Successful Liver Transplantation for Adolescent Patient With Pyruvate Kinase Deficiency-induced Cirrhosis
We present the case of a successful liver transplant in a young adult patient with cholestasis and cirrhosis secondary to severe pyruvate kinase (PK) deficiency. Liver transplant resulted in resolution of liver dysfunction, decreased need for blood transfusions and eligibility for bone marrow transplantation. This case represents the third reported patient in the literature with severe PK deficiency who successfully underwent liver transplant as a result of profound cholestasis and liver failure. Explant pathology demonstrated a lack of significant iron deposition indicating that PK deficiency predisposes the liver to inju...
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research

Dysfunctional Immune System Reconstitution After Rituximab Exposure In Utero
Rituximab is an antibody that binds to B-lymphocytes and is increasingly used during pregnancy. As an immunoglobulin G, it will transfer across the placenta. Previous case reports describe a diversity of clinical presentations in neonates born following rituximab exposure in utero. Our case is the first to offer the long-term experience in the care of an infant with severe neutropenia and prolonged profound hypogammaglobulinemia and class-switching B cell defect after in utero rituximab exposure. (Source: Journal of Pediatric Hematology Oncology)
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research

Evaluation of Inflammatory Biomarkers in Pediatric Hematology-Oncology Patients With Bloodstream Infection
Bloodstream infection (BSI) is a serious complication in pediatric hematology-oncology patients. To evaluate the clinical significance of C-reactive protein (CRP), procalcitonin (PCT), albumin, fibrinogen, and D-dimer as potential biomarkers to differentiate among various subtypes of BSIs in pediatric patients with hematologic and oncologic diseases, we retrieved and analyzed the medical records of pediatric hematology-oncology patients diagnosed with BSI at our hospital between January 2016 and December 2017. The demographic (sex and age) and clinical (primary diseases) characteristics, and laboratory test results (white ...
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research

A Novel PUS1 Mutation in 2 Siblings with MLASA Syndrome: A Review of the Literature
Conclusion: This report adds to the growing number of mutations causing complex clinical manifestations of MLASA including lactic acidosis, sideroblastic anemia, chronic diarrhea, and myopathy. (Source: Journal of Pediatric Hematology Oncology)
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research

Successful Treatment With Bortezomib for Refractory and Complicated Acquired Thrombotic Thrombocytopenic Purpura in an Adolescent Girl
Thrombotic thrombocytopenic purpura (TTP) is a rare, dangerous, life-threatening disease characterized by microangiopathic hemolytic anemia and thrombocytopenia, along with organ dysfunction due to microangiopathy-related ischemia. Plasma exchange and steroids are used for initial treatment, and rituximab is often used in refractive patients. Caplacizumab, cyclophosphamide, and splenectomy are among other treatment options. It has been reported that bortezomib, a proteasome inhibitor, can be used in the management of refractory acquired TTP. Herein, we present a 16-year-old female patient who was monitored for acquired TTP...
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research

Potential Risk Factors Associated With Graft Failure of Haploidentical Hematopoietic Stem Cell Transplantation in Children With Sickle Cell Disease
Nonmyeloablative (NMA) haploidentical hematopoietic stem cell transplantation for sickle cell disease has significantly increased donor availability for transplant and is increasingly used as curative treatment. The authors describe 3 pediatric patients who rejected grafts after an NMA regimen, previously reported to result in good engraftment rates in the mainly adult population. In this manuscript, potential factors contributing to rejection are described and discussed. The authors emphasize the need to further optimize the NMA regimens in pediatric patients and perform haploidentical transplants for sickle cell disease ...
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research

Recurrent Intracranial Bleed in 3 Siblings: Short of a Shot of Vitamin K!
We present a family who suffered recurrent sibling losses due to vitamin K deficiency bleed. The index child was asymptomatic at presentation, had normal clinical examination, and was investigated for coagulation disorders in view of previous 3 sibling losses as a result of intracranial hemorrhage. His investigations showed deranged coagulogram and clotting factors’ assay. The baby was given vitamin K1 1 mg intramuscularly following which his coagulogram and clotting factors’ assay returned to normal. The genetic analysis did not identify any inherited cause of bleeding tendency. The significant family h...
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research

Hereditary Thrombotic Thrombocytopenic Purpura in a 9-Month-old: Diagnosing and Managing an Ultra-rare Disorder
We present the diagnostic evaluation of a patient with hereditary thrombotic thrombocytopenic purpura. Genetic testing revealed one known pathogenic mutation and one novel mutation of ADAMTS13 classified as likely pathogenic on the basis of parental genetic testing and in silico analyses. We further discuss off-label use of prophylactic plasma-derived Factor VIII (Koate-DVI) and the benefit of rare disease registries. (Source: Journal of Pediatric Hematology Oncology)
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research

Neuroimaging and Pathology Findings Associated With Rapid Onset Obesity, Hypothalamic Dysfunction, Hypoventilation, and Autonomic Dysregulation (ROHHAD) Syndrome
We present the case of a 36-month-old boy with the classic symptoms of ROHHAD and a neuroendocrine tumor, who progressed rapidly and subsequently succumbed to cardiorespiratory arrest because of hypoventilation. His magnetic resonance imaging findings at the initial diagnosis and the brain autopsy results are detailed. The literature was reviewed to summarize the current understanding of the underlying mechanism of this rare disorder. (Source: Journal of Pediatric Hematology Oncology)
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research

Report of 2 Pediatric Cases With Li-Fraumeni Syndrome Related Malignancy in a Family
We report 2 pediatric cases with LFS-related malignancy in a family. Eight-year-old elder brother was diagnosed with adrenocortical carcinoma and was found to have a heterozygous missense germline mutation c.736A>G: p.Met246Val in the TP53 gene. Cancer screening led to the diagnosis of rhabdomyosarcoma at a curable stage in his 2-year-old younger brother. Comprehensive surveillance resulted in early tumor detection and improved survival. (Source: Journal of Pediatric Hematology Oncology)
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research

Asymptomatic Right Atrial Thrombosis After Acute Lymphoblastic Leukemia Treatment
Right atrial thrombosis is a rare, but potentially serious complication of acute lymphoblastic leukemia treatment. We conducted a retrospective multicenter study to assess the incidence, treatment, and outcome of asymptomatic right atrial thrombosis detected at routine echocardiography of children after acute lymphoblastic leukemia treatment in the Nordic and Baltic countries. Eleven (2.7%, 95% confidence interval, 1.4-4.9) of 406 patients had asymptomatic right atrial thrombosis, ranging from 10 to 25 mm at detection. Three patients were treated with anticoagulation. None of the thromboses affected cardiac function...
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research

Bleeder With a Clot: Thrombosis Following Treatment of Bleeding in a Child With Severe Hemophilia B
A 16-year-old boy with severe hemophilia B and minimal bleeding manifestations in his early childhood presented with gastrointestinal bleeding at 11 years of age. Following administration of prothrombin complex concentrate, he developed peripheral venous thrombosis and cerebral sinovenous thrombosis, posing a management dilemma. His cerebral sinovenous thrombosis resolved spontaneously, proving watchful waiting to be a useful strategy. He developed spontaneous intracranial bleed at 14 years of age for which he was treated with factor IX concentrate and commenced on prophylaxis. We discuss the factors contributing to genoty...
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research

Type 1 Plasminogen Deficiency With Pulmonary Involvement: Novel Treatment and Novel Mutation
Type 1 plasminogen deficiency is a rare genetic disorder. Type 1 plasminogen deficiency is characterized by fibrin-rich pseudomembrane formation on mucosal surfaces, particularly the conjunctiva. Tracheobronchial tree involvement is a less common reported manifestation of type 1 plasminogen deficiency. Pseudomembranes in the tracheobronchial tree may result in respiratory compromise and ultimately fail if not recognized and treated. Currently, there is no specific replacement therapy approved for the treatment of congenital plasminogen deficiency. In the present paper, we report that type 1 plasminogen deficiency with nove...
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research

Catheter-directed Thrombolysis for Neonatal IVC and Bilateral Renal Vein Thrombosis: A Case Report
This report presents the case of a full-term neonate presenting with bilateral renal vein thrombosis with inferior vena cava involvement treated with catheter-directed thrombolysis. This case report intends to highlight the value of a multidisciplinary approach to pediatric venous thromboembolism and to outline relevant procedural details and current laboratory and imaging monitoring of catheter-directed thrombolysis. (Source: Journal of Pediatric Hematology Oncology)
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research

Trametinib-associated Hyponatremia in a Child With Low-grade Glioma is Not Seen Following Treatment With Alternative MEK Inhibitor
This report describes severe hyponatremia associated with trametinib in an infant with progressive low-grade glioma without underlying endocrine dysfunction, which recurred despite significant dose reduction. Therapy with an alternative MEK inhibitor, binimetinib, provided excellent tumor response without hyponatremia, suggesting that some toxicities may be avoided by changing MEK inhibitor agents within the same class. (Source: Journal of Pediatric Hematology Oncology)
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research

Novel CUBN Mutation in a Young Child With Megaloblastic Anemia
In this report, we describe a novel CUBN pathogenic variant in a child with megaloblastic anemia. (Source: Journal of Pediatric Hematology Oncology)
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research

Pediatric Paroxysmal Nocturnal Hemoglobinuria Presenting as Acute Kidney Injury
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease characterized by variable and diverse symptoms including the classic triad of hemolytic anemia, thrombosis, and bone marrow failure. It is a disorder primarily seen in the adult population. The authors report a unique case of an 8-year-old girl diagnosed with PNH after initially presenting with a febrile illness and acute kidney injury. Though rare in children, PNH should remain in the differential diagnosis of a child presenting with acute kidney injury. The disease has serious long-term complications, mandating timely diagnosis and appropriate therapy. (Source: ...
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research

Diamond-Blackfan Anemia: 2 Cases With a Twist
Conclusions: A thorough evaluation of all family members is imperative to identify possible ‘silent carriers’ who are those with no physical stigmata and minor or absent hematologic manifestations. New mutations could add in the map of the disease. (Source: Journal of Pediatric Hematology Oncology)
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research

Coincidental Expression of Classic Hodgkin Lymphoma and Neurofibromatosis Type I and Literature Review
We report an adolescent with NF1 who developed classic Hodgkin lymphoma. Although there is an unclear association between mutations in the NF1 gene and classic Hodgkin lymphoma, further studies are warranted. (Source: Journal of Pediatric Hematology Oncology)
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research

Second Osteosarcoma in a 16-Year-old Woman Diagnosed With Rothmund-Thomson Syndrome
We describe our patient treatment and recommend a possible screening-surveillance for RTS type II patients. (Source: Journal of Pediatric Hematology Oncology)
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research

Malignant Granular Cell Tumor of the Lower Extremity in an Adolescent Male
We present a case of a 14-year-old male who presented with a large mass in his left lower extremity. After being biopsied, the mass was diagnosed as a malignant GCT. The tumor was completely excised with wide margins and close follow-up with the patient continued. (Source: Journal of Pediatric Hematology Oncology)
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research

Retroperitoneal Sclerosing Lipogranuloma in an Adolescent With Congenital Atresia of the Inferior Vena Cava: Case Report and Literature Review
Sclerosing lipogranuloma (SLG) in children is a rare, benign disease of unknown etiology suspected to be due to abnormal fatty tissue reaction. A 13-year-old girl presented with progressively worsening back pain. Cross-sectional imaging identified a retroperitoneal mass compressing the left ureter as well as infrarenal inferior vena cava atresia with extensive venous collaterals and chronic partially occlusive thromboses of the iliac veins. Surgical biopsy was consistent with SLG and it resolved spontaneously. SLG is typically a disease of adulthood but may be seen in children. The association between inferior vena cava at...
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research

Anaplastic Lymphoma Kinase Inhibitor in Treatment of Inflammatory Myofibroblastic Tumor of the Tongue in Infancy
We describe a case of IMT of the tongue in a neonate treated with debulking and an anaplastic lymphoma kinase inhibitor. The patient achieved complete response and remains disease-free 1.5 year following completion of therapy. (Source: Journal of Pediatric Hematology Oncology)
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research

Pediatric Myelodysplastic Syndrome With Germline RRAS Mutation: Expanding the Phenotype of RASopathies
The RAS/mitogen-activated protein kinase pathway plays a significant role in cell cycle regulation. Germline mutation of this pathway leads to overlapping genetic disorders, RASopathies, and is also an important component of tumorigenesis. Here we describe a rare case of myelodysplastic syndrome with monosomy 7 in a pediatric patient with a germline RRAS mutation. RRAS mutations have been implicated in the development of juvenile myelomonocytic leukemia, but our case suggests RRAS mutations display a broader malignant potential. Our case supports the recommendation that genetic testing should include RRAS in suspected RASo...
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research

WNT-activated Pediatric Medulloblastoma Associated With Metastasis to the Suprasellar Region and Hypopituitarism
We report on a rare association of WNT-activated medulloblastoma with metastasis to the suprasellar region. Medulloblastoma is the commonest brain tumor in children, and the most common pattern of metastatic disease is that of leptomeningeal involvement and spinal metastasis. Historically, medulloblastoma patients were categorized into different risk groups on the basis of age, histology, size of residium after surgery, and metastatic status, but the discovery of at least 4 molecular subgroups has changed the way these tumors are now treated. We report a 6-year-old patient who had a rare association of WNT-activated medull...
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research

Partial Response to Sorafenib in a Child With a Myeloid/Lymphoid Neoplasm, Eosinophilia, and a ZMYM2-FLT3 Fusion
Dysregulated tyrosine kinases in myeloid/lymphoid neoplasms with eosinophilia are rare, but do occur in children. To increase awareness of this diagnosis, we present a child who was diagnosed after a 3-year disease history. The patient was initially treated according to a T-cell lymphoblastic lymphoma protocol, but genetic analyses at recurrence revealed microdeletions resulting in an in-frame fusion of ZMYM2 and FLT3. Treatment with sorafenib, an FLT3 tyrosine kinase inhibitor, rapidly resulted in significant reduction of lymphadenopathy and normalization of white blood cell and eosinophil counts. At 17 months of treatmen...
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research

Intracranial Growing Teratoma Syndrome With Intraventricular Lipid Accumulation
Growing teratoma syndrome is a well-recognized condition associated with both intracranial and extracranial nongerminomatous germ cell tumors (NGGCTs), which mostly manifest as rapid growth of cystic and solid components during or within several months after treatment. Here, we report a patient with NGGCT who experienced slow growth of intracranial growing teratoma syndrome with intraventricular lipid accumulation over 10 years without any clinical symptoms. Considering the clinicopathologic heterogeneity of this syndrome, long-term clinical and radiologic follow-up is required for all patients with intracranial NGGCT. (So...
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research

Precursor B-Cell Acute Lymphoblastic Leukemia With MYC and BCL2 Rearrangements Presenting as Extensive Extranodal Disease in an Adolescent
Combined rearrangements of MYC and BCL2 are rare in precursor B-cell acute lymphoblastic leukemia (B-ALL). A 14-year-old boy presented with swelling of the knee and face. Imaging revealed diffuse infiltration of lacrimal glands, parotid glands along with the extensive epidural disease. Morphology and immunophenotype of knee joint aspirate were consistent with precursor B-ALL. Fluorescent in situ hybridization identified rearrangements of MYC and BCL2 genes. The disease was refractory to intensive treatment. The patient died of progressive disease. Precursor B-ALL with combined MYC and BCL2 rearrangements is rare, character...
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research

Successful Rituximab Monotherapy in Advanced-stage, Epstein-Barr Virus-positive Diffuse Large B-Cell Lymphoma in an Adolescent With Systemic Juvenile Idiopathic Arthritis
We report an 18-year-old female individual with refractory systemic juvenile idiopathic arthritis, treated with multiple immunosuppressive agents, who was diagnosed with stage III, EBV+ DLBCL. The patient achieved sustained complete remission after 4 weekly doses of rituximab monotherapy and reduction of immunosuppression. This case suggests that a post-transplant lymphoproliferative disease-like treatment approach can be a safe and effective therapy in a nontransplant, yet severely immunosuppressed, patient with EBV+ DLBCL. (Source: Journal of Pediatric Hematology Oncology)
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research

Hyperdiploid Precursor B-acute Lymphoblastic Leukemia Presenting as a Cavernous Sinus Mass in a 4-Year-old Male
We report the case of a 4-year-old male with a lymphomatous cavernous sinus mass, a previously undescribed presentation of newly diagnosed hyperdiploid B-ALL. Few case reports in the literature describe lymphomatous involvement in this region, but none are associated with pediatric B-ALL. This case presented unique treatment and risk assignment challenges given the intracranial location of this tumor and proximity to the central nervous system. (Source: Journal of Pediatric Hematology Oncology)
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research

Association of Anemia and Blood Pressure With Novel Markers of Diastolic Function in Pediatric Sickle Cell Disease
Diastolic dysfunction is a known cause of mortality in adults with sickle cell disease (SCD). Left atrial function (LAf) and strain (LAS) are novel echocardiographic parameters to assess early diastolic dysfunction, which have not been assessed in pediatric SCD. Through a retrospective single-center study, we describe echocardiographic parameters of diastology in children with SCD and evaluate their relationship with clinical variables including anemia and blood pressure. Baseline clinical data, 24-hour ambulatory blood pressure monitoring data and echocardiography results were collected. LAf and LAS were measured using vo...
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Original Articles Source Type: research

An Evaluation of a Fluorescence In Situ Hybridization Strategy Using Air-dried Blood and Bone-marrow Smears in the Risk Stratification of Pediatric B-Lineage Acute Lymphoblastic Leukemia in Resource-limited Settings
Cytogenetic abnormalities (CAs), one of the strongest influencers of therapeutic outcome in pediatric B-cell precursor acute lymphoblastic leukemia (BCP-ALL), can be identified by different techniques. Despite several technological advances, many centers with resource-limited settings continue to use either reverse-transcriptase polymerase chain reaction (RT-PCR) and/or fluorescence in situ hybridization (FISH) to identify prognostically relevant CAs. We evaluated a simple and cost-effective triple-probe FISH strategy on air-dried blood and bone-marrow smears and compared its performance with a multiplex RT-PCR-based appro...
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Original Articles Source Type: research

A Case of Ewing Sarcoma of the Bladder Presenting in Early Infancy
A 6-week-old female presented with gross hematuria and was diagnosed with Ewing sarcoma of the bladder through ultrasound and cystoscopic biopsies, along with a negative metastatic workup. She was treated with transurethral resection, chemotherapy consisting of with vincristine, cycolphosphamide, doxorubicin, ifosfamide and etoposide, and partial cystectomy. After completing chemotherapy, the patient has been doing well with no evidence of disease. There have been 14 other cases, 4 pediatric, of Ewing sarcoma of the bladder reported. To our knowledge, our case is the youngest patient reported with this disease. (Source: Jo...
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Original Articles Source Type: research

Increased Malignancy Rate in Children With IgE Deficiency: A Single-center Experience
Background: Immunoglobulin (Ig) E-deficient adults (IgE (Source: Journal of Pediatric Hematology Oncology)
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Original Articles Source Type: research

IAP Chemotherapy Regimen Is a Viable and Cost-effective Option in Children and Adolescents With Osteosarcoma: A Comparative Analysis With MAP Regimen on Toxicity and Survival
Conclusions: OS and EFS with both regimens were similar. However, the MAP regimen was associated with a statistically significant increase in incidence of supportive care admissions, delay in next cycle of chemotherapy, and predicted higher cost of treatment. (Source: Journal of Pediatric Hematology Oncology)
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Original Articles Source Type: research

Posterior Reversible Encephalopathy Syndrome in Childhood Hematological/Oncological Diseases: Multicenter Results
In conclusion, PRES may develop during the follow-up and treatment of hematological diseases. In addition to steroid and intense combined chemotherapies, immunosuppressive agents and hypertension are also factors that may be responsible for PRES. (Source: Journal of Pediatric Hematology Oncology)
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Original Articles Source Type: research

Decrease of Postchemotherapy Complications With the Use of Probiotics in Children With Acute Lymphoblastic Leukemia
Conclusions: The results indicate that the use of probiotics can be a great alternative in the improvement of gastrointestinal symptoms and the adverse effects associated with chemotherapy. (Source: Journal of Pediatric Hematology Oncology)
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Online Articles: Original Articles Source Type: research

Towards Optimizing Risk-adapted Treatment of APML in ATRA/ATO Era: How Can Prediction of Early Mortality Help?
No abstract available (Source: Journal of Pediatric Hematology Oncology)
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Letters to the Editor Source Type: research

Abnormally Hypersegmented Neutrophilia in Pediatric Acute Myeloid Leukemia Associated With t(2;11)(q31;p15) and NUP98 Rearrangement
No abstract available (Source: Journal of Pediatric Hematology Oncology)
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Letters to the Editor Source Type: research

Haploidentical Stem Cell Transplant With Post Transplant Cyclophosphamide for Chronic Granulomatous Disease With Thiotepa, Busulfan, and Fludarabine as Conditioning
No abstract available (Source: Journal of Pediatric Hematology Oncology)
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Letters to the Editor Source Type: research

Use of CD19-directed CAR T-Cell Therapy in an Infant With Refractory Acute Lymphoblastic Leukemia
Infants with KMT2A-rearranged acute lymphoblastic leukemia (ALL) have historically poor outcomes despite maximal intensification of chemotherapy. Chimeric antigen receptor (CAR) T-cell therapy has revolutionized our approach to pediatric patients with relapsed/refractory ALL. Unfortunately, infants were excluded from early CAR T-cell trials due to concerns regarding the feasibility of T-cell collection and expansion. Here, we report the use of tisagenlecleucel in an infant with chemotherapy-refractory KMT2A-rearranged ALL. While CAR T-cell therapy was not curative for this patient, collection and expansion of T-cells prove...
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Clinical and Laboratory Observations Source Type: research

Pediatric Extraskeletal Ewing Sarcoma Originating in the Heart: A Case Report and Review of the Literature
We report a case of extraosseous Ewing sarcoma of primary cardiac origin in a 9-year-old girl, treated with debulking surgery, adjuvant chemotherapy, and radiotherapy. (Source: Journal of Pediatric Hematology Oncology)
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Clinical and Laboratory Observations Source Type: research

Primary Central Nervous System Posttransplant Lymphoproliferative Disorder After Kidney Transplant
We report a case of primary central nervous system posttransplant lymphoproliferative disorder presenting as a single rim enhancing lesion with central restricted diffusion mimicking an intracranial abscess. (Source: Journal of Pediatric Hematology Oncology)
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Radiology Corner Source Type: research

Vision Outcomes for Pediatric Patients With Optic Pathway Gliomas Associated With Neurofibromatosis Type I: A Systematic Review of the Clinical Evidence
Children with neurofibromatosis type I (NF1) have a higher predisposition for low-grade astrocytomas of the optic pathway, commonly referred to as optic pathway gliomas (OPGs). OPGs can result in visual deterioration. Treatment outcomes in OPG-NF1 management are often reported around tumor stabilization. We sought to compare vision outcomes associated with different OPG treatment strategies to inform about this important functional metric. A meta-analysis exploring the different modalities to treat children with OPG-NF1 was conducted following Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guid...
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Review Articles Source Type: research

Patient-reported Outcome Measures in Pediatric Non-Malignant Hematology: A Systematic Review
The objective of this systematic review is to identify and list which validated PROMs have been used to monitor health-related quality of life in pediatric patients with nonmalignant hematology (hemophilia, immune thrombocytopenia, sickle cell disease, and thalassemia). Databases (MEDLINE, Embase, HaPI, CINAHL, and PsycTESTS) were searched to identify publications that validated or used PROMs as an outcome measure in the 4 disease groups. Overall, 209 articles met the inclusion criteria, identifying 113 PROMs. Of the 113 identified PROMs, 95 are generic and can be used in multiple disease groups. The Pediatric Quality of L...
Source: Journal of Pediatric Hematology Oncology - April 23, 2021 Category: Hematology Tags: Review Articles Source Type: research

Refractory and Fatal Presentation of Severe Autoimmune Hemolytic Anemia in a Child With the DNASE1L3 Mutation Complicated With an Additional DOCK8 Variant
We reported a case with a loss-of-function mutation in DNASE1L3, a gene described previously in families with systemic lupus erythematosus. In addition, the patient showed a novel homozygous missense variant in DOCK8, a gene known to be responsible for the hyper-IgE recurrent infection syndrome (HIES). A 3-year-old girl born to consanguine parents presented with chronic urticarial rash, hemolytic anemia, pulmonary hemorrhage, and hypovolemic shock findings. She had a low hemoglobin level, a positive direct antiglobulin test, antinuclear antibody and anti-double stranded DNA, low C3 and C4, third-degree tricuspid regurgitat...
Source: Journal of Pediatric Hematology Oncology - March 31, 2021 Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research

Use of Infliximab in the Treatment of Macrophage Activation Syndrome Complicating Kawasaki Disease
We present 2 cases of KD complicated with macrophage activation syndrome, including 1 patient with DiGeorge syndrome successfully treated with a combined treatment of IV gammaglobulin, corticosteroids, cyclosporine, and infliximab. (Source: Journal of Pediatric Hematology Oncology)
Source: Journal of Pediatric Hematology Oncology - March 31, 2021 Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research

A Chinese Boy With Lupus Anticoagulant-hypoprothrombinemia Syndrome: A Case Report and Review of the Literature
We herein report an unusual case of lupus with bleeding diathesis in a Chinese adolescent boy. In the presence of lupus anticoagulant and hypoprothrombinemia, the diagnosis of lupus anticoagulant-hypoprothrombinemia syndrome was made. He responded promptly to immunosuppressive agents and achieved disease remission. (Source: Journal of Pediatric Hematology Oncology)
Source: Journal of Pediatric Hematology Oncology - March 31, 2021 Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research

Intracerebral Nontraumatic Hemorrhagic Stroke in Children: Case Series and Literature Review
We described a case series of pediatric patients with intracerebral nontraumatic hemorrhagic stroke from different etiologies. Although increasingly recognized, such situations are still poorly described in children and our report offers a good overview on this topic. (Source: Journal of Pediatric Hematology Oncology)
Source: Journal of Pediatric Hematology Oncology - March 31, 2021 Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research

Line Associated Thrombosis in Pediatric Patients With NF-κB Pathway Variants
Our report explores the complex role that nuclear factor-kappa B (NF-κB) plays in thrombosis formation, inflammation, and immunity; while additionally demonstrating that patients with NF-κB pathway pathogenic variants appear to carry a substantial thrombotic risk, particularly when secondary thrombotic risk factors are present. We propose that prophylactic anticoagulation should be strongly considered in such patients during high-risk situations and provide additional hematologic management strategies for those with NF-κB pathway alterations. We hope our work also calls to attention the potential need for...
Source: Journal of Pediatric Hematology Oncology - March 31, 2021 Category: Hematology Tags: Online Articles: Clinical and Laboratory Observations Source Type: research