Aaron B. Ross, Kriti Puri, Thomas L. Johnson, Lilliam D. Correa, Tami D. John, Brian Friend, Shoba A. Navai, Nabil Ahmed, Meenakshi G. Hegde, Bilal Omer, Andras Heczey, Baheyeldin M. Salem, Hossein Tcharmtchi, Rayne H. Rouce, David H. Steffin, Robert A Krance, Nino C. Rainusso, Saleh Bhar
{"title":"Cardiovascular Complications of Immune Effector Cell Therapies in Pediatric Hematological and Solid Tumors","authors":"Aaron B. Ross, Kriti Puri, Thomas L. Johnson, Lilliam D. Correa, Tami D. John, Brian Friend, Shoba A. Navai, Nabil Ahmed, Meenakshi G. Hegde, Bilal Omer, Andras Heczey, Baheyeldin M. Salem, Hossein Tcharmtchi, Rayne H. Rouce, David H. Steffin, Robert A Krance, Nino C. Rainusso, Saleh Bhar","doi":"10.1002/pbc.31557","DOIUrl":"10.1002/pbc.31557","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Immune effector cell (IEC) therapies, including chimeric antigen receptor (CAR)-modified T-cell therapy, have shown efficacy in pediatric B-cell acute lymphoblastic leukemia (B-ALL) and are being investigated for other malignancies. A common toxicity associated with IEC therapy is cytokine release syndrome (CRS), which can lead to cardiovascular decompensation due to systemic inflammation. Data are limited regarding cardiovascular adverse effects in children. This study aims to describe the cardiovascular adverse effect profile of IEC therapies in pediatric patients with hematologic and solid tumor malignancies.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>We retrospectively reviewed patients who received IECs directed towards various targets in patients with hematologic, solid, and brain tumor malignancies from January 2014 to June 2023 at Texas Children's Hospital. The primary end point was hypotension requiring vasoactive support and/or heart failure within 30 days of infusion.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>A total of 203 patients met inclusion criteria. Pretreatment echocardiogram was available for 142 (70%) pediatric patients, of whom 140 (96%) had normal baseline systolic function. Hypotension requiring vasoactive support occurred in 26 (13%) patients. Hematologic malignancy indications (<i>p</i> = 0.002), total body irradiation (TBI) (<i>p</i> = 0.002), and allogenic hematopoietic stem cell transplants (HCT) (<i>p</i> = 0.035) were associated with increased risk of hypotension requiring vasoactive support. Follow-up echocardiograms were available for 14 patients who met the primary end point, and all showed return to baseline within 6 months.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>Significant hemodynamic compromise occurred in a minority of patients treated with IEC therapies. All experiencing cardiac dysfunction had recovery of function, and there was no cardiovascular-related mortality.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 4","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-01-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143009577","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}
Pierpaolo Berti, Mauro Montanari, Massimo La Raja, Renato Giua, Irene Terrenato, Marco Becilli, Valeria Paganelli, Valentina Di Ruscio, Daniele Deriu, Eleonora D'Agostino, Ilaria Ferruzzi, Marta Conte, Ottavia Porzio, Michele Vacca
{"title":"Impact of Platelet Transfusion at Different Doses in Oncohematology Pediatric Inpatients and Outpatients: A Retrospective Study","authors":"Pierpaolo Berti, Mauro Montanari, Massimo La Raja, Renato Giua, Irene Terrenato, Marco Becilli, Valeria Paganelli, Valentina Di Ruscio, Daniele Deriu, Eleonora D'Agostino, Ilaria Ferruzzi, Marta Conte, Ottavia Porzio, Michele Vacca","doi":"10.1002/pbc.31550","DOIUrl":"10.1002/pbc.31550","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Platelet (PLT) transfusion is an essential strategy to prevent bleeding in children with thrombocytopenia associated to cancer treatment. However, data on optimal pediatric dosing and transfusion thresholds are limited.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>This retrospective study analyzed data from 607 pediatric patients with hematologic malignancies, nonmalignant disorders, and solid tumors who developed hypoproliferative thrombocytopenia during therapy. In the first phase (Objective 1), the effective response to transfusion (ERTR) was assessed following International Collaboration for Transfusion Medicine guidelines (ICTMG), comparing low-dose (1.1 × 10<sup>11</sup> PLT/m<sup>2</sup> body surface area) transfusions for inpatients and medium dose (2.2 × 10<sup>11</sup>) for outpatients. Transfusion thresholds were set at less than 10,000/µL versus 10,000–20,000/µL, and overall PLT concentrate consumption was analyzed. The second phase (Objective 2) examined the total number of transfusions per patient, incidence of major bleeding events, and bleeding-related mortality rates across dosing groups.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>ERTR ranged from 65% to 82%, with significantly higher rates in outpatients compared to inpatients. In outpatients with PLT less than 10,000/µL, the medium dose showed no significant advantage over the lower inpatient dose. Similar efficacy was observed between low (<10,000/µL) and high (10,000–20,000/µL) transfusion triggers, with no statistically significant difference in the incidence of major bleeding events across groups. The low-dose strategy was significantly associated with a reduction in PLT volume transfused compared to the standard expected dose.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>These findings support the application of ICTMG in pediatric settings. The lower PLT dose for inpatients is safe and effective, providing benefits in resource utilization, while a higher transfusion trigger (20,000/µL) does not improve outcomes.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 4","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-01-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31550","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143009616","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}
Arshia Madni, Deena R. Levine, Kimberly E. Sawyer, Yasmine Azzi, Belinda N. Mandrell, Yimei Li, Emily Ashcraft, Jacklyn Boggs, Rushil Acharya, Mary Caples, Jami S. Gattuso, Laurie A. Shoulders, Liza-Marie Johnson
{"title":"Impacts of Ethical Dilemmas and Moral Distress in Pediatric Hematology Oncology Nurses","authors":"Arshia Madni, Deena R. Levine, Kimberly E. Sawyer, Yasmine Azzi, Belinda N. Mandrell, Yimei Li, Emily Ashcraft, Jacklyn Boggs, Rushil Acharya, Mary Caples, Jami S. Gattuso, Laurie A. Shoulders, Liza-Marie Johnson","doi":"10.1002/pbc.31546","DOIUrl":"10.1002/pbc.31546","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Purpose</h3>\u0000 \u0000 <p>To assess the level of moral distress (MD) and perceptions of ethical climate among pediatric hematology/oncology (PHO) nurses and to identify bioethics topics where increased education was desired.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>In this cross-sectional study, we administered the 26-item Swedish Moral Distress Scale-Revised (sMDS-R), specifically revised and validated for pediatric oncology, in conjunction with the Clinical Ethics Needs Assessment Survey (CENAS). Electronic surveys were sent to inpatient and outpatient PHO nurses. Analysis included descriptive statistics, Spearman correlation, and the Mann–Whitney <i>U</i> test.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Of 123 nurse respondents, the overall mean MDS-R score was 2.75 (range: 0–16). Distressing encounters occurred infrequently (frequency mean 0.96, range: 0–4), resulting in relatively low distress (mean 2.24, range: 0–4). The scenarios resulting in the highest overall MD included performing painful procedures on children (2.46), lack of meaningful conversations due to time constraints (2.41), and poor team communication (2.68). Inpatient nurses reported higher levels of MD compared to outpatient nurses (<i>p</i> = 0.0002). The overall CENAS score was greater than 3 (range: 1–4), suggesting a positive ethical climate. There was a moderate negative relationship between sMDS-R combined scores and CENAS scores (<i>p</i> < 0.0001). Inpatient nurses reported a less positive ethical climate compared to outpatient nurses (<i>p</i> = 0.01). Nurses expressed a need for additional ethics education around withholding/withdrawing of life-sustaining treatments, providing end-of-life care, and working with challenging patients/families.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>Although the ethical climate was overall positive, several clinical situations appear to result in higher moral distress despite their infrequency, especially on inpatient units. Additional resources and education in navigating ethical dilemmas were requested by the nurses.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 4","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-01-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143009624","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}
Marc Bienias, Svenja Feldmann, Angela Wawer, Andreas Osterman, Stephan Böhm, Oliver T. Keppler, Julia Hauer
{"title":"Management of accidental live attenuated virus vaccination during treatment of a child with acute lymphoblastic leukemia","authors":"Marc Bienias, Svenja Feldmann, Angela Wawer, Andreas Osterman, Stephan Böhm, Oliver T. Keppler, Julia Hauer","doi":"10.1002/pbc.31398","DOIUrl":"10.1002/pbc.31398","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 3","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-01-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143009657","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}
Priya Mahajan, Sarah Beth May, Meghan Shekar, David Werny, Briana C. Patterson, Christine M. Chan, Jennifer H. Aldrink, Kyle Z. Horvath, Douglas S. Hawkins, Thomas A. Olson, Rajkumar Venkatramani
{"title":"Multicenter Retrospective Analysis of Pediatric Differentiated Thyroid Carcinoma: Treatment Practices and Outcomes Prior to Pediatric American Thyroid Association Guidelines Implementation","authors":"Priya Mahajan, Sarah Beth May, Meghan Shekar, David Werny, Briana C. Patterson, Christine M. Chan, Jennifer H. Aldrink, Kyle Z. Horvath, Douglas S. Hawkins, Thomas A. Olson, Rajkumar Venkatramani","doi":"10.1002/pbc.31545","DOIUrl":"10.1002/pbc.31545","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Differentiated thyroid carcinoma (DTC) is the most common pediatric thyroid malignancy, with papillary thyroid carcinoma (PTC) representing 90% of the cases. In 2015, the American Thyroid Association (ATA) developed management guidelines for pediatric DTC.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Procedure</h3>\u0000 \u0000 <p>Patients less than 21 years of age diagnosed with DTC between 2000 and 2015 at Texas Children's Hospital, Seattle Children's Hospital, Children's Healthcare of Atlanta, Children's Hospital Colorado, and Nationwide Children's Hospital were retrospectively analyzed to evaluate treatment practices before the implementation of the ATA guidelines.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>We included 216 pediatric patients with DTC. A majority were female (76%) with a median age at diagnosis of 14.8 years. Neck swelling (56%) and palpable nodules (49%) were common presenting symptoms. Diagnostic evaluations typically included thyroid ultrasound, with common features being microcalcifications (47.7%) and hypoechogenicity (39.0%). Fine-needle aspiration cytology confirmed diagnosis in 50.5%. The majority of patients underwent total thyroidectomy (78.7%) and lymph node dissection (75.5%). ATA risk stratification was low, intermediate, and high risk in 111 (51.4%), 29 (13.4%), and 76 patients (35.2%), respectively. Upfront radioactive iodine (RAI) was administered in 83%. At 1-year follow-up, 46% were disease-free (66% low risk, 59% intermediate risk, and 13% high risk). The 5-year progression-free survival for low, intermediate, and high risk were 86%, 53%, and 43%, respectively. The disease-related overall survival was 100%.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>Prior to the introduction of the ATA guidelines, a majority of patients underwent total thyroidectomy with lymph node dissection followed by RAI. Selective use of RAI for low-risk disease as recommended in the current guidelines may be appropriate.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 4","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-01-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143008011","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}
Cécile Olivia Muller, Fanny Delehaye, Christopher T. Gordon, Aurore Pire, Alaa Cheikhelard, Carmen Capito, Véronique Rousseau, Jeanne Amiel, Sabine Sarnacki
{"title":"Familial History in Ovarian Teratomas Is a Frequent Event: 22 Years’ Experience at a Single Center","authors":"Cécile Olivia Muller, Fanny Delehaye, Christopher T. Gordon, Aurore Pire, Alaa Cheikhelard, Carmen Capito, Véronique Rousseau, Jeanne Amiel, Sabine Sarnacki","doi":"10.1002/pbc.31548","DOIUrl":"10.1002/pbc.31548","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Ovarian mature teratoma represents the most common benign neoplasm among pediatric germ cell tumors. This study reports the prevalence and characteristics of familial forms identified in a single center over 22 years in order to better understand possible familial predispositions to ovarian teratoma.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>The records of all patients who were surgically treated for ovarian teratoma between 2000 and 2022 were retrospectively reviewed. Families were systematically asked about a possible family history of ovarian tumors (benign or malignant) or testicular germ cell tumors.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Among the 136 patients operated on for ovarian teratoma, 24 cases of familial form (18%) were identified. Patients with familial history of ovarian mature teratoma were more likely to have multifocal tumors at diagnosis and metachronous lesions over time (<i>p</i> = 0.032 and <i>p</i> = 0.018, respectively) compared with patients without familial history. The family history concerned first-degree relatives in 12/24 cases (50%) including one case of twin monozygotic sisters, and affected two consecutive generations in three cases.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>A systematic examination made it possible to find up to 18% of familial forms in mature teratomas of the ovary, which in these cases seem to be more likely multifocal. The underlying genetic mechanisms are likely to be heterogeneous. Further work seems to be necessary to provide a better understanding of the genesis of germ cell tumors.</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":"142983137","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}
Jenny Ruiz, Joanna M. Robles, Luisanna M. Sánchez, Alisa Khan, Amy D. Lu, Stacey Marjerrison, Andres Morales La Madrid, Paula Aristizabal
{"title":"Achieving Language Justice in Pediatric Hematology-Oncology: A Multinational Perspective for Language-Concordant Equitable Patient- and Family-Centered Care and Research Inclusion","authors":"Jenny Ruiz, Joanna M. Robles, Luisanna M. Sánchez, Alisa Khan, Amy D. Lu, Stacey Marjerrison, Andres Morales La Madrid, Paula Aristizabal","doi":"10.1002/pbc.31521","DOIUrl":"10.1002/pbc.31521","url":null,"abstract":"<p>Language-discordant healthcare encounters—when the patient/caregiver and clinician are not able to communicate directly in the patient's/caregiver's preferred language—are associated with worse quality of care, increased adverse events, and research exclusion. Here, we describe the current state of language justice in clinical practice and research in the United States, Canada, and Spain, discuss the role of social determinants of health and language in patient safety and health outcomes, and review an example of culturally and linguistically concordant interventions to increase research participation. We close with practical and global strategies to increase multilingual research participation and to provide equitable patient- and family-centered care in pediatric hematology-oncology.</p>","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":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31521","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142984557","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}
Timothy Rogers, Andreas Schmidt, Amanda F Buchanan, Thomas Scharschmidt, Roshni Dasgupta, Ekene A Onwuka, David A Rodeberg
{"title":"Rhabdomyosarcoma Surgical Update.","authors":"Timothy Rogers, Andreas Schmidt, Amanda F Buchanan, Thomas Scharschmidt, Roshni Dasgupta, Ekene A Onwuka, David A Rodeberg","doi":"10.1002/pbc.31496","DOIUrl":"https://doi.org/10.1002/pbc.31496","url":null,"abstract":"<p><p>Rhabdomyosarcoma (RMS) tumors arise from mesenchymal tissue and represent half of pediatric sarcomas, which in turn make up 7% of pediatric tumors. Advances in local control therapy of RMS have improved outcomes after surgical resection of the primary tumor, either before or after induction chemotherapy, even in the setting of metastatic disease. The utilization of diagnostic core needle and sentinel node biopsy techniques for lymph node staging are becoming more widely used. Over the past several years, refinement of prognostic factors with adoption of fusion status instead of histology, and optimized risk stratification schemas have been developed to assure appropriate therapy. There have been efforts between North American and European surgical oncology cooperative groups to standardize the care we provide, and yet there are still some philosophical differences to overcome.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31496"},"PeriodicalIF":2.4,"publicationDate":"2025-01-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142984138","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}