Long-term effects of therapeutic education for patients with rheumatoid arthritis

IntroductionThe objective of this study was to assess the effects of an educational program on the course of rheumatoid arthritis (RA) after 3 years.MethodsFrom December 2002 to December 2003, 39 RA patients participated in a 3-day education program delivered to groups of four or five patients. Effects of the program were evaluated after 3 years in 33 patients, comparatively to baseline, based on the following variables: knowledge of RA (self-questionnaire), disease activity (DAS 28), functional impairment (health assessment questionnaire [HAQ]) and quality of life (arthritis impact measurement scale 2 [AIMS2], short-form). We also compared patient knowledge in the educational program participants and in 38 controls with RA. Direct questions were used to evaluate the program after 3 years.ResultsPatient knowledge 3 years after the education program was significantly improved compared to baseline (P < 0.0001) and was significantly better than in the controls (P < 0.0001). Disease activity was significantly lower in the education group after 3 years than at baseline (DAS28, 3.1 vs. 3.8, P < 0.005). Neither the HAQ nor the AIMS2 scores changed significantly after 3 years compared to baseline. The replies to the direct questions indicated a very high level of overall satisfaction with the educational program.ConclusionAn educational program tailored to patient needs can produce lasting improvements in knowledge of the disease and may help to control the activity of RA. These results warrant the development of education programs for patients with chronic inflammatory joint disease.     

Bone ultrasonometry, bone density, and turnover markers in type 1 Gaucher disease

In 12 patients (mean age, 33 ± 13 years) with type 1 Gaucher disease (GD), we evaluated bone mass by broadband ultrasound attenuation (BUA) of the calcaneus and dual X-ray absorptiometry (DXA) of the total body, lumbar spine, and hip. In all patients, we measured serum levels of osteocalcin (OC) and bone-specific alkaline phosphatase (BAP) and urinary excretion of pyridinoline (Pyr/Cr) and deoxypyridinoline (D-Pyr/Cr) cross-links. Compared to age- and sex-matched healthy controls, patients with GD showed marked osteopenia at all measuring sites as expected. Values of BUA (67.25 ± 15.83 dB/MHz) were also significantly reduced. OC and BAP concentrations were within the normal range. Pyr/Cr and D-Pyr/Cr were significantly higher than in controls. Calculating T- and Z scores, we found a significant correlation between the Bone Severity Score Index (BSSI) and both BUA and BMD measurements. A significant correlation was also found between pyridinoline urinary excretion and both BSSI and BUA at the calcaneus. Our data suggest that type 1 GD in adulthood is associated with increased bone resorption and that BUA at the calcaneus may be a relevant tool in the assessment of bone status in these patients. Received: April 2, 2001 / Accepted: August 6, 2001     

Lepirudin use in a neonate with heparin-induced thrombocytopenia

OBJECTIVE: To describe a case of heparin-induced thrombocytopenia (HIT) in a premature infant and the doses of danaparoid and lepirudin needed to achieve appropriate therapeutic endpoints. CASE SUMMARY: A 30-week gestational age infant was diagnosed with HIT with heparin antibodies. Danaparoid 2.0-2.4 units/kg/h achieved anti-Xa levels of 0.2-0.4 U/mL, but thrombocytopenia failed to resolve. Lepirudin was started in place of danaparoid. Lepirudin doses of 0.03-0.05 mg/kg/h achieved target activated partial thromboplastin time values of 1.5-2.0 times baseline. DISCUSSION: Dosing information for danaparoid in neonates is limited, and information for lepirudin appears only in German literature at this time. HIT is well documented in newborns, and lepirudin use in these situations is likely to increase. This report provides some guidance for optimal dosing. It also provides some guidance for HIT evaluation in preterm infants, in whom blood volume for laboratory tests is a major issue. CONCLUSIONS: HIT is an important and potentially fatal problem in neonates. Lepirudin may be the drug of choice, especially since danaparoid is now unavailable. Initial lepirudin dosing should not exceed 0.05 mg/kg/h.     

Meeting report: FDA public meeting on patient‐focused drug development and medication adherence in solid organ transplant patients

The Food and Drug Administration (FDA) held a public meeting and scientific workshop in September 2016 to obtain perspectives from solid organ transplant recipients, family caregivers, and other patient representatives. The morning sessions focused on the impact of organ transplantation on patients’ daily lives and the spectrum of activities undertaken to maintain grafts. Participants described the physical, emotional, and social impacts of their transplant on daily life. They also discussed their posttransplant treatment regimens, including the most burdensome side effects and their hopes for future treatment. The afternoon scientific session consisted of presentations on prevalence and risk factors for medication nonadherence after transplantation in adults and children, and interventions to manage it. As new modalities of Immunosuppressive Drug Therapy are being developed, the patient perceptions and input must play larger roles if organ transplantation is to be truly successful.     

The biology of stem cell factor,a new hematopietic growth factor involved in stem cell regulation

Summary Recently, a new hematopoietic growth factor, stem cell factor, the ligand for the c-kit-proto-oncogene, has been cloned. The gene for this factor or for its receptor are deleted in two well know series of mice mutants which display pleiotropic stem cell defects. Therefore, this factor supposedly plays an important role in stem cell biology. This paper reviews some of the elegant genetic work which led to the discovery of the factor and of its receptor, the biological effects that this factor exerts in the hematopoietic system in normal individuals and in patients with Diamond-Blackfan anemia and speculates on some of its potential clinical applications.