ABVE-PC and modified BEACOPP regimen in Indian children with Hodgkin lymphoma: Feasibility and efficacy.

Aims: To study the toxicity of ABVE-PC (doxorubicin, bleomycin, vincristine, etoposide, prednisone and cyclophosphamide) and modified-BEACOPP (bleomycin, etoposide, doxorubicin, cyclophosphamide, vincristine, procarbazine, prednisone) in intermediate-risk and high-risk Hodgkin lymphoma patients.

Methods: High-risk patients received 4 cycles of modified-BEACOPP (m-BEACOPP) plus 4 cycles of ABVD. Intermediate-risk patients received 4 cycles of ABVE-PC plus 2 cycles of ABVD.

Low-dose rasburicase in hematologic malignancies.

Objective: To define the efficacy and safety of low-dose rasburicase in children from south India with hematologic malignancies.

Methods: This study is a retrospective analysis of data on 41 children with hematologic malignacies with laboratory evidence of tumor lysis syndrome (TLS) or clinical features indicating high risk for developing TLS. Patients were treated with rasburicase in doses of 0.

Inflammatory myofibroblastic tumor of the liver in children.

Inflammatory pseudotumors, now more aptly termed inflammatory myofibroblastic tumors (IMTs), are uncommon benign neoplasms, which have been reported in most organs and tissues in the body. Originally described and commonly found in the lung, they are also found in the liver of children and adults. We review the literature and analyze the features of the hepatic IMTs reported in children, along with a case report of a 15-month-old boy who had a persistent IMT in the liver and underwent surgical resection for the same following a trial of conservative management.

Surgical placement of totally implantable venous access device-an institutional experience.

Objectives: To investigate the advantages and disadvantages of Totally implantable venous access devices (TIVAD) catheter in pediatric age group and also to review this subject in the recent literature and compare the results with the present results.

Methods: A total of 61 cases, 2 mo to 14 y old, were included in the index study. TIVADs were implanted in these patients for chemotherapy.

Acquired thrombotic thrombocytopenic purpura in children: a single institution experience.

Objective: To describe the clinical features, treatment and prognosis of acquired thrombotic thrombocytopenic purpura (TTP) in children based on a single institution experience.

Methods: This study is a retrospective review of all 12 children with TTP seen at New York Medical College- Westchester Medical Center during a period of 15 y from 1993 to 2008.

Results: There were 7 females and 5 males with acquired TTP, with a median age of 13 (range, 6-17); and no cases of congenital TTP.

Urinary tract infections in pediatric oncology patients with fever and neutropenia.

The relevancy of the urinary tract as a source of infection during febrile neutropenia is not known. The authors sought to determine the frequency of urinary tract infections (UTIs) in pediatric cancer patients with febrile neutropenia. Urine was collected from a mid-stream void before the administration of antibiotics.

Rituximab therapy to prevent relapse in chronic relapsing thrombotic thrombocytopenic purpura (TTP) in a child.

Our patient first developed thrombotic thrombocytopenic purpura (TTP) at age 10 years with an initial platelet count of 10,000/microL. She achieved remission with plasmapheresis (PE), but suffered 2 relapses in the next 2 years, each approximately 1 year from PE, with ADAMTS13 levels of <5%. Early in her third remission, with vincristine (weekly x 4 doses) and prednisone (for 2 weeks) her ADAMTS13 increased to 99% in 24 weeks, but decreased to <4% in the next 38 weeks.

NSC114792, a novel small molecule identified through structure-based computational database screening, selectively inhibits JAK3.

Background: Human or animals lacking either JAK3 or the common gamma chain (gammac) expression display severe combined immunodeficiency disease, indicating the crucial role of JAK3 in T-cell development and the homeostasis of the immune system. JAK3 has also been suggested to contribute to the pathogenesis of tumorigenesis. Recent studies identified activating JAK3 mutations in patients with various hematopoietic malignancies, including acute megakaryoblastic leukemia.

MS-1020 is a novel small molecule that selectively inhibits JAK3 activity.

In order to identify Janus kinase/signal transducer and activator of transcription (JAK/STAT) signalling inhibitors, a cell-based high throughput screening was performed using a plant extract library that identified Nb-(alpha-hydroxynaphthoyl)serotonin called MS-1020 as a novel JAK3 inhibitor. MS-1020 potently inhibited persistently-active STAT3 in a cell type-specific manner. Further examination showed that MS-1020 selectively blocked constitutively-active JAK3 and consistently suppressed interleukin-2-induced JAK3/STAT5 signalling but not prolactin-induced JAK2/STAT5 signalling.

Early recognition of renal toxicity of high-dose methotrexate therapy: a case report.

A 10-year-old boy with osteosarcoma and normal renal function manifested laboratory evidence of impending renal toxicity and extreme elevation of aspartate aminotrasferase and alanine aminotransferase within 2 hours after the completion of a 4-hour infusion of high-dose methotrexate (MTX) (12 g/m2), and went on to develop acute renal failure with life-threatening hyperkalemia 29 hours later. Although his renal function recovered completely with high-dose leucovorin, hemodialysis, charcoal hemoperfusion, and carboxypeptidase G2, we present this case to emphasize that signs of renal toxicity may be present as early as 2 hours after the completion of a 4-hour MTX infusion, and to suggest that monitoring for MTX toxicity should perhaps begin within a few hours after the completion of 4-hour MTX infusion.

A small-molecule compound identified through a cell-based screening inhibits JAK/STAT pathway signaling in human cancer cells.

Inappropriate activation of JAK/STAT signaling occurs with high frequency in human cancers and is associated with cancer cell survival and proliferation. Therefore, the development of pharmacologic STAT signaling inhibitors has therapeutic potential in the treatment of human cancers. Here, we report 2-[(3,5-bis-trifluoromethyl-phenyl)-hydroxy-methyl]-1-(4-nitro-phenylamino)-6-phenyl-1,2,4a,7a-tetrahydro-pyrrolo[3,4-b]-pyridine-5,7-dione (AUH-6-96) as a novel small-molecule inhibitor of JAK/STAT signaling that we initially identified through a cell-based high-throughput screening using cultured Drosophila cells.

Incidence and clinical implications of GATA1 mutations in newborns with Down syndrome.

Somatic mutations in the GATA1 gene are present in almost all cases of Down syndrome (DS)-associated acute megakaryoblastic leukemia (AMKL) and transient leukemia (TL). An in utero origin of the GATA1 mutation suggests it is an early leukemogenic event. To determine the detectable incidence and clinical relevance of GATA1 mutations in DS newborns, we screened Guthrie cards from 590 DS infants for mutations in the GATA1 gene.

Cleft palate, bilateral external auditory canal atresia, and other midline defects associated with Diamond-Blackfan anemia: case report.

Diamond-Blackfan anemia (DBA) is associated with congenital anomalies especially of the midline. When present, facial anomalies are reminiscent of Treacher-Collins syndrome, and both DBA and Treacher-Collins syndrome are disorders of ribosomal biogenesis. Herein, we describe a female infant with multiple midline defects associated with DBA and reaffirm the absence of RPS-19 mutations in DBA patients with facial anomalies.

Recurrent immune thrombocytopenic purpura in children.

Recurrent immune thrombocytopenic purpura (ITP) is defined as the recurrence of ITP after at least 3 months of remission sustained without treatment. Among 340 children with ITP, 14 had recurrent ITP (4.1%).

The safety of central line placement prior to treatment of pediatric acute lymphoblastic leukemia.

Background: Central venous lines are placed in children with acute lymphoblastic leukemia at diagnosis, despite significant cytopenias, to facilitate the administration of chemotherapy and blood sampling. The present study aimed to determine the safety of central line placement in these patients.

Methods: We reviewed the charts of 115 consecutive patients treated during a 10-year period.

GATA1 as a new target to detect minimal residual disease in both transient leukemia and megakaryoblastic leukemia of Down syndrome.

Acquired mutations in exon 2 of the GATA1 gene are detected in most Down syndrome (DS) patients with transient leukemia (TL) and acute megakaryoblastic leukemia (AMKL). We sought to determine if GATA1 mutations can be utilized as markers for minimal residual disease (MRD). GATA1 mutations were screened by SSCP analysis and sequenced.

Eyelid mass as the presenting finding in a child with Down syndrome and acute megakaryoblastic leukemia.

Children with Down syndrome are at increased risk of developing acute megakaryoblastic leukemia (AML). Therefore, physicians caring for children with Down syndrome should be familiar with the presenting features of AML. We describe a child with Down syndrome presenting with a left eyelid mass 1 month before a diagnosis of acute AML.

Detection of central nervous system leukemia in children with acute lymphoblastic leukemia by real-time polymerase chain reaction.

Accurate detection of central nervous system (CNS) involvement in children with newly diagnosed acute lymphoblastic leukemia (ALL) could have profound prognostic and therapeutic implications. We examined various cerebrospinal fluid (CSF) preservation methods to yield adequate DNA stability for polymerase chain reaction (PCR) analysis and developed a quantitative real-time PCR assay to detect occult CNS leukemia. Sixty CSF specimens were maintained in several storage conditions for varying amounts of time, and we found that preserving CSF in 1:1 serum-free RPMI tissue culture medium offers the best stability of DNA for PCR analysis.

A pilot trial of tandem autologous peripheral blood progenitor cell transplantation following high-dose thiotepa and carboplatin in children with poor-risk central nervous system tumors.

This is a pilot study performed to determine the maximum tolerated number of courses of high-dose thiotepa and carboplatin with autologous peripheral blood progenitor cell (PBPC) transplantation in poor-risk pediatric central nervous system (CNS) tumor patients. Twelve patients were enrolled and a total of 24 PBPC transplants were performed. The median age was 7.

Long-term outcome of chronic idiopathic thrombocytopenic purpura in children.

Objectives: Children with chronic idiopathic thrombocytopenic purpura (ITP) generally have a favorable outcome, but it is not known whether there are any prognostic factors to predict outcome. The objectives of this study were to assess the spontaneous remission rate and the prognostic significance of age, gender, initial platelet count, initial treatment, and response to treatment.

Methods: In this retrospective review of 62 consecutive children with chronic ITP, 37 were girls and 27 were 10 years of age or older (median age 9 years; range, 0.

Trends in diagnosis and management of iron deficiency during infancy and early childhood.

Iron deficiency remains a public health challenge. The neurocognitive deficits that are associated with iron deficiency are most worrisome. Moreover, iron deficiency-associated elevated blood lead levels magnify the problem.