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Journal of Pediatric Hematology/Oncology
Journal Prestige (SJR): 0.459  Citation Impact (citeScore): 1 Number of Followers: 8  Hybrid journal (It can contain Open Access articles)ISSN (Print) 1077-4114 - ISSN (Online) 1536-3678 Published by LWW Wolters Kluwer  [297 journals]
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- Drug Induced Diabetes Mellitus in Pediatric Acute Lymphoblastic Leukemia:
Approach to Diagnosis and Management-
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Authors: Handattu, Koushik; Sharma, Lokesh K.; Vijayasekharan, Kalasekhar; Bhat K; Vasudeva; Aroor, Shrikiran; Sudhanshu, Siddhnath
Abstract:
Corticosteroids and l-asparaginase used in the treatment of pediatric acute lymphoblastic leukemia (ALL) can cause drug-induced diabetes mellitus (DIDM). DIDM can lead to dyselectrolytemia, a higher risk of infections including cellulitis, bacteremia, fungemia, and a higher incidence of febrile neutropenia and may have an impact on the outcome of ALL. Literature on the management of DIDM among children with ALL is sparse and the diagnostic criteria for pediatric diabetes should be carefully applied considering the acute and transient nature of DIDM during ALL therapy. Insulin remains the standard of care for DIDM management and the choice of Insulin regimen (stand-alone Neutral Protamine Hagedorn or basal bolus) should be based on the type and dose of steroids used for ALL and the pattern of hyperglycemia. A modest glycemic control (postmeal 140 to 180 mg/dL, premeal
PubDate: Mon, 01 Aug 2022 00:00:00 GMT-
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- New Approaches Promise to Improve Local Ewing Sarcoma Results
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Authors: Cohen; Ian J.; Ash, Shifra
Abstract: The study by Whelan and colleagues showed that addition of busulfan and melphalan conditioning and autologous stem cell rescue to conventional EURO-E.W.I.N.G STUDY chemotherapy in local nonmetastatic Ewing sarcoma improves prognosis. However, almost 30% of these study patients will have relapsed before this stage of therapy is reached, and 78% of his patients were at high risk because of inadequate response to the initial chemotherapy given. Further improvement could be achieved by the integration of other novel advances with this approach. Ash and colleagues have shown that the separation of such cases into high- and low-risk groups by using CD56 negativity of the tumor cells is an improvement over current methods with a 100% 10-year progression-free survival in CD56− nonpelvic local isolated Ewing sarcoma patients. Their patients were treated on the SCMCIE 94 protocol, associated with no relapses before 30 months in 24 consecutive patients independent of the CD status. Integration of these novel approaches in diagnosis and treatment would allow truly high-risk patients, who would benefit from the procedure, to reach the busulfan and melphalan stage of therapy and delineate those patients who can be cured without such therapy. Details of the SCMCIE 94 protocol are given and the possible reasons for the different relapse patterns are discussed.
PubDate: Mon, 01 Aug 2022 00:00:00 GMT-
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- Longer Time Intervals From Symptom Onset to Diagnosis Affect the Overall
Survival in Children With Acute Lymphoblastic Leukemia-
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Authors: Dai; Qingkai; Liu, Rui; Wang, Yuefang; Ye, Lei; Peng, Luyun; Shi, Rui; Guo, Siqi; He, Jiajing; Yang, Hao; Zhang, Ge; Jiang, Yongmei
Abstract:
Background: Acute lymphoblastic leukemia (ALL) is the most common childhood cancer. Early diagnosis and timely treatment are essential for effective cancer control and have been widely analyzed in childhood cancer. However, few studies have described the time to diagnosis and treatment in children with ALL. This study investigated delays in diagnosis and treatment initiation and their impact on survival.Methods: This retrospective cohort study included 419 patients 0 to 14 years old at a tertiary hospital between 2011 and 2015. The optimal cutoff values for delays were determined by X-tile software. The Kaplan-Meier method and Cox regression models were used to evaluate the impact of delays on survival.Results: The median diagnosis, treatment, and total delays were 21 (interquartile range [IQR]: 11-35), 4 (IQR: 2-7), and 26 (IQR: 16-43) days, respectively. The results of multivariate analyses showed that diagnosis delay, risk stratification, and minimal residual disease level were independent predictors for treatment outcome in childhood ALL.Conclusions: These findings suggested that a longer time to diagnosis negatively affected the clinical outcome of childhood ALL. Reducing the time to diagnosis could help to improve survival in these patients.
PubDate: Mon, 01 Aug 2022 00:00:00 GMT-
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- Multifarious Functions of Butyrylcholinesterase in Neuroblastoma: Impact
of BCHE Deletion on the Neuroblastoma Growth In Vitro and In Vivo-
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Authors: Baranowska-Kortylewicz; Janina; Kortylewicz, Zbigniew P.; McIntyre, Erin M.; Sharp, John G.; Coulter, Don W.
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The physiological functions of butyrylcholinesterase (BChE) and its role in malignancy remain unexplained. Our studies in children newly diagnosed with neuroblastoma indicated that BChE expressions is proportional to MYCN amplification suggesting that pathogenesis of high-risk disease may be related to the persistent expression of abnormally high levels of tumor-associated BChE. BChE-deficient neuroblastoma cells (KO [knockout]) were produced from MYCN-amplified BE(2)-C cells (WT [wild-type]) by the CRISPR-Cas9 targeted disruption of the BCHE locus. KO cells have no detectable BChE activity. The compensatory acetylcholinesterase activity was not detected. The average population doubling time of KO cells is 47.0±2.4 hours,>2× longer than WT cells. Reduced proliferation rates of KO cells were accompanied by the loss of N-Myc protein and a significant deactivation of tyrosine kinase receptors associated with the aggressive neuroblastoma phenotype including Ros1, TrkB, and Ltk. Tumorigenicity of WT and KO cells in male mice was essentially identical. In contrast, KO xenografts in female mice were very small (0.37±0.10 g), ~3× smaller compared with WT xenografts (1.11±0.30 g). Unexpectedly, KO xenografts produced changes in plasma BChE similarly to WT tumors but lesser in magnitude. The disruption of BCHE locus in MYCN-amplified neuroblastoma cells decelerates proliferation and produces neuroblastoma cells that are less aggressive in female mice.
PubDate: Mon, 01 Aug 2022 00:00:00 GMT-
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- Is Detection of Relapse by Surveillance Imaging Associated With Longer
Survival in Patients With Rhabdomyosarcoma'-
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Authors: Fetzko; Stephanie; Fonseca, Adriana; Frances Wedekind, Mary; Gupta, Abha A.; Setty, Bhuvana A.; Schraw, Jeremy; Lupo, Philip J.; Guillerman, Robert P.; Butala, Anish A.; Russell, Heidi; Nicholls, Lauren; Walterhouse, David; Hawkins, Douglas S.; Okcu, Mehmet F.
Abstract:
Background: We investigated whether surveillance imaging had an impact on post-relapse survival in patients with rhabdomyosarcoma (RMS). We hypothesized that relapse detected by imaging (group IM) would be associated with longer survival compared with relapse detected with a clinical sign or symptom (group SS).Materials and Methods: We performed an observational multi-institutional study in 127 patients with relapsed RMS comparing overall survival (OS) after relapse using Kaplan-Meier and Cox proportional hazards analyses.Results: Relapse was detected in 60 (47%) group IM and 67 (53%) SS patients. Median follow-up in survivors was 4 years (range 1.0 to 16.7 y). Four-year OS rates were similar between group IM (28%, 95% confidence interval [CI]: 14%-40%) and SS (21%, 95% CI: 11%-31%) (P=0.14). In multivariable analyses accounting for institution, age at diagnosis, time to relapse, risk group at diagnosis, and primary site, not receiving chemotherapy (hazard ratio [HR]: 6.8, 95% CI: 2.8-16.6), radiation (HR: 3, 95% CI: 1.7-5.3), or surgery (HR: 2.8, 95% CI: 1.6-4.8) after relapse were independently associated with poor OS.Conclusion: These results on whether surveillance imaging provides survival benefit in patients with relapsed RMS are inconclusive. Larger studies are needed to justify current surveillance recommendations. Chemotherapy, radiotherapy and surgery to treat recurrence prolong OS.
PubDate: Mon, 01 Aug 2022 00:00:00 GMT-
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- Considerations in Methods and Timing for Delivery of Genetic Counseling
Information to Pediatric Oncology Patients and Families-
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Authors: Li; Kathleen A.; Sloat, Lauren M.; Kung, Julia; Jung, Jessica; Li, Ashley; Smith, Christy H.; Schratz, Kristen E.; Cooper, Stacy L.; Pratilas, Christine A.; Frankenfield, Pamela; Bodurtha, Joann
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Many pediatric oncology patients and their families may benefit from genetic counseling and testing; however, identifying the best timing and delivery method for these referrals is sometimes a challenge. The goal of this study was to understand how and when caregivers prefer to receive information about genetic counseling and testing. A total of 56 surveys completed by caregivers at The Johns Hopkins Hospital Pediatric Oncology unit in Baltimore, Maryland were analyzed. A sizeable subset of respondents was interested in receiving information about the availability of genetic counseling from an oncology doctor or nurse, but not a genetic counselor (n=13/55, 24%). Most respondents preferred to be informed about genetic services at diagnosis (n=28/54, 52%) or within 1 to 2 months of diagnosis (n=14/54, 26%). In conclusion, patients and their families may benefit from prompt and early recognition of the risk of cancer predisposition syndromes, preferably within the first 2 months following diagnosis. Oncology professionals are an important source of information, and can introduce the availability of genetic counseling services and motivate families to undergo genetic testing, though alternative communication methods such as brochures may also be useful.
PubDate: Mon, 01 Aug 2022 00:00:00 GMT-
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- Pediatric Benign Neutropenia: Assessing Practice Preferences in Canada
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Authors: Chok; Rozalyn; Price, Victoria; Steele, MacGregor; Corriveau-Bourque, Catherine; Bruce, Aisha
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Pediatric benign neutropenia is a self-limited condition with a benign clinical course. An approach to this condition is not well-defined in the literature. Our objective was to use a case-based survey to elucidate trends in the diagnosis and management of benign neutropenia among pediatric hematology/oncology practitioners in Canada. We received 46 completed surveys (response rate 66%). At initial presentation with fever and neutropenia, 67% of respondents recommended partial septic workup but 11% recommended no investigations. Nearly 70% recommended admission for empiric intravenous antibiotics, while 24% would discharge home without antibiotics. In a patient with fever and known neutropenia, respondents were more likely to pursue outpatient antibiotic therapy. For investigation of chronic neutropenia, most respondents (60%) do not use antineutrophil antibody testing. Common indications for bone marrow biopsy were severe infection, prolonged neutropenia, or before initiating granulocyte colony stimulating factor. Indications for granulocyte colony stimulating factor were based on severity and frequency of infection. Most respondents (84%) would not recommend antibiotic prophylaxis. Results demonstrate the considerable variability in management of benign neutropenia among pediatric hematology/oncology practitioners in Canada and highlight the need for prospective studies to establish diagnostic criteria for benign neutropenia and evaluate management of fever in this population.
PubDate: Mon, 01 Aug 2022 00:00:00 GMT-
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- Coagulation Abnormalities and Clinical Complications in Children With
SARS-CoV-2: A Systematic Review of 48,322 Patients-
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Authors: McDaniel; Charles Griffin; Commander, Sarah J.; DeLaura, Isabel; Cantrell, Sarah; Leraas, Harold J.; Moore, Carrie B.; Reed, Christopher R.; Pahl, Kristy S.; Tracy, Elisabeth T.
Abstract:
Given the limited information on the coagulation abnormalities of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) in pediatric patients, we designed a systematic review to evaluate this topic. A comprehensive literature search was conducted for “SARS-CoV-2,” “coagulopathy,” and “pediatrics.” Two authors independently screened the articles that the search returned for bleeding, thrombosis, anticoagulant and/or antiplatelet usage, and abnormal laboratory markers in pediatric patients with SARS-CoV-2, and the authors then extracted the relevant data. One hundred twenty-six publications were included. Thirty-four (27%) studies reported thrombotic complications in 504 patients. Thirty-one (25%) studies reported bleeding complications in 410 patients. Ninety-eight (78%) studies reported abnormal laboratory values in 6580 patients. Finally, 56 (44%) studies reported anticoagulant and/or antiplatelet usage in 3124 patients. The variety of laboratory abnormalities and coagulation complications associated with SARS-CoV-2 presented in this review highlights the complexity and variability of the disease presentation in infants and children.
PubDate: Mon, 01 Aug 2022 00:00:00 GMT-
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- Predictive Value of Breg and Serum IL-10 Concentration Levels for Acute
ITP Progression to Chronic Phase-
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Authors: Aref; Salah; El Menshawy, Nadia; Darwish, Ahmad; Farag, Nora A.
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Introduction: Pediatric immune thrombocytopenia (ITP) is a potentially life threating autoimmune disorder with different responses to therapy and different bleeding phenotypes in critical organs. The molecular basis for the variable response has not yet been fully elucidated. This study was designed to address the predictive value of regulatory B-cell (Breg) count and interleukin-10 (IL-10) serum levels for acute ITP patients who progress to chronic phase. The present study included 80 children with acute ITP )38 males and 42 females (with median age of 8 years and 40 matched healthy controls. Assessment of Breg (CD19+CD24hiCD38hi) was carried out by a multicolor flowcytometry, however, IL-10 serum levels were evaluated by enzyme-linked immunosorbent assay. A significant reduction of Breg percentage and a significant increase in serum IL-10 levels were identified in children with acute ITP as compared with controls (P
PubDate: Mon, 01 Aug 2022 00:00:00 GMT-
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- Children With Asparaginase-associated Pancreatitis Present Elevated Levels
of Insulin, Total Cholesterol, and HOMA-IR Before Starting Acute
Lymphoblastic Leukemia Treatment-
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Authors: Chávez-Aguilar; Lénica A.; Ávila-Castro, David; Merino-Pasaye, Laura E.; Peña-Vélez, Rubén
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Asparaginase-associated pancreatitis frequently occurs in children with cancer. It is unknown if other factors can influence the development of pancreatitis. A total of 33 pediatric patients with a confirmed diagnosis of acute lymphoblastic leukemia were included in this study. Before acute lymphoblastic leukemia drug treatment, the metabolic parameters (glucose, insulin, homeostasis model assessment insulin resistance, total cholesterol, triglycerides) and body mass index percentile were compared. Children who had acute pancreatitis had higher levels of insulin, homeostasis model assessment insulin resistance, and total cholesterol, compared with children who did not develop acute pancreatitis. These metabolic alterations could play a role in the development of pancreatitis.
PubDate: Mon, 01 Aug 2022 00:00:00 GMT-
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- Shwachman-Diamond Syndrome With Congenital Myogenic Ptosis: Case Report of
a Rare Association'-
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Authors: Wang; Nai-Wei; Georgara, Natalia; Khan, Haji S.
Abstract: Background: Shwachman-Diamond syndrome (SDS) is a multisystem disorder characterized by exocrine pancreatic insufficiency and bone marrow failure. There is considerable variation in the phenotypes of SDS. We present a case of an infant presenting with SDS and left-sided ptosis.Observation: We report a case of an infant who presented with 2 episodes of severe sepsis and cytopenia, without overt symptoms of exocrine pancreatic deficiency or skeletal abnormalities. Persistent left-sided ptosis was noted in both presentations. Genetic testing confirmed the diagnosis of SDS. The left-sided ptosis was diagnosed as congenital myogenic ptosis.Conclusion: The association of ptosis and other congenital bone marrow failure syndromes is well established, but this is the first description of SDS with ptosis. This association may expand our understanding of SDS phenotypes if similar cases are reported in the future.
PubDate: Mon, 01 Aug 2022 00:00:00 GMT-
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- Pyrites: Proptosis
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Authors: Lyvannak; Sam; Khauv, Phara; Keller, Frank G.; Leventaki, Vasiliki; Camitta, Bruce
Abstract:
No abstract available
PubDate: Mon, 01 Aug 2022 00:00:00 GMT-
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- Endothelium-derived Microparticles Are Increased in Teenagers With
Cobalamin Deficiency-
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Authors: Dundar; Mehmet A.; Torun, Yasemin A.; Cetin, Feyza; Oz, Hatice T.
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Introduction: Vitamin B12 (cobalamin) deficiency may be a significant cause of hyperhomocysteinemia, and high homocysteine (Hcy) levels are associated with an increased risk of cardiovascular disease. Endothelium-derived microparticles (EMPs) are a new marker in endothelial dysfunction and atherosclerosis, which play a role in cardiovascular diseases’ pathogenesis. This study aimed to evaluate the EMPs, the markers of endothelial dysfunction and atherosclerosis, and lipid profile in teenagers with cobalamin deficiency.Materials and Methods: This prospective study included 143 teenagers, 75 vitamin B12 deficient patients and 68 healthy controls between 11 and 18 years of age. Routine laboratory tests, hemogram, vitamin B12, folic acid, ferritin, Hcy, lipid profile and EMPs were examined and compared. EMP subgroups were analyzed by flow cytometry method according to the expression of membrane-specific antigens. The microparticles released from the endothelium studied were VE-cadherin (CD144), S-endo1 (CD146), and Endoglin (CD105).Results: The present study demonstrates that circulating CD105+ EMP, CD144+ EMP, CD146+ EMPs, and Hcy were increased, and high-density lipoprotein (HDL) cholesterol was reduced in teenagers with cobalamin deficiency. Vitamin B12 showed a negative correlation with EMPs and Hcy, positive correlation with folate and HDL. All EMPs showed a significant positive correlation with triglyceride, vitamin B12, and HDL.Conclusion: Vitamin B12 deficiency may predispose to endothelial damage and atherosclerosis by increasing EMPs and harms lipid metabolism in the long term.
PubDate: Mon, 01 Aug 2022 00:00:00 GMT-
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- Spirometric Changes After Initiation of Hydroxyurea in Children With
Sickle Cell Anemia-
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Authors: Kotwal; Nidhi; Pillai, Dinesh K.; Darbari, Deepika S.; Sun, Kai; Koumbourlis, Anastassios C.
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Individuals with sickle cell disease (SCD) develop a decline in lung function over time. Hydroxyurea (HU) is the most common disease-modifying therapy used in SCD. We hypothesized that children with SCD treated with HU will have a slower decline in pulmonary function. We performed a retrospective chart review of children with HbSS and HbS-beta zero thalassemia referred to pulmonology for respiratory symptoms. We compared the spirometry results at 2 time points between children on HU (HU group) and not on HU (control group). For the HU group, these endpoints were evaluated before and after being on HU. The mean time interval between 2 spirometry studies was not significantly different between the groups (2.6±1.5 y for HU group vs. 3.0±1.8 y for the control group; P=0.33). The mean age of patients in the HU group was 9.8±3.8 years (55% male) and 10.7±4.9 years (50% male) in the control group. The spirometry data was compared within and between the groups using t test. There was a significant increase in forced vital capacity in HU group during follow-up, while children in the control group showed a decline (7.2±17.1 vs. −3.4±18.2; P
PubDate: Mon, 01 Aug 2022 00:00:00 GMT-
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- Pulmonary Outcomes After Autologous Stem Cell Transplant for Hodgkin
Lymphoma-
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Authors: Davidow; Kimberly; Bunin, Nancy; Goldfarb, Samuel; Li, Yimei; Freedman, Jason L.
Abstract:
Autologous hematopoietic stem cell transplant (ASCT) may be curative therapy for pediatric patients with relapsed/refractory Hodgkin lymphoma (HL). Therapy for HL may involve pulmonary toxic modalities. Little information exists regarding pulmonary function in these patients post-ASCT. A retrospective chart review was performed for patients undergoing ASCT from February 2012 to December 2019. Lung disease was defined as a z-score ≤−1.7 in forced expiratory volume in the first second (FEV1), forced vital capacity (FVC), total lung capacity (TLC), or diffusing capacity of lung for carbon monoxide. Descriptive and limited statistical analyses were performed. Twenty-eight patients were included. Median age at diagnosis was 15 (2 to 19) and was 17 (4 to 21) at ASCT. Twenty-three received radiation before ASCT. Fourteen received brentuximab before, and 9 after, transplant. Nineteen met criteria for lung disease post-ASCT. Sixteen had lung disease before ASCT. Longitudinal trends for pulmonary function testing parameters did not reach statistical significance, however, FEV1, FVC, and TLC trended towards worsening immediately post-transplant. There was no statistically significant change in FEV1, FVC, or TLC at 2 years as compared with pretransplant data, suggesting no substantial difference from baseline. Diffusing capacity of lung for carbon monoxide showed statistically significant improvement at the 2 year timepoint (P=0.03). This data reinforces the importance of close follow-up for these patients. Large cohort studies are necessary to identify risk factors so that possible mitigative strategies or alternate regimens could be used.
PubDate: Mon, 01 Aug 2022 00:00:00 GMT-
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- Myeloablative Conditioning Regimen in Haploidentical Stem Cell
Transplantation With Posttransplant Cyclophosphamide in Children With
High-risk Hematologic Malignancies-
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Authors: Dufort y Alvarez; Gustavo
Abstract:
Limited information is available on outcomes of haploidentical stem cell transplantation (haploSCT) with posttransplant cyclophosphamide using myeloablative conditioning regimens in children and adolescents. We report the results of a single-institution retrospective study of myeloablative haploSCT in 36 children and adolescents (median age, 8 y; range, 9 mo to 22 y) with high-risk hematologic malignancies. Donor engraftment occurred in 31 of 33 evaluable patients (94%). Recovery of neutrophils and platelets occurred at a median of 15 and 20 days. Cumulative incidence of acute graft-versus-host-disease (GVHD) grades II to IV and grades III to IV at 100 days was 36±8.7% and 10±5.4% and of chronic GVHD at 1 year was 55±9.2%, with 31±8.6% moderate to severe. Nonrelapse mortality was 16±6.1% and 22±6.9% at 100 days and 1 year. The cumulative incidence of relapse at 4 years was 32±8.8%. With a median follow-up of 57 months (range, 8 to 89 mo), the overall survival and event-free survival at 4 years was 55.6±8.7% and 44.8±8.5%. Myeloablative conditioning T-replete haploSCT with posttransplant cyclophosphamide is a viable alternative to matched unrelated transplantation for children and adolescents with high-risk hematologic malignancies. The high rates of nonrelapse mortality and chronic GVHD is a concern and deserves careful consideration.
PubDate: Mon, 01 Aug 2022 00:00:00 GMT-
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- Effect of BCG HSP70 Gene Transfection on Dendritic Cells Derived From Bone
Marrow in Children With Acute Leukemia-
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Authors: Wang; Junlin; Li, Xiaoling; Liu, Chunlei; Wang, Shuli; Li, Jianqin
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Objectives: In this study, immature dendritic cells (imDCs) were transfected with the Bacillé Calmette-Guérin (BCG) heat shock protein 70 (HSP70) gene to investigate the impact on the maturity and function of imDCs from the bone marrow of pediatric patients with acute leukemia.Materials and Methods: Bone marrow mononuclear cells were isolated from pediatric patients with acute lymphoblastic leukemia who had achieved complete remission at least 6 months prior. The recombinant vector pDisplay-HSP70 was transfected into imDCs. The test groups included 5 subgroups: imDCs (imDCs without special processing), imDC-neos (imDCs transfected with the pDisplay vector), HSP70 (imDCs transfected with the pDisplay-HSP70 vector), tumor necrosis factor α (TNF-α) (imDCs induced with rhTNF-α), and HSP70+TNF-α. Mature dendritic cells (mDCs) from different groups (HSP70, TNF-α, and HSP70+TNF-α) and T cells were cultured. An equal number of lymphocytes and mDCs were used as controls. The proliferation indices of T cells and the cytokine contents (interleukin-12 and interferon-γ) were determined.Results: The HSP70 group and the TNF-α group expressed higher levels of HLA-DR, CD80, and CD86 but lower levels than the HSP70+TNF-α group; there was no significant difference between the HSP70 group and the TNF-α group. The combination of HSP70 and TNF-α induced the highest levels of interleukin-12 and interferon-γ.Conclusions: The outcomes of this study indicated that gene transfection with BCG HSP70 evidently promoted imDC maturity and the antitumor effects of mDC-mediated T cells. It could serve as a candidate gene-modified cell vaccine for tumor immunotherapy.
PubDate: Mon, 01 Aug 2022 00:00:00 GMT-
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- Vanishing Bile Duct Syndrome Secondary to Hodgkin Lymphoma in a Child
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Authors: Palla Velangini; Swetha; Boddu, Deepthi; Balakumar, Shailaja; Premanand, Arul; Kishore, Ravi; Mathew, Leni G.
Abstract:
Vanishing bile duct syndrome (VBDS) is a condition resulting from progressive destruction and loss of intrahepatic bile ducts leading to cholestasis, biliary cirrhosis, and liver failure. It occurs secondary to various pathologic conditions like autoimmune diseases, graft versus host disease, drug reactions, and as a paraneoplastic syndrome in malignancies. We here described a 9-year-old girl who presented with cervical lymphadenopathy and jaundice. This child was diagnosed as a case of Hodgkin lymphoma. All other causes of cholestasis were ruled out by appropriate investigations (particularly autoimmune, metabolic, infections, and drug-induced possibilities). On liver biopsy, her diagnosis was established as VBDS. In view of hepatic dysfunction, alternative chemotherapy with dexamethasone, high-dose cytarabine, and cisplatin (DHAP) was given, and she was started on hepatoprotective measures with ursodeoxycholic acid. Hepatic function gradually improved after the initiation of chemotherapy. VBDS is considered a dismal paraneoplastic syndrome with a high-case fatality. This case report highlights the importance of early recognition and initiation of appropriate full-dose chemotherapy as the only way to achieve complete resolution of VBDS.
PubDate: Mon, 01 Aug 2022 00:00:00 GMT-
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- First-line Anti-GD2 Therapy Combined With Consolidation Chemotherapy in 3
Patients With Newly Diagnosed Metastatic Ewing Sarcoma or Ewing-like
Sarcoma-
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Authors: Spasov; Neofit J.; Dombrowski, Frank; Lode, Holger N.; Spasova, Mariya; Ivanova, Liliya; Mumdjiev, Ivan; Burnusuzov, Hassan; Siebert, Nikolai
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Despite multimodal therapy, the prognosis of patients with metastatic Ewing sarcoma (ES) remains poor, with new treatments urgently needed. The disialoganglioside GD2, a well-established tumor-associated antigen, is expressed in 40% to 90% of ES cells, making it a suitable therapeutic target. Here we report 3 cases with newly diagnosed, metastatic, GD2-positive ES or Ewing-like sarcoma treated with the anti-GD2 antibody dinutuximab beta in addition to standard chemotherapeutic regimens. Treatment was well-tolerated, and all patients achieved complete remission, without evidence of relapse. First-line anti-GD2 immunotherapy in patients with metastatic, GD2-positive ES or Ewing-like sarcoma represents a promising therapeutic option that warrants further clinical evaluation.
PubDate: Mon, 01 Aug 2022 00:00:00 GMT-
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- Prevalence of COVID-19 in Egyptian Children With Hemoglobinopathies and
Inherited Anemias-
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Authors: Ismail; Ahlam M.; Mahmoud, Nagwa M.S.; Ghazawy, Eman R.; Mousa, Suzan O.
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Since the World Health Organization (WHO) announced coronavirus disease-2019 (COVID-19) to be a pandemic, children’s COVID-19 cases were generally less severe than adults. The aim of the study was to determine the prevalence of COVID-19 cases among children with hemoglobinopathies and other inherited anemias living in El-Minya Governorate, Egypt, who are at high risk of exposure to infection. This cross-sectional study evaluated data from 258 children with hemoglobinopathies and inherited anemias. A questionnaire was used to collect data about COVID-19 symptoms coupled with appropriate investigations (complete blood count, d-dimer, anti-COVID antibodies, chest computed tomography scans, and polymerase chain reaction). We found 38 of 258 (14.7%) children had mild to moderate COVID-19, while there were no cases with severe form of COVID-19. COVID-19 cases were significantly older (8.63±3.37 vs. 6.71±3.56 y, P=0.01), noncompliant to iron chelators (63.2% vs. 11.8%, P=0.01), had higher serum ferritin (2639.47±835.06 vs. 1038.95±629.87 ng/mL, P
PubDate: Mon, 01 Aug 2022 00:00:00 GMT-
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- Allogeneic Bone Marrow Transplant as a Cure for Refractory T-Cell Large
Granular Lymphocytic Leukemia in an Adolescent-
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Authors: Fries; Carol; Evans, Andrew G.; Cheon, HeeJin; Korones, David N.; Loughran, Thomas P. Jr; Andolina, Jeffrey R.
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T-cell large granular lymphocytic (T-LGL) leukemia is a rare, typically indolent neoplasm with a median age of onset above 60 years. Pathogenesis involves clonal T-cell expansion, and nearly all reported pediatric cases have been associated with concurrent autoimmune disease. Immunosuppressive therapy often mitigates sequelae, but definitive cure is not routinely achieved. Here we present an otherwise healthy 13-year-old with T-LGL leukemia refractory to all standard treatments. Our patient ultimately underwent allogeneic bone marrow transplant (BMT) and is now stable in remission 3 years post-BMT. BMT may offer a viable definitive cure for refractory T-LGL leukemia in very young patients.
PubDate: Mon, 01 Aug 2022 00:00:00 GMT-
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