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Journal of Pediatric Hematology/oncology

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https://www.readbyqxmd.com/read/28538091/wolman-disease-a-mimic-of-infant-leukemia
#1
Kaduveettil G Gopakumar, Priyakumari Thankamony, Sheela Nampoothiri, Deeksha Bali, Jubie Raj, Jayasudha A Vasudevan, Ramachandran K Nair
BACKGROUND: Infant leukemia most commonly present with pallor and hepatosplenomegaly. The possibility of other differential diagnosis also has to be kept in mind during evaluation, as identifying the precise etiology for this clinical presentation is crucial for management. OBSERVATION: An infant, was referred to us with suspected infant leukemia and was subsequently diagnosed to have lysosomal acid lipase deficiency/Wolman disease with a novel 5 bp deletion "c...
May 23, 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/28538090/late-effects-in-pediatric-high-risk-neuroblastoma-survivors-after-intensive-induction-chemotherapy-followed-by-myeloablative-consolidation-chemotherapy-and-triple-autologous-stem-cell-transplants
#2
Amy E Armstrong, Karina Danner-Koptik, Shannon Golden, Jennifer Schneiderman, Morris Kletzel, Jennifer Reichek, Yasmin Gosiengfiao
Multimodal treatment in high-risk neuroblastoma has modestly improved survival; limited data exist on the late effects from these regimens. We report the sequelae of treatment incorporating 3 consecutive cycles of high-dose therapy and autologous stem cell transplants (ASCTs) without the use of total body irradiation (TBI). We reviewed the medical records of 61 patients treated on or following the Chicago Pilot 2 protocol between 1991 and 2008. Of the 25 patients who are alive (41%), 19 had near complete data to report...
May 23, 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/28538089/central-hematocrit-levels-in-fetal-malnourished-term-infants
#3
Kerem Ertaş, Özgül Salihoğlu, Emrah Can, Sami Hatipoğlu, Şahin Hamilçikan
BACKROUND: Fetal malnutrition is especially important for common chronic diseases in adult life. They could potentially be prevented by achieving optimal fetal nutrition. OBJECTIVE: The aim of this study was to investigate hematocrit levels of malnourished, term, appropriate for gestational age (AGA) neonates. SUBJECTS AND METHODS: A total of 80 AGA neonates (between 10% and 90% percentiles interval according to birth week), born with spontaneous vaginal delivery between 37 and 42 weeks of gestation, detected by both last menstrual period and ultrasonography measurements, were included in the study...
May 23, 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/28538514/long-term-outcome-of-4-patients-with-transcobalamin-deficiency-caused-by-2-novel-tcn2-mutations
#4
Marwan Nashabat, Gustavo Maegawa, Peter H Nissen, Ebba Nexo, Hussain Al-Shamrani, Mohammed Al-Owain, Majid Alfadhel
Cobalamin (vitamin B12 [Cbl]) is an essential cofactor for many biochemical pathways. Transcobalamin (TC) is required to internalize Cbl into the cells through membrane receptor-mediated endocytosis. Cbl is then processed in the cytoplasm and mitochondria by complementation factors leading to its active metabolites; methylcobalamin and 5-deoxyadenosyl-cobalamin. Deficiency of TC results in an elevation in methylmalonic acid and homocysteine. Patients usually present with macrocytic anemia, pancytopenia, failure to thrive, gastrointestinal symptoms, and neurological dysfunction...
May 22, 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/28538513/attention-deficit-hyperactivity-disorder-in-children-with-sickle-cell-disease-referred-for-an-evaluation
#5
Melissa A Acquazzino, Meghan Miller, Matthew Myrvik, Robert Newby, John Paul Scott
Neuropsychological deficits, including difficulties with attention, are well described in children with sickle cell disease (SCD). Very little is known about attention deficit hyperactivity disorder (ADHD) in children with SCD. The objective of this study was to determine the proportion of ADHD in children with SCD referred for neuropsychological evaluation. This prospective, cross-sectional study included patients (age, 4 to 18 y) with SCD and completion of a neuropsychological evaluation between December 2013 and March 2016...
May 22, 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/28538512/necrotizing-ulcer-after-bcg-vaccination-in-a-girl-with-leukocyte-adhesion-deficiency-type-1
#6
Hidemitsu Kurosawa, Tomoyuki Mizukami, Hiroyuki Nunoi, Masaya Kato, Yuya Sato, Mayuko Okuya, Keitaro Fukushima, Yoshihiko Katsuyama, Osamu Arisaka
Leukocyte-adhesion deficiency-1 is a recessively inherited disorder associated with recurrent bacterial infections, severe periodontitis, peripheral leukocytosis, and impaired wound healing. We diagnosed moderate-type leukocyte-adhesion deficiency-1 in a 7-year-old girl who developed a necrotizing ulcer after Bacillus Calmette-Guerin vaccination. The patient showed moderate expression of CD18 in neutrophils with a homozygous splice mutation with c.41_c.58+2dup20 of ITGB2 and experienced recurrent severe infections complicated with systemic lupus erythematosus...
May 22, 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/28538511/successful-treatment-of-transplantation-associated-atypical-hemolytic-uremic-syndrome-with-eculizumab
#7
Daiichiro Hasegawa, Atsuro Saito, Nanako Nino, Suguru Uemura, Satoru Takafuji, Takehito Yokoi, Aiko Kozaki, Toshiaki Ishida, Keiichiro Kawasaki, Takahiro Yasumi, Naoki Sakata, Yasufumi Ohtsuka, Satoshi Hirase, Takeshi Mori, Noriyuki Nishimira, Mayumi Kusumoto, Yoshiharu Ogawa, Kenta Tominaga, Taku Nakagawa, Kyoko Kanda, Ryojiro Tanaka, Yoshiyuki Kosaka
We herein reported a 4-month-old boy with transplantation-associated atypical hemolytic uremic syndrome (TA-aHUS) who was successfully treated with eculizumab. The patient diagnosed with type 3 of familial hemophagocytic lymphohistiocytosis underwent cord blood transplantation. After transplantation, he developed TA-aHUS, but plasma exchanges were unsuccessful. We identified deletions in CFH-related gene 1 (del-CFHR1) by the multiplex ligation-dependent probe amplification testing procedure and CFH autoantibodies...
May 22, 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/28538510/thalidomide-induced-stroke-in-a-child-with-thalassemia-major
#8
Sushil Gunaseelan, Anand Prakash
β-thalassemia major is a hereditary anemia resulting from defects in β-globin production. It is also characterized by a hypercoagulable state with an increased risk of thrombosis. Thalidomide, a drug known for its immunomodulating and antiangiogenic properties, has recently been demonstrated to induce γ-globin gene expression and to increase the proliferation of erythroid cells. An increasing incidence of thromboembolic events in thalidomide-treated patients has been reported. This is often in the context of thalidomide combinations with other drugs, including steroids and particularly anthracycline-based chemotherapy, and with very low incidence of thrombosis with single-agent thalidomide treatment...
May 22, 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/28538509/clinical-features-and-prognosis-according-to-immunophenotypic-subtypes-including-the-early-t-cell-precursor-subtype-of-t-lymphoblastic-lymphoma-in-the-japanese-pediatric-leukemia-lymphoma-study-group-alb-nhl03-study
#9
Reiji Fukano, Shosuke Sunami, Masahiro Sekimizu, Tetsuya Takimoto, Tetsuya Mori, Tetsuo Mitsui, Takeshi Mori, Akiko Moriya Saito, Tomoyuki Watanabe, Koichi Ohshima, Junichiro Fujimoto, Atsuko Nakazawa, Nobutaka Kiyokawa, Ryoji Kobayashi, Keizo Horibe, Masahito Tsurusawa
We reviewed the immunophenotypic subtypes of pediatric T-cell lymphoblastic lymphoma in the Japanese Pediatric Leukemia/Lymphoma Study Group ALB-NHL03 study. Of the 104 patients, 40 patients each had sufficient data to evaluate the immunophenotypes and early T-cell precursor (ETP) subtype. Pro-T, pre-T, intermediate T, and mature T cells were observed in 1, 9, 21, and 9 cases, respectively. The 3-year event-free survival (EFS) rates of those with pro-T/pre-T, intermediate T, and mature T cells were 80.0±12...
May 22, 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/28538508/central-nervous-system-disease-education-and-race-impact-radiation-refusal-in-pediatric-cancer-patients
#10
Chirayu G Patel, Mark Stavas, Stephanie Perkins, Eric T Shinohara
To investigate the determinants of radiation therapy refusal in pediatric cancer, we used the Surveillance, Epidemiology, and End Results registry to identify 24,421 patients who met the eligibility criteria, diagnosed between 1974 and 2012. Patients had any stage of cancer, were aged 0 to 19, and received radiation therapy or refused radiation therapy when it was recommended. One hundred twenty-eight patients (0.52%) refused radiation therapy when it was recommended. Thirty-two percent of patients who refused radiation therapy ultimately died from their cancer, at a median of 7 months after diagnosis (95% confidence interval, 3-11 mo), as compared with 29...
May 22, 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/28538507/female-sex-bilateral-disease-age-below-3-years-and-apprehension-for-enucleation-contribute-to-treatment-abandonment-in-retinoblastoma
#11
Anish Bhargav, Usha Singh, Amita Trehan, Zoramathara Zadeng, Deepak Bansal
Treatment abandonment is a major obstacle for treating retinoblastoma in developing countries. The aim was to evaluate plausible causes, outcome, and rate of treatment abandonment in patients with retinoblastoma. The study was retrospective and conducted in a University hospital in North-India. Two cohorts of patients were studied. One was a larger cohort (n=602) of all patients with retinoblastoma in the institution from 2000 to 2014. Limited variables (age, sex, and laterality) were examined in this cohort due to incomplete data/lack of contact...
May 22, 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/28538506/allogeneic-hematopoietic-cell-transplantation-for-dyskeratosis-congenita-a-report-of-3-cases
#12
Shinichi Tamura, Toshihiko Imamura, Takayo Urata, Miki Kobayashi, Mari Gen, Toshihiro Tomii, Junko Do, Shinya Osone, Hiroyuki Ishida, Hajime Hosoi, Hiroshi Kuroda
Although bone marrow failure in patients with dyskeratosis congenita (DKC) can be successfully treated with allogeneic hematopoietic cell transplantation (allo-HCT) using a reduced intensity conditioning (RIC) regimen, the outcome of nonhematological disorders in patients with DKC treated with allo-HCT using RIC has not been fully elucidated. Here, we describe the clinical course of nonhematological disorders after allo-HCT with RIC in 3 consecutive patients with DKC. Allo-HCT with RIC was feasible in all cases; however, patient 1 developed lethal pulmonary disease and patient 2 experienced progression of hepatic fibrosis...
May 22, 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/28538505/thalassemia-major-and-priapism-a-case-report-of-an-adolescent
#13
Sibel Öz, Serhan Küpeli, Gülay Sezgin, İbrahim Bayram
Priapism is defined as a prolonged pathologic penile erection without sexual stimulation. In children, priapism secondary to sickle cell disease or hematological malignancy is a frequent condition. Appropriate treatment of priapism varies; the treatment is primarily etiological, conservative management. In the present report, we aimed to present a case of asplenic thalassemia major who developed priapism, improved with hydration and ibuprofen treatment. Clinicians should take into account that priapism can be encountered in patients with thalassemia major...
May 22, 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/28538504/clofarabine-dosing-in-a-patient-with-acute-myeloid-leukemia-on-intermittent-hemodialysis-case-report-and-review-of-the-literature
#14
Lydia L Benitez, Katherine Gharibian, David Frame, Rajen Mody, Erika Mora
Clofarabine containing chemotherapeutic regimens have demonstrated efficacy in the treatment of relapsed refractory acute myeloid leukemia. Nonetheless, there are limited data on the use of clofarabine in patients with renal failure. The present report describes the use of clofarabine in a patient with renal failure undergoing intermittent dialysis. We describe our rationale for dosing, clofarabine plasma levels obtained, and discuss our findings in the context of other available literature. Consistent with previous findings, intermittent hemodialysis was not found to be a reliable method of removing clofarabine in patients with renal insufficiency...
May 22, 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/28538503/complete-resection-of-pancreatoblastoma-with-portal-vein-obstruction-after-high-dose-chemotherapy-a-case-report
#15
Souji Ibuka, Shuichiro Uehara, Takehisa Ueno, Takaharu Oue, Takako Miyamura, Yoshiko Hashii, Hiroomi Okuyama
BACKGROUND: As no standard chemotherapy exists for pancreatoblastoma (PB), surgical resection is the most effective way of achieving complete remission. CASE REPORT: A 3-year-old girl with PB causing portal vein obstruction was referred to our hospital. Because of the portal vein involvement, she initially received 5 cycles of preoperative chemotherapy after biopsy, consisting of cyclophosphamide, vincristine, pirarubicin, and cisplatin. After chemotherapy, she underwent distal pancreatectomy, and the tumor was resected completely...
May 22, 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/28538502/successful-reduced-intensity-allogeneic-transplant-with-full-donor-chimerism-and-good-quality-of-life-in-adolescent-patient-with-wiskott-aldrich-syndrome
#16
Salah Ali, Anna Gacsadi, Elizabeth McDougall, Christine Armstrong, Joerg Krueger, Tal Schechter, Muhammad Ali
Wiskott-Aldrich syndrome (WAS) is an X-linked disease characterized by microthrombocytopenia, eczema, immune deficiency, and autoimmune phenomena. Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative treatment. Myeloablative conditioning is the most common regimen used for HSCT in patients with WAS to avoid the risk of mixed donor chimerism and autoimmunity post-HSCT. There is limited data on the use of reduced intensity conditioning for HSCT in patients with WAS. Here, we report a case with severe phenotype of WAS transplanted successfully with reduced intensity conditioning, which is an acceptable conditioning regimen and can be considered in patients with WAS with significantly impaired organ functions...
May 22, 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/28538501/invasive-pneumococcal-disease-in-patients-with-sickle-cell-disease
#17
Pournima Navalkele, Bülent Özgönenel, Eric McGrath, Paul Lephart, Sharada Sarnaik
BACKGROUND: Patients with sickle cell disease (SCD) are at risk of fatal sepsis with encapsulated bacteria, such as Streptococcus pneumoniae, because of the inherent autosplenectomy that occurs in SCD. This risk is thwarted with oral penicillin prophylaxis during the first 5 years of life, and with stringent vaccination against S. pneumoniae alongside routine childhood immunization. But compared with the general African American pediatric population, the rate of invasive pneumococcal disease (IPD) in patients with SCD still remains high, resulting in hospitalization and fatality...
May 22, 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/28538500/parental-perceptions-of-obesity-and-obesity-risk-associated-with-childhood-acute-lymphoblastic-leukemia
#18
Gary L Jones, Wendy McClellan, Sripriya Raman, Ashley Sherman, Erin Guest, Keith August
The prevalence of obesity and related comorbidities in survivors of childhood acute lymphoblastic leukemia (ALL) is well established and ranges anywhere from 29% to 69% depending on the study. We sought to explore the awareness of parents of survivors of childhood ALL regarding the increased risk of obesity and their perceptions regarding the overall health of their child. One hundred twenty-one parents of 99 survivors of pediatric ALL completed surveys regarding perceptions of obesity risk in survivors. Eighty percent of parents of overweight and obese survivors correctly identified their child as "a little overweight" or "overweight...
May 22, 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/28486261/assessing-national-cancer-control-plan-knowledge-prioritization-and-engagement-through-a-pediatric-oncology-cancer-control-workshop
#19
Meaghann S Weaver, Scott C Howard, Lorna Renner, Mhamed Harif, Catherine G Lam
This study represents findings from a questionnaire completed before a cancer control workshop by 47 pediatric cancer control stakeholder participants from 27 countries. Respondents reported their knowledge of their country's cancer control plan, their understanding of childhood cancer control priorities in their local setting, and barriers to high-quality pediatric cancer care. Findings reveal a knowledge gap in pediatric oncology professionals' awareness of national cancer control plan existence, with under-recognition of existing plans, and uncertainty regarding whether pediatric cancer was included in the plans...
May 8, 2017: Journal of Pediatric Hematology/oncology
https://www.readbyqxmd.com/read/28486260/demodicosis-in-pediatric-cancer
#20
Jenneke E van Atteveld, Marlies de Graaf, Martine van Grotel, Marry M van den Heuvel-Eibrink
Demodicosis is a rare condition that most often occurs in immunocompromised patients. We here describe a boy with T-cell non-Hodgkin lymphoma who developed a facial papulopustular eruption just before finalizing T-cell non-Hodgkin lymphoma treatment. He was treated for several infectious diseases without improvement. Demodicosis was considered and complete resolution was finally reached with topical metronidazole. We conducted a systematic search of all previously described cases of this condition in children with cancer, which showed that almost all demodicosis cases occurred in patients with lymphoreticular malignancies during maintenance chemotherapy...
May 8, 2017: Journal of Pediatric Hematology/oncology
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