousefian, Hamid Farajifard, Leila Jafari, Amir Ali Hamidieh
{"title":"Unmanipulated Haploidentical Stem Cell Transplantation Outcome in a Pediatric Patient With CD27 Deficiency and EBV-Induced Diffuse Large B-Cell Lymphoma With Primary CNS Involvement.","authors":"Mohammad Jahanpanah, Maryam Behfar, Saeed Yousefian, Hamid Farajifard, Leila Jafari, Amir Ali Hamidieh","doi":"10.1097/MPH.0000000000003242","DOIUrl":"10.1097/MPH.0000000000003242","url":null,"abstract":"<p><strong>Background: </strong>CD27 deficiency is a rare primary immunodeficiency associated with severe Epstein-Barr virus (EBV)-related complications, including lymphoma.</p><p><strong>Observations: </strong>We report a pediatric patient with CD27 deficiency who developed EBV-associated diffuse large B-cell lymphoma with primary CNS involvement and early relapse. The patient underwent unmanipulated haploidentical stem cell transplantation using post-transplant cyclophosphamide. Engraftment was successful, graft-versus-host disease was controlled, and full donor chimerism was achieved.</p><p><strong>Conclusions: </strong>After 40 months, the patient remains free of GVHD, PID, and lymphoma relapse, demonstrating successful lymphoma control and correction of the underlying immunodeficiency, thereby highlighting the feasibility of haploidentical HSCT in these patients.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e289-e291"},"PeriodicalIF":0.9,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148308527","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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 Novel Pathogenic Variant in RTEL1 Causes Dyskeratosis Congenita/Hoyeraal-Hreidarsson Syndrome: A Report of Two Cases. 一种新的RTEL1致病变异导致先天性角化不良/Hoyeraal-Hreidarsson综合征：附两例报告
IF 0.9 4区 医学
Journal of Pediatric Hematology/Oncology Pub Date : 2026-09-30 DOI: 10.1097/MPH.0000000000003276
Michelle Nash, Alexandra Satty, Ariella Reiter, Michael Kho, Susan Schuval, Adrianna Vlachos, Jonathan D Fish
{"title":"A Novel Pathogenic Variant in RTEL1 Causes Dyskeratosis Congenita/Hoyeraal-Hreidarsson Syndrome: A Report of Two Cases.","authors":"Michelle Nash, Alexandra Satty, Ariella Reiter, Michael Kho, Susan Schuval, Adrianna Vlachos, Jonathan D Fish","doi":"10.1097/MPH.0000000000003276","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003276","url":null,"abstract":"<p><strong>Background: </strong>Telomere biology disorders (TBD) are a spectrum of conditions that result from shortened or dysfunctional telomeres. Dyskeratosis congenita (DC) is the prototypic TBD, classically presenting with leukoplakia, dysplastic nails, and reticular skin pigmentation. Immunologic abnormalities are common, and severe infection may be the presenting sign of DC. RTEL1 gene mutations are known to cause DC and are associated with a severe disease phenotype (Hoyeraal-Hreidarsson syndrome).</p><p><strong>Observation: </strong>In this report, we describe 2 patients with severe immunodeficiency and very early onset inflammatory bowel disease found to have novel homozygous mutations in RTEL1 (c.1670T>C, p.L557P).</p><p><strong>Conclusion: </strong>The clinical presentation of these 2 patients with the same novel mutation suggests this mutation is pathogenic and causative of TBD.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2026-09-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149110695","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
BK Virus-Associated Hemorrhagic Cystitis in Nontransplanted Children With Hematological Malignancies: A Single-Center Case Series. BK病毒相关出血性膀胱炎在未移植的儿童血液系统恶性肿瘤：单中心病例系列
IF 0.9 4区 医学
Journal of Pediatric Hematology/Oncology Pub Date : 2026-09-30 DOI: 10.1097/MPH.0000000000003288
Georgina Morón-Cazalilla, Marina Pons-Espinal, Anna Alonso-Saladrigues, Laura Arqués, Nazaret Sánchez-Sierra, Sílvia Simó-Nebot, Julià Gotzens, Ana Valls-Lafon, Cristina Esteva, Jose L Dapena, Susana Rives, Antoni Noguera-Julian
{"title":"BK Virus-Associated Hemorrhagic Cystitis in Nontransplanted Children With Hematological Malignancies: A Single-Center Case Series.","authors":"Georgina Morón-Cazalilla, Marina Pons-Espinal, Anna Alonso-Saladrigues, Laura Arqués, Nazaret Sánchez-Sierra, Sílvia Simó-Nebot, Julià Gotzens, Ana Valls-Lafon, Cristina Esteva, Jose L Dapena, Susana Rives, Antoni Noguera-Julian","doi":"10.1097/MPH.0000000000003288","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003288","url":null,"abstract":"<p><strong>Background: </strong>BK virus-associated hemorrhagic cystitis (BKV-HC) is well recognized after hematopoietic stem-cell transplantation but remains poorly described in nontransplanted children with hematological malignancies.</p><p><strong>Observations: </strong>We retrospectively identified 6 children with leukemia or lymphoma who developed BKV-HC over a 17-year period. Dysuria and macroscopic hematuria were the most common manifestations. One patient developed urinary obstruction and acute kidney injury. Median symptom duration was 16 days. Most patients received supportive care, and all but one recovered completely without sequelae.</p><p><strong>Conclusions: </strong>BKV-HC is an uncommon complication in nontransplanted children receiving chemotherapy for hematological malignancies. Clinical outcomes are generally favorable with conservative management.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2026-09-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149110790","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Clinical Experience With RIST-Based Multimodality Therapy in Relapsed Ewing Sarcoma: A Descriptive Single-center Case Series. 基于rist的多模式治疗复发尤文氏肉瘤的临床经验：描述性单中心病例系列。
IF 0.9 4区 医学
Journal of Pediatric Hematology/Oncology Pub Date : 2026-09-28 DOI: 10.1097/MPH.0000000000003284
Mahdi Asleh, Yotam Eshel, Miriam Ben Harosh, Aya Khalaila, Abed Abu Quider, Daniel Levin, Hagit Miskin
{"title":"Clinical Experience With RIST-Based Multimodality Therapy in Relapsed Ewing Sarcoma: A Descriptive Single-center Case Series.","authors":"Mahdi Asleh, Yotam Eshel, Miriam Ben Harosh, Aya Khalaila, Abed Abu Quider, Daniel Levin, Hagit Miskin","doi":"10.1097/MPH.0000000000003284","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003284","url":null,"abstract":"<p><strong>Background: </strong>Outcomes for relapsed Ewing sarcoma remain poor. The RIST regimen (rapamycin, irinotecan, dasatinib, temozolomide) provides a targeted biological alternative to standard salvage chemotherapy.</p><p><strong>Methods: </strong>We report 3 patients treated with RIST for relapsed Ewing sarcoma.Results:One patient with measurable disease achieved an early partial response before progressing at 9 months. Two patients received RIST as post-local-therapy consolidation with no evidence of disease, maintaining disease control for 41 and 44 months. Toxicities were manageable and primarily hematologic, without treatment discontinuations.</p><p><strong>Conclusion: </strong>Prolonged outpatient RIST administration is feasible, and its role as multimodality consolidation warrants further prospective evaluation.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2026-09-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149075141","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Hypofractionated Proton Beam Therapy for Localized Residual or Recurrent Unresectable Hepatoblastoma. 低分割质子束治疗局部残留或复发不可切除肝母细胞瘤。
IF 0.9 4区 医学
Journal of Pediatric Hematology/Oncology Pub Date : 2026-09-28 DOI: 10.1097/MPH.0000000000003291
Yu-Chen Chang, Tang-Her Jaing, Ting-Chi Yeh, Yao-Yu Wu, Wei-Heng Kao, Chen-Kan Tseng
{"title":"Hypofractionated Proton Beam Therapy for Localized Residual or Recurrent Unresectable Hepatoblastoma.","authors":"Yu-Chen Chang, Tang-Her Jaing, Ting-Chi Yeh, Yao-Yu Wu, Wei-Heng Kao, Chen-Kan Tseng","doi":"10.1097/MPH.0000000000003291","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003291","url":null,"abstract":"<p><strong>Background: </strong>We evaluated hypofractionated proton beam therapy (PBT) feasibility for unresectable hepatoblastoma (HBL).</p><p><strong>Observations: </strong>Six patients with residual/relapsed localized HBL received PBT (50 to 60 Gy (RBE) in 10 fractions) between April 2019 and September 2024, with follow-up through September 2025. After 55-month follow-up, 5 patients remained alive without disease. One marginal recurrence was successfully salvaged by re-irradiation, and another bridged to hepatectomy (10% viable tumor). Toxicities were minimal, with one grade 2 duodenitis. One patient died of treatment-unrelated myelodysplastic syndrome while remaining HBL-free.</p><p><strong>Conclusions: </strong>Hypofractionated PBT is a feasible, well-tolerated salvage option for refractory HBL, providing encouraging local control.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2026-09-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149111016","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Comment on: Assessment of Immunization Status Following Non-HSCT Chemotherapy-Treated Pediatric Leukemia Survivors-Toward Recovery-Guided Revaccination. 评论：非造血干细胞移植化疗后儿童白血病幸存者免疫状况的评估——以恢复为导向的再接种。
IF 0.9 4区 医学
Journal of Pediatric Hematology/Oncology Pub Date : 2026-09-28 DOI: 10.1097/MPH.0000000000003292
Wamiq Saeed, Zain U A Rao
{"title":"Comment on: Assessment of Immunization Status Following Non-HSCT Chemotherapy-Treated Pediatric Leukemia Survivors-Toward Recovery-Guided Revaccination.","authors":"Wamiq Saeed, Zain U A Rao","doi":"10.1097/MPH.0000000000003292","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003292","url":null,"abstract":"","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2026-09-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149075114","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Application of Dual-Energy X-Ray Absorptiometry for Bone Mineral Density Screening in Pediatric, Adolescent, and Young Adult Cancer Survivors: A Multi-Institutional Study of 6 California Children's Hospitals. 应用双能x线骨密度仪筛查儿童、青少年和青年癌症幸存者：加州6家儿童医院的多机构研究
IF 0.9 4区 医学
Journal of Pediatric Hematology/Oncology Pub Date : 2026-09-28 DOI: 10.1097/MPH.0000000000003271
Art Kulatti, Mrinal Joshirao, Lilibeth Torno, Carol Lin, Arun Panigrahi, Robert Raphael, Griselda Alvarez, Robert Goldsby, Jacqueline Casillas, Dennis Kuo
{"title":"Application of Dual-Energy X-Ray Absorptiometry for Bone Mineral Density Screening in Pediatric, Adolescent, and Young Adult Cancer Survivors: A Multi-Institutional Study of 6 California Children's Hospitals.","authors":"Art Kulatti, Mrinal Joshirao, Lilibeth Torno, Carol Lin, Arun Panigrahi, Robert Raphael, Griselda Alvarez, Robert Goldsby, Jacqueline Casillas, Dennis Kuo","doi":"10.1097/MPH.0000000000003271","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003271","url":null,"abstract":"<p><p>Deficits in bone mineral density (BMD) represent a major late effect on survivors of childhood cancer and can lead to increased fracture risks and morbidity that can persist in late adulthood. Low BMD is defined as >2 SDs below the mean of the age- and sex-specific reference population (Z-score ≤-2). Current guidelines recommend screening for low BMD with dual-energy x-ray absorptiometry (DXA) when at-risk patients enter long-term follow-up. We performed a retrospective chart review to study the application of BMD screening guidelines across 6 California pediatric cancer survivorship programs to assess variations in practice patterns. Our study showed inter-institutional variability in the management of pediatric, adolescent, and young adult cancer survivors with low bone mineral density between 2015 and 2019. We found differences in rates of referral to subspecialists such as endocrinology and orthopedics. There was no significant difference in the distribution of diagnoses of patients who had a DXA scan at each institution. The incidence of complications from low BMD was very rare in the studied population. Further studies on the risks and management of low BMD are necessary to inform evidence-based guidelines that can foster increased harmonization of practice patterns among survivorship groups.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2026-09-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149110754","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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 Pediatric European Society for Blood and Marrow Transplantation Criteria as a Sensitive Tool for Identifying Pediatric Sinusoidal Obstruction Syndrome Associated With Nonrelapse Mortality: A Retrospective Single-Center Study. 欧洲儿童血液和骨髓移植学会标准作为鉴别与非复发死亡率相关的儿童窦状窦阻塞综合征的敏感工具的应用：一项回顾性单中心研究。
IF 0.9 4区 医学
Journal of Pediatric Hematology/Oncology Pub Date : 2026-09-28 DOI: 10.1097/MPH.0000000000003278
Teruki Sugiyama, Shunsuke Nakagawa, Shogo Horikawa, Jun Nagahama, Tatsuro Nakamura, Takanari Abematsu, Takuro Nishikawa, Yasuhiro Okamoto
{"title":"Utility of Pediatric European Society for Blood and Marrow Transplantation Criteria as a Sensitive Tool for Identifying Pediatric Sinusoidal Obstruction Syndrome Associated With Nonrelapse Mortality: A Retrospective Single-Center Study.","authors":"Teruki Sugiyama, Shunsuke Nakagawa, Shogo Horikawa, Jun Nagahama, Tatsuro Nakamura, Takanari Abematsu, Takuro Nishikawa, Yasuhiro Okamoto","doi":"10.1097/MPH.0000000000003278","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003278","url":null,"abstract":"<p><p>Sinusoidal obstruction syndrome (SOS) is a potentially fatal complication of hematopoietic stem cell transplantation (HSCT). Although SOS has traditionally been diagnosed using the Baltimore or modified Seattle criteria (conventional criteria), the European Society for Blood and Marrow Transplantation recently proposed diagnostic criteria for children (pEBMT). This study assessed how conventional and pEBMT criteria affect SOS incidence, severity, and outcomes. We retrospectively analyzed pediatric HSCT procedures conducted from 2007 to 2023. Out of 186 HSCT procedures, 36 met at least 1 SOS criterion. Of these, 30 met the pEBMT criteria, 16 met the conventional criteria, and 10 met both. Patients meeting either criterion had a significantly worse prognosis than those without SOS. All SOS cases with nonrelapse mortality (NRM) fulfilled the pEBMT criteria, and the cumulative incidence of NRM was higher in the pEBMT group than in the conventional group. Furthermore, even in all HSCT cases, the pEBMT criteria were associated with NRM and served as a risk factor. The finding that pEBMT may serve as a sensitive tool for predicting NRM is new and provides a noteworthy aspect of this study. Following the pEBMT criteria closely and initiating early treatment for SOS may help reduce NRM in affected patients.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2026-09-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149075097","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Effects of Palliative Oral Etoposide on Survival and Treatment Burden in Patients With Relapsed/Refractory Pediatric Malignancies. 姑息性口服依托泊苷对复发/难治性儿童恶性肿瘤患者生存和治疗负担的影响。
IF 0.9 4区 医学
Journal of Pediatric Hematology/Oncology Pub Date : 2026-09-28 DOI: 10.1097/MPH.0000000000003285
Yannan J Wang, Alicia Lenzen, Jennifer Reichek, Amy L Walz, Soyang Kwon, David Walterhouse, Elizabeth Sokol
{"title":"Effects of Palliative Oral Etoposide on Survival and Treatment Burden in Patients With Relapsed/Refractory Pediatric Malignancies.","authors":"Yannan J Wang, Alicia Lenzen, Jennifer Reichek, Amy L Walz, Soyang Kwon, David Walterhouse, Elizabeth Sokol","doi":"10.1097/MPH.0000000000003285","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003285","url":null,"abstract":"<p><p>Oral etoposide has been given as palliative treatment for pediatric patients with relapsed/refractory malignancies. There is limited literature regarding the efficacy of oral etoposide for relapsed/refractory pediatric malignancies, and even less on its treatment burden for patients and families. We conducted a single-institution retrospective review evaluating patients with relapsed/refractory pediatric malignancies treated with single-agent oral etoposide over an 11-year period. Clinical efficacy and treatment burden measures were collected. Seventy six patients received at least one dose of oral etoposide. Median event-free survival was 47 days (95% CI: 25-68 d). Median overall survival was 113 days (95% CI: 73-149 d). Forty eight patients (63%) had imaging performed; 20 (41%) had stable disease (SD) lasting 11 to 1286 days (median 134 d) as their best response. There was a reduction in the number of clinic visits (P=0.02), hospitalizations (P=0.02), and hospital length of stay (P=0.01) compared with the most recent prior cancer treatment. Our experience suggests that palliative oral etoposide benefits pediatric patients with relapsed/refractory malignancies based on imaging documenting SD lasting a median of 3 months in a significant proportion of patients, and a reduction in treatment burden compared with their most recent prior cancer treatment.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":""},"PeriodicalIF":0.9,"publicationDate":"2026-09-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149075136","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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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