Correlation of mucosal healing endpoints with long-term clinical and patient-reported outcomes in ulcerative colitis.

Background: We evaluated the clinical relevance of achieving histologic endoscopic mucosal improvement (HEMI) and the more stringent target of histologic endoscopic mucosal remission (HEMR) in the phase 3 maintenance trial of upadacitinib for moderately to severely active ulcerative colitis.

Methods: Clinical and patient-reported outcomes were assessed in patients with clinical response after 8- or 16-week upadacitinib induction who received 52-week upadacitinib maintenance treatment. Cross-sectional and predictive analyses evaluated the relationship between HEMR or HEMI at Week 8/16 and Week 52, respectively, and outcomes at Week 52.

Development and testing of an alternative responder definition for EULAR Sjögren's Syndrome Patient Reported Index (ESSPRI).

Objectives: Dryness, fatigue and joint/muscle pain are typically assessed in Sjögren's trials using European Alliance of Associations for Rheumatology Sjögren's Syndrome Patient Reported Index (ESSPRI). A Patient Acceptable Symptom State of <5 and a Minimal Clinically Important Improvement (MCII)/responder definition (RD) of ≥1 point or 15% on ESSPRI have previously been defined. This study explored alternative RDs to better discriminate between active treatment and placebo in trials.

Relationship between COMLEX-USA scores and performance on the American Osteopathic Board of Emergency Medicine Part I certifying examination.

Context: Few studies have investigated how well scores from the Comprehensive Osteopathic Medical Licensing Examination-USA (COMLEX-USA) series predict resident outcomes, such as performance on board certification examinations.

Objectives: To determine how well COMLEX-USA predicts performance on the American Osteopathic Board of Emergency Medicine (AOBEM) Part I certification examination.

Methods: The target study population was first-time examinees who took AOBEM Part I in 2011 and 2012 with matched performances on COMLEX-USA Level 1, Level 2-Cognitive Evaluation (CE), and Level 3.

Phase II study of high-dose chemotherapy before radiation in children with newly diagnosed high-grade astrocytoma: final analysis of Children's Cancer Group Study 9933.

Background: High-grade astrocytomas (HGA) carry a dismal prognosis and compose nearly 20% of all childhood brain tumors. The role of high-dose chemotherapy (HDCT) in the treatment of HGA remains unclear.

Methods: In a nationwide study, The Children's Cancer Group (CCG) prospectively evaluated 102 children with HGA and postoperative residual disease for efficacy and toxicity of four courses of HDCT before radiotherapy (RT).

Phase II evaluation of interferon-alpha-2a for progressive or recurrent craniopharyngiomas.

Object: Craniopharyngiomas originate from the same cells as squamous cell skin carcinoma, which can be treated successfully with interferon-alpha (IFNalpha)-2a. The authors evaluated the activity and toxicity of systemic IFN in young patients with craniopharyngiomas.

Methods: Fifteen patients between the ages of 4.

Health status in 52 long-term survivors of pediatric brain tumors.

The percentage of children who survive childhood brain tumors is increasing. A number have neurological and other sequelae which impact on the quality of their survival. We reviewed long-term survivors using a standardized health status instrument.

Reverse transformation and genome exposure in the C6 glial tumor cell line.

Reexpression of growth control and differentiation in response to physiological inducers can be demonstrated in some malignant cell lines, showing that they are not irreversibly transformed. This switch in phenotype is likely to reflect a changing pattern of gene expression, but it has not been known whether such cellular transitions involve major or only minor modulation of chromatin structure. We have studied growth control and accessibility of chromatin to DNase I in C6 glioma cells subjected to different growth regimens using an in situ nick translation assay to label the most exposed regions of nuclear chromatin.

Pediatric leukemia/lymphoma with t(8;14)(q24;q11).

A variety of chromosomal translocations occur in pediatric T-cell acute lymphoblastic leukemia (T-ALL) in which a cellular oncogene or growth-related gene is translocated to the alpha/delta locus of the T-cell receptor gene. The t(8;14)(q24;q11) has been described at the cytogenetic and molecular level, but the disease associated with this translocation has not been defined clinically. Fifteen pediatric cases of leukemia/lymphoma with a t(8;14)(q24;q11) chromosomal translocation were collected from previous publications and institutional records.

Choroid plexus carcinoma--responses to chemotherapy alone in newly diagnosed young children.

Choroid plexus carcinoma (CPC) arising in the infant poses several treatment dilemmas. The tumor is often not totally resectable at presentation given its large size and tendency to invade adjacent brain. Because of its predisposition to regrow and metastasize, some form of postoperative cytotoxic therapy is required.

Childhood cancer stereotype: impact on adult perceptions of children.

Explored the possibility that biased expectations might affect how adults respond to children diagnosed as in remission from cancer. The presence of a childhood cancer stereotype was investigated experimentally by assessing reactions of undergraduates and medical students to children described with either an in remission from leukemia label (RLL) or healthy label (HL). RLL children were rated as less sociable, less cognitively competent, less behaviorally active and well behaved, less physically potent, littler, and less likely to adjust well to the future than HL children.

The nature of the defect in cobalamin G mutation.

Cobalamin G mutation (cblG) typically presents as a severe megaloblastic anemia during the first few weeks of life. The anemia responds completely to treatment with high doses of Cbl but the neurologic manifestations respond more slowly and not always completely. Cultured fibroblasts from two affected infants and virus-transformed lymphoblasts from one of the infants expressed the following: poor growth in the absence of methionine, the ability to take up and internalize Cbl bound to transcobalamin II, impaired synthesis of methionine from homocysteine, the ability to bind incoming Cbl to the respective coenzymes, but an inability to synthesize methylcobalamin in spite of a normal capacity to synthesize adenosylcobalamin.

Bone marrow transplantation for acute leukemia and Down syndrome: report of a successful case and results of a national survey.

We report a child with Down syndrome (DS) and acute lymphoblastic leukemia who is a healthy survivor 38 months after bone marrow transplantation (BMT). Psychometric evaluations performed before and after BMT indicate no demonstrable therapy-related change in intellectual function. A survey of BMT centers in the United States indicated that 16 leukemic DS children have had transplants at 10 BMT centers.

Improved survival following bone marrow transplantation for aplastic anaemia.

We transplanted 46 patients with severe aplastic anaemia with a new pretransplant immunosuppressive regimen consisting of cyclophosphamide (200 mg/kg) and low-dose total body irradiation (3 Gy). This regimen (CY-TBI-2) was designed to decrease the high risk of graft rejection associated with the use of cyclophosphamide alone, without increasing the incidence of graft-versus-host disease (GHVD) or interstitial pneumonia (IPn). Two-year actuarial disease-free survival of patients conditioned with CY-TBI-2 was 62% (95% CI: 47-77%).

Allogeneic bone marrow transplantation for chronic myelogenous leukemia in chronic or accelerated phase.

Eight patients with Ph1-positive chronic myelogenous leukemia (CML) in chronic or accelerated phase received high-dose cyclophosphamide, total body irradiation, and bone marrow transplantation from an HLA-identical sibling donor. All patients had prompt engraftment and achieved complete hematologic remission. Six patients remain alive and in continuous remission with a normal bone marrow karyotype 3-20+ mo posttransplant.

Prevention of graft rejection following bone marrow transplantation.

Bone marrow transplantation from an HLA-identical sibling is increasingly used in the treatment of severe aplastic anemia. One major problem with this approach is graft rejection that occurs in 25%-60% of patients conditioned for transplantation with cyclophosphamide. At most transplant centers it has been difficult to accurately identify patients at high risk for graft rejection.

Volumetric and functional heterogeneity of human monocytes.

Volume analysis of purified human blood monocytes revealed distinct populations of large and small cells. Computer curve fitting suggested a third, intermediate-sized population. These monocytes were designated M1, M2, and M3 in order of increasing size, and their approximate volumes were 150, 250, and 480 micron3, respectively.

Monocyte subsets in neonates and children.

Volumetrically distinct subpopulations of peripheral blood monocytes, termed M1, M2, and M3, were identified in healthy normal adults and children. Because normal neonates have abnormal monocyte chemotaxis, it was determined whether monocyte subpopulations have different chemotactic capabilities and, if so, whether chemotactically active subpopulations were quantitatively deficient in neonates. Chemotaxis tests with zymosan-activated normal human serum as the chemoattractant and purified monocyte subpopulations revealed that large M3 monocytes were capable of significantly more directed migration than were small M1 and M2 monocytes.

Neuroblastoma in father and son.

A growing literature supports the concept that some cases of neuroblastoma are hereditary. To this we add the first known case, to our knowledge, of neuroblastoma in a parent and child. Various factors, such as the remarkable tendency for this tumor to regress spontaneously, as well as its frequent fatal outcome, have reduced the number of observed familial cases.

Preliminary report: treatment of the hemolytic-uremic syndrome with aspirin and dipyridamole.

Three children with the hemolytic-uremic syndrome were treated with heparin, aspirin, and dipyridamole. Two of the children had remained profoundly thrombocytopenic in spite of platelet transfusion and heparin therapy. All three patients responded with prompt elevation of their platelet counts and apparent termination of the pathologic consumption of platelets.