Journal of Pediatric Hematology/Oncology最新文献

{"title":"Clonorchiasis, Marked Eosinophilia, and the Lack of Blasts in the Circulation Delaying the Diagnosis of Pediatric B-Lymphocytic Leukemia.","authors":"Ke Cao, Xiaojuan Luo, Defa Li, Xueyan Chen","doi":"10.1097/MPH.0000000000002989","DOIUrl":"10.1097/MPH.0000000000002989","url":null,"abstract":"<p><p>Eosinophilia is rare in pediatric acute lymphoblastic leukemia. In this report, we present a case of acute lymphoblastic leukemia with marked eosinophilia, whose diagnosis was delayed because of clonorchiasis.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e118-e120"},"PeriodicalIF":0.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142962295","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}

{"title":"Contributions of Pediatric Hematology/Oncology to the Diagnosis, Treatment, and Cure of Acute Lymphoblastic Leukemia-Part 2b (Numbers 16 to 20).","authors":"Denis R Miller","doi":"10.1097/MPH.0000000000002981","DOIUrl":"10.1097/MPH.0000000000002981","url":null,"abstract":"<p><p>This offering represents part 2b of a second set of 5 additional contributions of pediatric hematology/oncology to the diagnosis, treatment, and potential cure of precursor B-cell acute lymphoblastic leukemia. It contains numbers 16 to 20 and includes (16) allogeneic hematopoietic stem cell transplantation, newer immunotherapies including (17) blinatumomab, and (18) inotuzumab ozogamicin, (19) ploidy, and (20) creation of the \"day hospital\" to administer outpatient care to children with acute lymphoblastic leukemia and other cancers. These and the other reviewed contributions have had a significant role in improving the quality and duration of the lives of children, most of whom faced tragic and painful death back in the 1950s and 1960s. Most of our early optimistic goals were achieved and have benefitted substantially our patients, providing those of us who participated in many of these key clinical trials, a profound sense of accomplishment.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"53-58"},"PeriodicalIF":0.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142801186","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}

{"title":"Severe Unexplained Iron Deficiency Anemia in Children: High Yield of Upper Gastrointestinal Endoscopy Regardless of Gastrointestinal Symptoms: Erratum.","authors":"","doi":"10.1097/MPH.0000000000003007","DOIUrl":"https://doi.org/10.1097/MPH.0000000000003007","url":null,"abstract":"","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":"47 2","pages":"85"},"PeriodicalIF":0.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143492459","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}

{"title":"Clinical Analysis of Pediatric Acute Leukemias of Ambiguous Lineage: A Single Institution Retrospective Review.","authors":"Chen Xia, Chen Xiaoli, Zhu Xiaofan","doi":"10.1097/MPH.0000000000002986","DOIUrl":"10.1097/MPH.0000000000002986","url":null,"abstract":"<p><p>Acute leukemias of ambiguous lineage (ALAL) is a rare type of acute leukemia, referring to a group of disorders characterized by a combination of myeloid, lymphoid, or more lineages, whose incidence is significantly lower in children than adults. Here, we summarized the clinical features and outcomes of 36 pediatric ALAL patients in past 16 years. The patients diagnosed as ALAL based on the criteria of EGIL scoring system in 1998 (EGIL 1998) and/or the 2016 revisions to the WHO classification (WHO 2016) from January 1, 2005 to December 1, 2021 were included, respectively. During follow-up for a median 22 months, the median leukemia-free survival (LFS) was 18 months (0 to 172 mo) and the median overall survival (OS) was 22 months (1 to 173 mo), with a 5-year LFS rate of 67.3±9.2% and a 5-year OS rate of 66.0±10.7%. Patients who sustained negative minimal residual disease after 2 courses of standardized chemotherapy contributed to better 5-year OS (100% vs. 37.2±22.0%, P =0.028) and LFS (100% vs. 46.7±16.6%, P =0.028).</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"91-98"},"PeriodicalIF":0.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142882400","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}

{"title":"The Diagnostic Yield of Panel Versus Exome Sequencing to Identify Hereditary Cancer Disorders in Pediatric Cancer.","authors":"Shannon M Lozinsky, Carina A Iezzi, Dorota Gruber, Kenan Onel, Carolyn Fein Levy","doi":"10.1097/MPH.0000000000003000","DOIUrl":"10.1097/MPH.0000000000003000","url":null,"abstract":"<p><p>This study aimed to assess whether targeted exome sequencing (TES) outperforms next- generation sequencing (NGS) panels in detecting clinically actionable cancer predisposition syndromes (CPS) in pediatric cancer patients. Patients with cancer underwent genetic counseling and NGS panel testing (27 or 64 genes). Simultaneously, a 616-gene targeted exome, including the NGS panel genes and 552 additional potential cancer-related genes, was conducted on the patients and their parents. Of 42 patients undergoing both tests, NGS panels identified an APC risk allele (RA) in a patient with ganglioglioma and a pathogenic RB1 variant in a patient with retinoblastoma. In addition to the variants found by NGS panels, TES detected a pathogenic MUTYH variant in a patient with acute lymphoblastic leukemia (ALL) and a likely pathogenic (LP) BLM variant in another patient with ALL. TES also revealed a variant in candidate CPS genes, MC1R (RA) and EXT2 (LP), in a patient with embryonal rhabdomyosarcoma and Ewing sarcoma, respectively. Despite identifying variants in candidate CPS genes ( MC1R , EXT2 ) not included on common NGS panels and known CPS genes ( MUTYH , BLM ) absent from this study's panels, the diagnostic yield of clinically actionable CPS variants did not substantially increase with TES compared with standard NGS panels in pediatric cancer patients. In conclusion, for most cases, panel testing remains appropriate for CPS diagnosis in pediatric cancer within typical clinical settings.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"74-79"},"PeriodicalIF":0.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143123004","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}

{"title":"Implementation of a Low-risk Algorithm for Outpatient Management of Febrile Pediatric Patients With Sickle Cell Disease.","authors":"Jason Erno, Laurence Noisette, Shayla Bergmann, Charyse Diaz, Brittany Depriest, Paul J Nietert, Michelle Hudspeth","doi":"10.1097/MPH.0000000000002992","DOIUrl":"10.1097/MPH.0000000000002992","url":null,"abstract":"<p><strong>Background: </strong>Splenic dysfunction in children with sickle cell disease (SCD) increases the risk of serious bacterial infections; therefore, families are instructed to seek medical care in the presence of fever. Recurrent hospital admissions of patients with SCD cause financial and resource burdens on caregivers and the health care system, contributing to a lower quality of life in this patient population. Recent studies have documented a reduction of the incidence of bacterial infections among these patients managed on an outpatient basis with no association of increased morbidity and mortality. We decided to establish a partnership between our pediatric hematology/oncology division and pediatric emergency medicine division to initiate an algorithm to identify low-risk patients eligible for outpatient management.</p><p><strong>Procedure: </strong>We conducted a retrospective review of patients with SCD younger than 18 years of age, followed at the Comprehensive Care Sickle Cell Center at the Medical University of South Carolina (MUSC), who presented to our Pediatric Emergency Department (ED) with a temperature ≥101°F from July 1, 2018 to June 30, 2020.</p><p><strong>Results: </strong>The mean length of stay and age at admission were nearly equal between pre-implementation and post-implementation of the algorithm. The admission rates from the study for were 55.2% and 43.6% pre-implementation and post-implementation, respectively. Patients revisited the ED within 72 hours in 6.7% of patients in pre-implementation and 5.9% of patients in post-implementation. There were no patient deaths.</p><p><strong>Conclusions: </strong>Our pathway helps standardize the treatment of febrile pediatric patients with SCD. Although the decrease in admissions did not reach statistical significance, the > 10% decrease in admissions was likely meaningful to reduce health care burdens for patients and families.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":"47 2","pages":"80-85"},"PeriodicalIF":0.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11864588/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143492456","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Comparison of Capizzi and High-dose Methotrexate Approaches in the Treatment of Pediatric B-cell Acute Lymphoblastic Leukemia.","authors":"Ezgi Yalcin Gungoren, Basak Koc, Bulent Zulfikar","doi":"10.1097/MPH.0000000000002995","DOIUrl":"10.1097/MPH.0000000000002995","url":null,"abstract":"<p><p>Childhood cancers, with leukemia at the forefront, comprise 97% acute leukemia and 3% chronic leukemia, with 75% of acute leukemias being of lymphoblastic origin. Over the past 50 years, survival rates have witnessed a remarkable increase, progressing from around 10% to achieving cure rates exceeding 90% in certain childhood ALL subgroups with the advent of combined therapies. Between 1999 and 2018, a total of 123 patients diagnosed with B-ALL were initially identified, but after applying exclusion criteria, 105 patients were included in the evaluation, who were treated with COG protocols at our center. The mean follow-up duration for patients was determined to be a median of 74 months (min to max: 2 to 228 months). When the cases were evaluated at the end of the study, 59 of 59 individuals in the standard risk group (100%), 21 of 26 individuals in the high-risk group (80.7%), and 14 of 20 individuals in the very high group (70%) were alive. Patients were categorized into 4 groups based on the methotrexate (MTX) doses they received during Phase 3 and Phase 5 of treatment. Event-free survival and overall survival were evaluated among these groups. It was observed that patients in the standard-risk group had significantly higher event-free and overall survival rates. However, no significant difference was found in survival rates when evaluated based on the treatment groups each risk group received by the patients.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"67-73"},"PeriodicalIF":0.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142962296","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}

{"title":"Knowledge, Attitude, and Practice of Patients Suffering From Sickle Cell Disease in an Endemic Zone.","authors":"Garima Nirmal, Dinesh Pendharkar, Neha Gupta, Abhishek Raj","doi":"10.1097/MPH.0000000000002987","DOIUrl":"10.1097/MPH.0000000000002987","url":null,"abstract":"<p><strong>Background and objective: </strong>Sickle cell disease (SCD) is a genetically inherited disorder that is associated with morbidity and mortality.</p><p><strong>Methods: </strong>This cross-sectional study was conducted on patients diagnosed with SCD to evaluate the knowledge, attitude, and practice of patients/guardians using a pretested questionnaire.</p><p><strong>Results and discussion: </strong>Of the 111 participants, 56 (50.4%) were male. Forty-five participants reported SCD as a hereditary disease, and only 31 (37%) subjects reported that SCD could be prevented. Fourteen (70%) married patients reported that their children had been screened. Ninety-three (84%) children reported fatigue in the past 1 year. Seventy-four of them reported no absence from work/school in the past 1 year. Fifty-seven participants (52%) experienced 1 to 4 episodes of pain in the past 1 year, 62 and the majority (55.86%) of them visited a nearby doctor for the pain episode. Ninety-four participants were taking regular hydroxyurea as medication, and 72% had not missed any dose in the past 1 month, while 14.4% missed 1 to 2 doses in the past 1 month. Only 19 participants (17%) were aware that transplantation was a curative option. The majority (78%) reported undergoing blood and other investigations irregularly in the preceding year.</p><p><strong>Conclusions: </strong>The knowledge and attitude of patients/guardians regarding the nature of the disease, preventive measures, curative options for transplantation, and screening of family members are low. At the same time, most people are educated. In contrast to the world data, adherence to hydroxyurea is extremely good. Patient and family education and counseling are the needs of the hour.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"86-90"},"PeriodicalIF":0.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142962297","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}

{"title":"Treatment of T-cell Leukemia/Lymphoma in Children and Young Adults With the Memorial Sloan Kettering Cancer Center New York IIB Protocol.","authors":"Peter G Steinherz, Audrey Mauguen, Stephanie Suser, Kavitha Ramaswamy, Rachel Kobos, Christopher J Forlenza, Neerav Shukla, Tanya Trippett, Suzanne Wolden, Laurel Steinherz","doi":"10.1097/MPH.0000000000002999","DOIUrl":"10.1097/MPH.0000000000002999","url":null,"abstract":"<p><p>We described the Memorial Sloan Kettering Cancer Center New York IIB (MSK-NYII) protocol pilot in 1993, for the treatment of acute lymphoblastic leukemia (ALL) in children at high risk of early relapse. We now report MSK-NYII for the treatment of T-ALL/T-lymphoma over a 15-year period. A review of all patient charts identified 63 treated from 1/1/2000-12/31/2015, with a median follow-up of 9.9 years. Further follow-ups were confounded by the COVID pandemic. Remissions (CR) were defined as <5% marrow blast count on Day 28 and resolution of extramedullary disease. Forty-four had T-ALL, and 19 had stage III/IV lymphoblastic lymphoma (T-LL). Median age at diagnosis was 13.6 years (range 0.4 to 23.7). At diagnosis central nervous system (CNS) leukemia was present in 7/63 patients (11%), cranial nerve palsy in 3 (5%), CNS2 [<5cells/µL cerebrospinal fluid with blasts seen on cytospin] in 11 (17%), testicular enlargement in 3 (5%), and mediastinal mass in 45 (71%). On Day 8, 37 T-ALL (86%) were rapid early responders with <25% marrow blasts. 54 patients had an examination, cerebrospinal fluid, and marrow evaluation on day 28. Remission was demonstrated in 53 (98%) after prior marrow and CNS disease. The 19 T-lymphoma patients had no evidence of disease on day 28. Four relapses in marrow were recorded during therapy. Second remissions were able to be achieved. One patient died without having relapsed and is counted as an event in the event-free survival (EFS) analysis. Four patients, including 3 after the second CR transplant, died during follow-up. One unusual case of T-ALL recurred as T-LL of the colon 5.5 years after diagnosis, 3.5 years after therapy discontinuation. Fifteen years after diagnosis 88% (95% CI=78%-98%) survived event-free, and 91% (95% CI=82%-100%) survived. Twenty-five patients received irradiation. Three had RT to the testes, 3 had cranial RT for cranial nerve palsies, and 19 had cranial radiation for either CNS leukemia, CNS2, or for initial white blood cell >100,000/ µL. The MSK-NYIIB protocol, with a 94% 5-year and 88% 15-year EFS, is an effective therapy for the treatment of T-ALL/lymphoma, with similar toxicity to other high-risk regimens.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"59-66"},"PeriodicalIF":0.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143123008","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}

{"title":"A Survey of Sedation Practices for Adolescents and Young Adults With Acute Lymphoblastic Leukemia Undergoing Lumbar Puncture.","authors":"Irim Salik, Aaron J Blizard, Rozalia Abramov, Aleena Khan, Elizabeth Drugge, Jeremy Rosenblum","doi":"10.1097/MPH.0000000000002983","DOIUrl":"10.1097/MPH.0000000000002983","url":null,"abstract":"<p><strong>Purpose: </strong>Lumbar puncture is a frequently performed procedure for patients undergoing treatment for acute lymphoblastic leukemia. This brief procedure is frequently performed with sedation in young patients but with only local anesthesia in adults. Adolescent and young adult patients may be cared for by physicians with different training backgrounds and sedation preferences, making the utilization of sedation for lumbar punctures variable among providers. The benefits of sedation for young adults with leukemia undergoing lumbar puncture (analgesia, anxiolysis, safety) must be weighed against the obligate fasting interval, hospital cost, and resource allocation that is required.</p><p><strong>Methods: </strong>We conducted a survey of pediatric and medical oncologists who care for patients with acute lymphoblastic leukemia to assess their use of sedation for adolescents and young adults undergoing lumbar punctures as part of their cancer therapy. (see Supplemental Digital Content 1, http://links.lww.com/JPHO/A723 ).</p><p><strong>Results: </strong>Twenty-six percent of pediatric oncologists and 28% of medical oncologists completed the survey. Pediatric oncologists were more likely to perform lumbar punctures under sedation as compared with medical oncologists. This pattern remained consistent across all patient age ranges surveyed, despite no significant difference in the expected cumulative number of lumbar punctures that a patient was to undergo. Medical oncologists reported topicalization with local anesthetics before lumbar puncture more often than pediatric oncologists.</p><p><strong>Conclusion: </strong>Sedation practices for lumbar puncture during acute lymphoblastic leukemia treatment varies by specialty of treating oncologist.</p>","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e99-e103"},"PeriodicalIF":0.9,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142962222","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}