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PubMed1999

Attitudes and experiences of restaurateurs regarding smoking bans in Adelaide, South Australia.

OBJECTIVES: To determine compliance with a voluntary code of practice (VCP) for restricting smoking in restaurants and to canvass the attitudes of restaurateurs towards tougher smoking restrictions. DESIGN: Cross-sectional survey conducted in 1996 using a telephone questionnaire. SETTING: Metropolitan restaurants and cafes in Adelaide, South Australia. PARTICIPANTS: 276 (86.8%) of a sample of randomly selected owners and managers. MAIN OUTCOME MEASURES: Restaurant non-smoking policies, reported and anticipated change in business, and restaurateurs' attitudes towards smoking restrictions. RESULTS: 26.8% of restaurants had a total smoking ban; 40.6% restricted smoking some other way; and 32.6% permitted unrestricted smoking. Only 15.1% of restaurants with a ban or restrictions had used the VCP to guide the development of their policy, and only half of these were complying with it. Although 78.4% of those with bans and 84.4% of those with restrictions reported that their non-smoking policy had been associated with either no change or a gain in business, only 33.3% of those allowing unrestricted smoking expected that this would be the case, if they were to limit smoking. A total of 50.4% of restaurateurs, including 45.3% of those with no restrictions, agreed that the government should ban smoking in all restaurants. CONCLUSIONS: The VCP made an insignificant contribution to adoption of non-smoking policies, and compliance with the code was poor. Despite concerns about loss of business, there was considerable support for legislation which would ban smoking in all dining establishments.

Attitude to Health↗
PubMed1996

Relationship between volume of bathing medium and ectoenzyme activity in monolayers of cultured BPAEC.

It is unclear whether all or a fraction of the capillary plasma volume (Vcp) serves as the reaction volume (Vr) for pulmonary capillary endothelial ectoenzymes, in vivo. Cultured endothelial cell (EC) monolayers provide a convenient model for studying EC-bound enzyme-Vr relationships. Because the Michaelis-Menten parameter [maximum velocity of enzyme reaction (Vmax) = E x kcat/Vr, where E is enzyme mass and kcat is the constant of product formation] is inversely proportional to Vr, we hypothesized that increasing the volume of medium (Vm) bathing EC monolayers would proportionally reduce the calculated Vmax (or Vmax/K(m), where K(m) is the Michaelis constant) values of an ectoenzyme reacting with a substrate only if, and as long as, Vm = Vr. To test this hypothesis, studies were performed in bovine pulmonary arterial EC grown to confluence. Activities of angiotensin-converting enzyme (ACE) and 5'-nucleotidase (NCT) were assayed in Earle's balanced salts solution utilizing [3H]benzoyl-Phe-Ala-Pro ([3H]BPAP) and 5'-[14C]AMP as substrates, respectively. Under first-order reaction conditions and at constant substrate concentrations ([BPAP] = 15 nM, [AMP] = 1 microM), Vmax/K(m) ratios of ACE and NCT declined to 20% of their original values, as Vm increased from 0.6 to 2 ml. ACE activity was also studied at constant substrate mass (BPAP = 7 pmol) under first-order reaction conditions. Again, enzyme activity (Vmax/K(m)) declined proportionally to increasing Vm. Under zero-order reaction conditions ([BPAP] = 250 microM), ACE activity (Vmax) was similarly related to Vm. Linear regression analyses revealed that ACE or NCT would recognize up to at least 3 ml Vm, a volume vastly exceeding that of Vcp in a section of the capillary bed composed of an equivalent number of ECs, thus suggesting that Vcp could serve as the reaction volume for pulmonary capillary EC ectoenzymes in vivo.

5'-Nucleotidase↗
PubMed1988

Effects of inhibitors on chloride outflux from cerebrospinal fluid.

Movement of chloride from cerebrospinal fluid (CSF) to brain or blood is one of the factors that may be involved in regulation of CSF [Cl-], which is important to CSF acid-base balance. We made quantitative measurements of the unidirectional outflux of radiolabeled chloride (38Cl, half-life 37.3 min) from CSF in anesthetized dogs, using ventriculocisternal perfusion (VCP). The outflux of 38Cl from CSF was determined from the difference between the movements of 38Cl and dextran using a one-compartment model. VCP was performed at a rate of 1.4 ml/min for 14 min, and then slowed to 0.28 ml/min. The 38Cl activity decreased to a steady-state level approximately 12% lower than that of dextran within 40-50 min. Under control conditions for the first run (n = 24), the flux was 0.042 +/- 0.003 (SE) ml/min. The outflux under control conditions (n = 6) tended to increase over three separate determinations in a 6-h period, being 136 +/- 19% of the first run on the second run, and 143 +/- 24% on the third. There were no significant changes in 38Cl outflux compared with control ratios after the inclusion of bumetanide in the VCP fluid (n = 6), which inhibits sodium-coupled Cl- transport, with acetazolamide (n = 6), which inhibits carbonic anhydrase, or with 4,4'-diisothiocyanostilbene-2,2'-disulfonic acid (n = 6), an inhibitor of carrier-mediated anion exchange. These results suggest that the outward movement of chloride from CSF occurs mostly by passive diffusion and is not by mediated transport.

4-Acetamido-4'-isothiocyanatostilbene-2,2'-disulfo↗
PubMed2007

Videocapillaroscopy in the differential diagnosis between psoriasis and seborrheic dermatitis of the scalp.

BACKGROUND: Videocapillaroscopy (VCP) can be used to explore microcirculatory modifications in skin diseases. Psoriasis shows specific capillary alterations reflecting typical histopathological changes. OBJECTIVE: To compare capillary morphology, distribution and density in psoriasis and seborrheic dermatitis of the scalp for differential diagnosis. METHODS: VCP was performed on histology-confirmed scalp lesions of 30 patients with chronic plaque psoriasis, 30 age- and sex-matched patients with seborrheic dermatitis and 30 healthy subjects. The morphology, mean density per mm(2) and mean diameter of capillary loops was measured. RESULTS: Scalp psoriasis exhibited homogeneously tortuous and dilated capillaries (bushy pattern), with a 73 +/- 17 mum (mean +/- standard deviation) diameter of capillary bushes. In contrast, scalp seborrheic dermatitis presented a multiform pattern, with mildly tortuous capillary loops and isolated dilated capillaries, but a substantial preservation of local microangioarchitecture. Mean diameter of capillary bush was significantly lower (27 +/- 4 microm; p < 0.001) and similar to that of the scalp of healthy subjects (21 +/- 5 microm). Capillary loop density was similar in patients with psoriasis (23 +/- 5/mm(2)) seborrheic dermatitis (24 +/- 2/mm(2)) and healthy scalp skin (24 +/- 3/mm(2)). CONCLUSION: VCP could be a useful noninvasive approach for differentiating between psoriasis and seborrheic dermatitis, especially when the scalp is the only affected site.

Adult↗
PubMed2005

Humanized recombinant vaccinia virus complement control protein (hrVCP) with three amino acid changes, H98Y, E102K, and E120K creating an additional putative heparin binding site, is 100-fold more active than rVCP in blocking both classical and alternative complement pathways.

Vaccinia virus complement control protein (VCP) is able to modulate the host complement system by regulating both pathways of complement activation. Efficient downregulation of complement activation depends on the ability of the regulatory protein to effectively bind the activated third (C3b) and fourth (C4b) complement components. Based on native crystallographic structure, molecular modeling, and sequence alignment with other Orthopoxviral complement control proteins (CCPs) and their host homologs, putative sites have been found on VCP as contact points for C3b/C4b. Here, we report that using site-directed mutagenesis, modified proteins have been generated. In addition, we report that the generated modified proteins with postulated contact point substitutions have shown greater ability to regulate both the classical and the alternative pathways of complement activation than the recombinant Western Reserve VCP, with one modified protein showing nearly 100-fold more potency in regulating both complement activation pathways independently. The augmented in vitro inhibitory activity of the modified protein together with the newly created putative heparin binding site suggests its promising potential as a competent therapeutic agent in modulating various complement-mediated ailments, for example, traumatic brain injury, Alzheimer's disease, rheumatoid arthritis, multiple organ dysfunction syndrome, reperfusion injury, and xenorejection.

Amino Acid Sequence↗
PubMed1986

Improved survival duration with combination chemotherapy induction for multiple myeloma: a Southwest Oncology Group Study.

Four hundred forty previously untreated patients with active multiple myeloma were entered into a randomized trial (Southwest Oncology Group [SWOG] study 7927/28) comparing vincristine, melphalan, Cytoxan (Mead Johnson & Company, Evansville, Ind), and prednisone (VMCP) alternating with vincristine, BCNU, Adriamycin (Adria Laboratories, Columbus, Ohio) and prednisone (VBAP) with or without levamisole with vincristine, Cytoxan, and prednisone (VCP) with or without levamisole for induction therapy. The treatment groups were well balanced for all of the known major prognostic factors. Patients receiving VMCP-VBAP responded (greater than or equal to 75% regression) more frequently to induction therapy, both without (54%) and with (44%) levamisole v VCP without (28%) or with (28%) levamisole (P less than .001). In addition, patients receiving VMCP-VBAP (+/- levamisole) had a survival duration determined to be significantly increased by all forms of analysis: 48 and 33 months for VMCP-VBAP without and with levamisole v 29 and 26 months for VCP without and with levamisole (P = .011 overall). Levamisole did not improve response rates or survival duration (P greater than or equal to .1), nor did it prolong remission in the maintenance phase (P = .85). Analysis of SWOG study 7704/05 (updated April 1985) confirmed improved survival for combination therapy v MP, but no benefit for levamisole. The overall findings support the use of VMCP-VBAP as an excellent treatment option for remission induction in patients with active myeloma of all stages and prognostic categories.

Actuarial Analysis↗
PubMed1999

Metastasis stage, adjuvant treatment, and residual tumor are prognostic factors for medulloblastoma in children: conclusions from the Children's Cancer Group 921 randomized phase III study.

PURPOSE: From 1986 to 1992, "eight-drugs-in-one-day" (8-in-1) chemotherapy both before and after radiation therapy (XRT) (54 Gy tumor/36 Gy neuraxis) was compared with vincristine, lomustine (CCNU), and prednisone (VCP) after XRT in children with untreated, high-stage medulloblastoma (MB). PATIENTS AND METHODS: Two hundred three eligible patients with an institutional diagnosis of MB were stratified by local invasion and metastatic stage (Chang T/M) and randomized to therapy. Median time at risk from study entry was 7.0 years. RESULTS: Survival and progression-free survival (PFS) +/- SE at 7 years were 55%+/-5% and 54%+/-5%, respectively. VCP was superior to 8-in-1 chemotherapy, with 5-year PFS rates of 63%+/-5% versus 45%+/-5%, respectively (P = .006). Upon central neuropathology review, 188 patients were confirmed as having MB and were the subjects for analyses of prognostic factors. Children aged 1.5 to younger than 3 years had inferior 5-year estimates of PFS, compared with children 3 years old or older (P = .0014; 32%+/-10% v 58%+/-4%, respectively). For MB patients 3 years of age or older, the prognostic effect of tumor spread (MO v M1 v M2+) on PFS was powerful (P = .0006); 5-year PFS rates were 70%+/-5%, 57%+/-10%, and 40%+/-8%, respectively. PFS distributions at 5 years for patients with M0 tumors with less than 1.5 cm2 of residual tumor, versus > or = 1.5 cm2 of residual tumor by scan, were significantly different (P = .023; 78%+/-6% v 54%+/-11%, respectively). CONCLUSION: VCP plus XRT is a superior adjuvant combination compared with 8-in-1 chemotherapy plus XRT. For patients with M0 tumors, residual tumor bulk (not extent of resection) is a predictor for PFS. Patients with M0 tumors, > or = 3 years with < or = 1.5 cm2 residual tumor, had a 78%+/-6% 5-year PFS rate. Children younger than 3 years old who received a reduced XRT dosage had the lowest survival rate.

Adolescent↗
PubMed2006

Immunophysical properties and prediction of activities for vaccinia virus complement control protein and smallpox inhibitor of complement enzymes using molecular dynamics and electrostatics.

We present immunophysical modeling for VCP, SPICE, and three mutants using MD simulations and Poisson-Boltzmann-type electrostatic calculations. VCP and SPICE are homologous viral proteins that control the complement system by imitating, structurally and functionally, natural regulators of complement activation. VCP and SPICE consist of four CCP modules connected with short flexible loops. MD simulations demonstrate that the rather complex modules of VCP/SPICE and their mutants exhibit a high degree of intermodular spatial mobility, which is affected by surface mutations. Electrostatic calculations using snapshots from the MD trajectories demonstrate variable spatial distribution of the electrostatic potentials, which suggests dynamic binding properties. We use covariance analysis to identify correlated modular oscillations. We also use electrostatic similarity indices to cluster proteins with common electrostatic properties. Our results are compared with experimental data to form correlations between the overall positive electrostatic potential of VCP/SPICE with binding and activity. We show how these correlations can be used to predict binding and activity properties. This work is expected to be useful for understanding the function of native CCP-containing regulators of complement activation and receptors and for the design of antiviral therapeutics and complement inhibitors.

Amino Acid Sequence↗
PubMed2000

Vulvitis circumscripta plasmacellularis mimicking child abuse.

Vulvitis circumscripta plasmacellularis (VCP) is a rare, benign vulvar disorder that is typically described in adult women. Our case occurred in an 8-year-old girl. The primary diagnostic concern was sexual abuse. VCP may also mimic lichen sclerosus, extramammary Paget's disease, pemphigus vulgaris, fixed drug eruption, squamous cell carcinoma, candidiasis, allergic contact dermatitis, and herpes simplex infection. Evaluation should include a biopsy because the histopathologic features of VCP are distinctive.

Biopsy↗
PubMed2003

Microvascular assessment in Behçet disease: videocapillaroscopic study.

The aim of this study was to evaluate microvascular assessment in patients with Behcet disease (BD) by means of an intravital videocapillaroscopic study. Sixteen BD patients were compared with an equivalent group of healthy subjects matched for age and sex. Videocapillaroscopy (VCP) was performed in peripheral areas and in conjunctiva, and morphological and quantitative parameters were assessed. In both areas VCP showed several morphological alterations (microaneurysms, megacapillaries, desertification areas) detectable in a high percentage of patients; quantitatively we found significant changes of incisuring and sludging score, of capillary loop intermediate branch length (in peripheral areas) and of arteriole/venule diameter (in conjunctiva). Therefore, vessel involvement included both the number and the whole vessel structure and was seen both in peripheral and conjunctival areas when the two different vascular beds of micro- and paramicrocirculation were examined. We conclude that an important rearrangement of microcirculation is detectable in BD and that VCP may have diagnostic and prognostic value, providing qualitative and quantitative information able to define the systemic extension of vascular damage and the degree of vessel wall alteration.

Adult↗
PubMed2003

[Study on the expression of the metastasis suppressor gene KAI1 in laryngeal squamous cell carcinoma].

OBJECTIVE: To investigate the expression of the metastasis suppressor gene KAI1 in laryngeal squamous cell carcinoma and the relationship between expression of KAI1 and it's role in tumorgenesis and development. METHODS: The sections of 84 samples of primary laryngeal squamous cell carcinoma (PLSCC), 27 samples of laryngeal precancerous lesion (LPL), 10 samples of vocal cords polyp (VCP) and 10 samples of normal laryngeal tissues (NLT) were examined by in situ hybridization for KAI1mRNA. RESULTS: It was found that 10 samples of NLT and 10 samples of VCP showed strong expression of KAI1, the Integrated A Total of KIA1 in these two types of tissues were 136,206.8 +/- 36,675.536 and 133,674.505 +/- 42,858.456, respectively, with no statistically significant difference between them, and high prevalence of decreased expression of KAI1 in LPL and PLSCC compared with that in VCP and in NLT (P < 0.01). There was a significant relationship between down-regulation of KAI1 expression with pathological grade, lymph node metastases, T stage and clinical stage (P < 0.01) and did not correlate with gender. CONCLUSION: KAI1 may play an important role on tumor genesis, development, invasion and metastasis of PLSCC, and become one of the molecular markers for early diagnosis and predicting of aggressive and metastasis potential to assess the clinical stage of PLSCC.

Adult↗
PubMed2004

[Videothoracoscopic collagen pleurodesis in malignant pleural effusion].

AIM: To devise the technique and evaluate the efficacy of videothoracoscopic collagen pleurodesis (VCP) in malignant pleural exudates (MPE). MATERIAL AND METHODS: VCP was performed in 24 patients with recurrent MPE. 20 patients were diagnosed to have non-small-cell cancer of the lung, 4 patients--breast cancer. The operation included videothoracoscopic revision of the pleural cavity under endobronchial anesthesia, pleural biopsy, liquid evacuation, dispersion of powder collagen (1 g) and drainage of the pleural cavity. RESULTS: Videothoracoscopy macroscopically diagnoses pleural carcinomatosis in 100% patients. Pathomorphological examination of the biopsies defined a histological type of the tumor in all 24 patients. A positive clinicoroentgenological effect with a compete disappearance of pleural exudates was achieved in all the cases. The recurrence was absent in 18 of 24 patients. Short- and long-term results, survival and quality of life are outlined. CONCLUSION: VCP is a simple and effective method of palliative treatment improving quality of life in patients with malignant pleural exudates.

Collagen↗
PubMed2004

[Distribution and prognostic significance of microvessel density in supraglottic laryngeal squamous cell carcinomas].

OBJECTIVE: To study the distribution and prognostic significance of microvessel density (MVD) in supraglottic laryngeal squamous cell carcinomas (SGLSCC). METHOD: The patients of 59 primary SGLSCC received surgical treatment and 19 vocal cord polyps (VCP) were studied with CD31 immunohistochemical staining using Two Steps System Kits. RESULT: There were many microvessels in the stroma of SGLSCC compared with the connective-tissue under epithelium of VCP. Kendall's ranked correlation analysis indicated that only MVD and nodal status had significant correlation (P<0.01) in all clinicopathological parameters. Both between N0 and N+ and between SGLSCC and VCP MVD were significantly different (P<0.01) by independent-samples t test. Kaplan-Meier survival analysis showed significant relationship existed in MVD and cumulative survival rate of SGLSCC patients (P<0.01). CONCLUSION: This study further proves that angiogenesis plays important roles in tumorigenesis, invasiveness and metastasis. MVD could predict the lymph node metastasis and are significantly associated with the prognosis of SGLSCC patients.

Adult↗
PubMed2005

[Expression and significance of PTEN and Survivin between the primary foci and the lymph node metastases foci of laryngeal squamous cell carcinoma].

OBJECTIVE: To investigate the molecular difference and significance between the primary foci and metastases foci of laryngeal squamous cell carcinoma (LSCC). METHOD: Tissues of 18 cases of positive lymph node metastases (N+) LSCC were detected by using SP method. The LSCC primary foci and metastases foci and negative metastases lymph nodes were included, 22 cases of vocal cord polyp (VCP) were regarded as control. expression of PTEN and Survivin were evaluated by SP immunohistochemistry. RESULT: Expression of neither PTEN nor Survivin were positive in negative nodes. The positive rates of PTEN in LSCC, N+, VCP were 94.4% (17/18), 94.4% (17/18) and 95.5% (21/22) respectively, there were no significant difference among them (P>0.05). But the expression degree were more powerful in N+ than in the LSCC (P<0.05). There was no expression of Survivin in VCP. The positive rates of Survivin in LSCC and N+ were 55.5% (10/18), 50.0% (9/18), the difference were significant (P<0.01); but between LSCC and N+, neither the positive rate nor the expression degree had significant difference (P>0.05). CONCLUSIONS: There were differences between the LSCC primary foci and the lymph node metastases foci in molecular level probably.

Adult↗
PubMed2006

Pennsaid therapy for osteoarthritis of the knee: a systematic review and metaanalysis of randomized controlled trials.

OBJECTIVE: To systematically review published randomized controlled trials (RCT) evaluating a new topical diclofenac solution (Pennsaid) in patients with osteoarthritis (OA) of the knee. METHODS: RCT were identified by searching electronic data sources as well as by contact with the manufacturer of Pennsaid. Details of study demographics, methodology, quality, and outcomes were analyzed. A metaanalysis evaluating the efficacy and safety of Pennsaid in OA of the knee was performed. RESULTS: Four RCT were analyzed in this systematic review (3 published reports and one published abstract). Mean trial duration was 8.5 weeks. Generally, these RCT were of excellent quality. The mean Jadad quality score was 4.5 out of 5. Many indicators of high quality in a RCT were found in these RCT, including adequate descriptions of the methods used for randomization, blinding, and allocation concealment. In comparison to a vehicle control placebo (VCP), the standardized mean differences (SMD) for the WOMAC pain, stiffness, and physical function subscales, as well as for patient global assessment, were all statistically significant in favor of Pennsaid, with SMD ranging from 0.30 to 0.39. Pennsaid was as safe as VCP, with the only exception that it was more likely to result in minor skin dryness at the site of application (relative risk 1.7). In a 12 week equivalence trial that used the WOMAC subscales to compare treatment response, Pennsaid was as effective as oral diclofenac, but was much better tolerated. CONCLUSION: Pennsaid is an effective topical NSAID in patients with OA of the knee. Apart from minor localized skin reactions, Pennsaid was as safe as VCP. It is not known whether the favorable results of Pennsaid can be extrapolated to other topical NSAID preparations. Pennsaid deserves further consideration when the existing treatment guidelines for OA of the knee are updated.

Administration, Topical↗
PubMedDate not supplied

[Use of enalapril, an angiotensin-converting enzyme inhibitor, in pulmonary artery hypertension].

Enalapril (E) was used to treat 16 patients with pulmonary arterial hypertension, 6 primary and 10 secondary, 5 of the latter with congenital heart disease and 5 with chronic obstructive pulmonary disease. The average dose of E was 20 mg/day. All patients underwent pre and post-treatment cardiac catheterization with determination of pressures at: right atrial (RA), main pulmonary artery (MPA), pulmonary capillary wedge pressure ( VCP) and systemic arterial (SA). Resistances forces were also measured as; total pulmonary (TPR), pulmonary arteriolar (PAR) and total systemic (TSR) as well as cardiac output (CO), and echo and electrocardiograms, chest x ray, stress test and respiratory function test. The functional class (NYHA) improved in all (p less than 0.001). The initial mean pressures were: RA 12.24 +/- 4.35; MPA 73.81 +/- 25.16; VCP 12 +/- 2.73 and SA 89 +/- 14; TPR 1477 +/- 761; PAR 1243 +/- 730 and TSR 1684 +/- 505.5; CO 4.5 +/- 1.29. The final values were: RA 9.66 +/- 2.46 (p less than 0.001); MPA 63.26 +/- 24.45 (p less than 0.001); VCP 11.33 +/- 2.38 (p = NS); SA 81 +/- 10 (p less than 0.001); TPR 1009.5 +/- 536.7 (p less than 0.001); PAR 829 +/- 511.5 (p less than 0.001); TSR 1309.6 +/- 296.3 (p less than 0.001); CO 5.2 +/- 1.44 (p less than 0.001). The average of minutes on treadmill was initially 8.2 +/- 2.45 and final 12.46 +/- 3.0 (p less than 0.001). It is concluded that enalapril is a useful drug in the treatment of pulmonary arterial hypertension of any etiology.

Adult↗
PubMed2007

Multisystem Proteinopathy

CLINICAL CHARACTERISTICS: Multisystem proteinopathy (MSP) is a genetically heterogeneous, multisystem degenerative disorder characterized by adult-onset proximal and distal muscle weakness (clinically resembling a limb-girdle muscular dystrophy syndrome), early-onset Paget disease of bone (PDB), and premature frontotemporal dementia (FTD). Muscle weakness progresses to involve other limb and respiratory muscles. PDB involves focal areas of increased bone turnover that typically lead to spine and/or hip pain and localized enlargement and deformity of the long bones; pathologic fractures occur on occasion. Early stages of FTD are characterized by dysnomia, dyscalculia, comprehension deficits, and paraphasic errors, with minimal impairment of episodic memory; later stages are characterized by inability to speak, auditory comprehension deficits for even one-step commands, alexia, and agraphia. Mean age at diagnosis for muscle disease is 43 years, PDB is 41 years, and FTD is 56 years. Dilated cardiomyopathy, amyotrophic lateral sclerosis, and Parkinson disease are now known to be part of the spectrum of findings associated with MSP. DIAGNOSIS/TESTING: The diagnosis of MSP is established in a proband with typical clinical findings and a heterozygous pathogenic variant in VCP, HNRNPA1, HNRNPA2B1, or SQSTM1 identified by molecular genetic testing. MANAGEMENT: Treatment of manifestations: Weight control to avoid obesity; physical therapy and stretching exercises to promote mobility and prevent contractures; occupational therapy and mechanical aids (canes, walkers, orthotics, wheelchairs) as needed for ambulation/mobility; surgical intervention for foot deformity and scoliosis as needed; respiratory aids when indicated; assisted living arrangements for muscle weakness and/or dementia; bisphosphonates to relieve pain and disability from PDB; social and emotional support; education regarding safety precautions. Surveillance: Echocardiogram and electrocardiogram with repeat cardiac evaluation every two to three years or earlier if symptomatic; annual pulmonary function studies; sleep studies as clinically indicated; annual alkaline phosphatase measurement; skeletal imaging as indicated for evaluation of PDB; neurologic and neuropsychological assessment every two to three years or more frequently as needed; multidisciplinary monitoring for respiratory, cardiac, musculoskeletal, and cognitive decline. GENETIC COUNSELING: MSP is inherited in an autosomal dominant manner. Most individuals diagnosed with MSP have an affected parent. Estimates based largely on VCP-MSP suggest that approximately 5% of individuals have a de novo pathogenic variant. Each child of an individual with MSP has a 50% chance of inheriting the MSP-related pathogenic variant. Marked intrafamilial variability may be observed among heterozygous family members, including differences in age at onset, severity, rate of progression, and the specific combination of manifestations. Once the MSP-related pathogenic variant has been identified in an affected family member, predictive testing for at-risk family members and prenatal/preimplantation genetic testing are possible.

Inclusion Body Myopathy with Early-Onset Paget Dis↗
PubMed2006

Six-month trial of bupropion with contingency management for cocaine dependence in a methadone-maintained population.

CONTEXT: No effective pharmacotherapies exist for cocaine dependence, although contingency management (CM) has demonstrated efficacy. OBJECTIVE: To compare the efficacy of bupropion hydrochloride and CM for reducing cocaine use in methadone hydrochloride-maintained individuals. DESIGN: This 25-week, placebo-controlled, double-blind trial randomly assigned participants to 1 of 4 treatment conditions: CM and placebo (CMP), CM and 300 mg/d of bupropion hydrochloride (CMB), voucher control and placebo (VCP), or voucher control and bupropion (VCB). SETTING: Outpatient clinic at the Veterans Affairs Connecticut Healthcare System. PARTICIPANTS: A total of 106 opiate-dependent, cocaine-abusing individuals. INTERVENTIONS: All study participants received methadone hydrochloride (range, 60-120 mg). Participants receiving bupropion hydrochloride were given 300 mg/d beginning at week 3. In the CM conditions, each urine sample negative for both opioids and cocaine resulted in a monetary-based voucher that increased for consecutively drug-free urine samples during weeks 1 to 13. Completion of abstinence-related activities also resulted in a voucher. During weeks 14 to 25, only completion of activities was reinforced in the CM group, regardless of sample results. The voucher control groups received vouchers for submitting urine samples, regardless of results, throughout the study. MAIN OUTCOME MEASURE: Thrice-weekly urine toxicologic test results for cocaine and heroin. RESULTS: Groups did not differ in baseline characteristics or retention rates. Opiate use decreased significantly, with all treatment groups attaining equivalent amounts of opiate use at the end of the study. In the CMB group, the proportion of cocaine-positive samples significantly decreased during weeks 3 to 13 (P<.001) relative to week 3 and remained low during weeks 14 to 25. In the CMP group, cocaine use significantly increased during weeks 3 to 13 (P<.001) relative to week 3, but then cocaine use significantly decreased relative to the initial slope during weeks 14 to 25 (P<.001). In contrast, by treatment end, the VCB and VCP groups showed no significant improvement in cocaine use. CONCLUSION: These findings suggest that combining CM with bupropion for the treatment of cocaine addiction may significantly improve outcomes relative to bupropion alone.

Adult↗