Granulocyte Transfusions in Patients with Chronic Granulomatous Disease Undergoing Hematopoietic Cell Transplantation or Gene Therapy

Journal of Clinical Immunology - Granulocyte transfusions are sometimes used as adjunctive therapy for the treatment of infection in patients with chronic granulomatous disease (CGD). However,...     

Update on gene therapy for immunodeficiencies

Primary immune deficiencies (PID) are due to blood cell defects and can be treated with transplantation of normal hematopoietic stem cells (HSC) from another person (allogeneic). Gene therapy in which a patient's autologous HSC are genetically corrected represents an alternative treatment for patients with PID, which could avoid the immunologic risks of allogeneic HSCT and confer similar benefits. Recent clinical trials using gene therapy have led to immune restoration in patients with X-linked severe combined immune deficiency (XSCID), adenosine deaminase (ADA)-deficient SCID and chronic granulomatous disease (CGD). However, severe complications arose in several of the patients in whom the integrated retroviral vectors led to leukoproliferative disorders. New approaches using safer integrating vectors or direct correction of the defective gene underlying the PID are being developed and may lead to safer and effective gene therapy for PID.     

Epidermal Langerhans' cells in Beh?et's disease.

Langerhans' cells were studied in the epidermis of two patients with active Behçet's disease and compared with those in two normal controls. Ultrastructural morphology and the percentage of Langerhans' cells found were similar in patients' (1.88%) and the control epidermis (1.79%). The density of Langerhans' cells in adjacent sites of the same epidermis was not homogeneous, being in the range of 0.8-2.8% in Behçet's disease and 0.6-4% in the controls. In the controls, Langerhans' cells were distributed unevenly. Some were located near the basal layer of the epidermis while the rest were in the mid and upper layers. In Behçet's disease most Langerhans' cells were in the mid-epidermis, but some were immediately beneath the stratum granulosum. In the Behçet's disease epidermis the area occupied by Langerhans' cells was increased by about 25% and the number of granules found increased by about 44%. It is suggested that in Behçet's disease the Langerhans' cells are in a more active state.     

Emergency health surveillance after severe flooding in Louisiana, 1995

INTRODUCTION: In disaster situations, timely surveillance systems that provide illness, injury, and mortality information to public health officials and hospitals are essential for planning and evaluating interventions. OBJECTIVES: To describe flood surveillance methodology, the impact of the event on hospitals, and the number of daily patient visits due to selected illnesses and injuries before, during, and after severe flooding in southeastern Louisiana in May 1995. METHODS: Survey of disaster-area hospitals regarding flood impact. Emergency department surveillance of injuries and illnesses for the week before, the two days during, and the week after the flood. RESULTS: There occurred an increase in the number of persons who drowned or were injured that presented to the moderately affected hospitals during the storm, but there was no increase in visits for gastroenteritis to any group of hospitals. Services were disrupted in more than half of hospitals. The severely affected hospitals had the least variation in the average number of daily visits. None of the drownings were reported by those hospitals that reported severe service disruption. CONCLUSIONS: Data should be collected from all hospitals in or near disaster areas, even if they were not directly affected by the disaster. Public education about the danger of drowning during flash flooding must be improved. The Louisiana experience emphasizes the need for a disaster-preparedness plan for rapid surveillance of illnesses and injuries.     

Diagnostic arthroscopy of the hip joint

Objective  Refinement of diagnosis of hip disorders with the possibility of adding minimally invasive surgical procedures. Indications  Hip symptoms and effusions not yet diagnosed. Disorders where a decision has to be taken whether to continue conservative care or to proceed with surgery. Intraarticular loose bodies. Labral lesions. Biopsies to establish a diagnosis. Synovial diseases. Localized articular cartilage defects. Purulent arthritis. Contraindications  Recent fracture of acetabulum. Advanced osteoarthritis. Impossibility to distract the joint. Surgical Technique  Supine. Arthrography for imaging of labrum and femoral head. Joint distension. 3-portal technique for arthroscopy of the central compartment of the joint under traction on a fracture table. 2-portal technique for viewing of the peripheral compartment without traction. Both techniques make use of 30° and 70° arthroscopes. Results  Between October 1997 and end of 2000, we performed 60 hip arthroscopies: 34 of the central and peripheral compartments, 16 of only the central and ten of only the peripheral compartment. Besides synovial reactions in all, 15 of 33 hips with early or moderate osteoarthritis showed additional intraarticular lesions. These findings justified an early total hip replacement in six patients. In four out of six patients with unclear hip symptoms, a final diagnosis could be established. In another five patients the diagnosis could be confirmed (4× chondromatosis, and 1× free body). In five patients indication for correctional osteotomy was validated and in one rejected due to advanced articular cartilage damage. Complications: transient hypoesthesia (4×), superficial cartilage damage (7×), instrument breakage (2×), small labral tears (4×), soft tissue edema and early termination of operation (2×).     

Intra-articular osteochondroma of the posterior cavity of the knee

A patient with a large intra-articular osteochondroma of the posterior cavity of the knee joint is presented. Preoperative imaging included radiographs and magnetic resonance imaging, which revealed a completely intra-articular, locally expanding, mineralised mass in the intercondylar tunnel and posterior joint cavity. Flexion was limited to 70 deg. The osteochondroma was removed from a posterior approach. Gross and histologic pictures are presented, and the origin and pathogenesis of the tumour are discussed. After removal, the clinical course was benign. Full flexion was regained within 2 months. At the 2-year follow-up, no signs of recurrence were evident on radiographs and magnetic resonance imaging.     

Parent-defined target symptoms respond to risperidone in RUPP autism study: customer approach to clinical trials

OBJECTIVE: A consumer-oriented efficacy assessment in clinical trials should measure changes in chief complaint and consumer request (symptoms of most concern to patient/caregiver), which may be diluted in change scores of multisymptom scales. METHOD: In the Research Units on Pediatric Psychopharmacology (RUPP) Autism Network 8-week double-blind trial of risperidone versus placebo, the chief concerns of parents were collected at 0, 4, and 8 weeks (endpoint), in addition to standardized primary measures. Blinded clinical judges rated change from baseline to 4 and 8 weeks on a 9-point scale (1 = normalized, 5 = unchanged, 9 = disastrous); 94 participants had usable data. RESULTS: The most common symptoms identified by parents were tantrums, aggression, and hyperactivity. Interrater reliability was excellent. Mean ratings at endpoint were 2.8 +/- 1.2 on risperidone and 4.5 +/- 1.3 on placebo (p <.001). Ratings were collinear with Clinical Global Impression-Improvement and Aberrant Behavior Checklist Irritability subscale (primary dimensional measure). Effect size d was 1.4, compared to 1.2 on the Aberrant Behavior Checklist Irritability subscale. Effect sizes varied twofold by symptom category, largest for self-injury (2.11) and tantrums (1.95). CONCLUSIONS: Risperidone was superior to placebo in reducing symptoms of most concern to parents of autistic children with irritable behavior. Rating individualized participant-chosen target symptoms seems a reliable, sensitive, efficient, and consumer-friendly way to assess treatment effect and might have clinical application.     

The epidemiology of major depressive episodes: results from the International Consortium of Psychiatric Epidemiology (ICPE) Surveys

Absence of a common diagnostic interview has hampered cross-national syntheses of epidemiological evidence on major depressive episodes (MDE). Community epidemiological surveys using the World Health Organization Composite International Diagnostic Interview administered face-to-face were carried out in 10 countries in North America (Canada and the US), Latin America (Brazil, Chile, and Mexico), Europe (Czech Republic, Germany, the Netherlands, and Turkey), and Asia (Japan). The total sample size was more than 37,000. Lifetime prevalence estimates of hierarchy-free DSM-III-R/DSM-IV MDE varied widely, from 3% in Japan to 16.9% in the US, with the majority in the range of 8% to 12%. The 12-month/lifetime prevalence ratio was in the range 40% to 55%, the 30-day/12-month prevalence ratio in the range 45% to 65%, and median age of onset in the range 20 to 25 in most countries. Consistent socio-demographic correlates included being female and unmarried. Respondents in recent cohorts reported higher lifetime prevalence, but lower persistence than those in earlier cohorts. Major depressive episodes were found to be strongly co-morbid with, and temporally secondary to, anxiety disorders in all countries, with primary panic and generalized anxiety disorders the most powerful predictors of the first onset of secondary MDE. Major depressive episodes are a commonly occurring disorder that usually has a chronic-intermittent course. Effectiveness trials are needed to evaluate the impact of early detection and treatment on the course of MDE as well as to evaluate whether timely treatment of primary anxiety disorders would reduce the subsequent onset, persistence, and severity of secondary MDE.     

Couples therapy at end of life

This study reports on a preliminary uncontrolled study of a treatment for couples in which one partner is diagnosed with a terminal illness. In this study nine couples, in which one partner was diagnosed with a terminal illness and had less than 18 months to live, were offered eight sessions of couples therapy. Follow-up data were available for six couples. Two patients died during treatment and a third patient moved out of the area prior to completing the follow-up assessment. Significant decreases were seen in the patients' distress about dying and the frequency of partners' worry about their partner dying. Improvements were also seen in relationship quality. While the size of the study precludes generalizing these findings to a larger population, this study supports further exploration of couples therapy as a potentially useful adjunct to end of life care.