scholarly journals The safety and effectiveness of chenodeoxycholic acid treatment in patients with cerebrotendinous xanthomatosis: two retrospective cohort studies

2019 ◽  
Vol 41 (4) ◽  
pp. 943-949 ◽  
Author(s):  
Aad Verrips ◽  
Maria Teresa Dotti ◽  
Andrea Mignarri ◽  
Bianca M. L. Stelten ◽  
Sue Verma ◽  
...  
Keyword(s):  
Cohort Studies ◽  
Chenodeoxycholic Acid ◽  
Retrospective Cohort ◽  
Signs And Symptoms ◽  
Term Treatment ◽  
Cerebrotendinous Xanthomatosis ◽  
Long Term Treatment ◽  
Retrospective Cohort Studies ◽  
The Impact

Abstract Objective To evaluate the safety and effectiveness of chenodeoxycholic acid (CDCA) treatment in patients with cerebrotendinous xanthomatosis (CTX). Methods Two retrospective cohort studies were conducted in CTX patients who underwent CDCA treatment: one in the Netherlands (NL; CDCA-STUK-15-001) and one in Italy (IT; CDCA-STRCH-CR-14-001). Eligible patients were aged 2–75 years, had been diagnosed with CTX, and were treated with CDCA orally for ≥1 year. The impact of CDCA treatment on biochemical markers (including serum cholestanol levels) and disease signs and symptoms were assessed, in addition to the safety and tolerability of CDCA treatment. Results A total of 35 patients were screened in the NL study and were diagnosed with CTX at 25.6 (± 13.7 SD) years on average. These patients were treated with CDCA and followed up for a median of 9.00 (range: 0.4–26.3) years. In addition, 28 patients were enrolled in the IT study and were diagnosed at 35.0 (± 11.4 SD) years on average (median duration of CDCA treatment: 5.75 [range: 0.0–25.0] years). Signs and symptoms of disease resolved, improved, or remained stable in many patients, with concomitant improvements in biochemical marker levels (serum cholestanol, p < 0.001; 7α-hydroxy-4-cholesten-3-one, p < 0.001 [IT study]). Conclusions The outcomes of these retrospective cohort studies indicate that CDCA is effective in the long-term treatment of CTX, with an acceptable safety profile.

scholarly journals Long-term Treatment of Cerebrotendinous Xanthomatosis (CTX) with Chenodeoxycholic Acid (CDCA) and CS-514

1988 ◽  
Vol 16 (3) ◽  
pp. 375-380
Author(s):  
Tadashi NAKAMURA ◽  
Yuji MATSUZAWA ◽  
Tadahisa NAKAJIMA ◽  
Hiroshi KAMIDO ◽  
Kaoru KAMEDA ◽  
...  
Keyword(s):  
Chenodeoxycholic Acid ◽  
Term Treatment ◽  
Cerebrotendinous Xanthomatosis ◽  
Long Term Treatment

Medical Consequences of Long-term Treatment of Acromegaly

2010 ◽  
Vol 06 ◽  
pp. 68
Author(s):  
Rosario Pivonello ◽  
Renata S Auriemma ◽  
Mariano Galdiero ◽  
Ludovica FS Grasso ◽  
Annamaria Colao ◽  
...  
Keyword(s):  
Signs And Symptoms ◽  
Premature Death ◽  
Term Treatment ◽  
Tumour Mass ◽  
Risk Of Death ◽  
Increased Risk ◽  
Long Term Treatment ◽  
A Minor ◽  
The Impact

This article discusses the impact of long-term treatment of acromegaly on cardiovascular, metabolic, respiratory and articular complications as well as on malignancies. The main goals of treatment of acromegaly include normalisation of biochemical markers of disease activity, improvement in signs and symptoms of the disease, removal or reduction of tumour mass and preservation of pituitary function, together with prevention of complications. Cardiovascular and respiratory complications are the main causes of morbidity and mortality, whereas neoplasms are a minor cause of increased risk of death. Other associated diseases are arthropathy, carpal tunnel syndrome and reproductive disorders. The prolonged elevation of growth hormone (GH) and insulin-like growth factor (IGF)-I levels results in premature death, whereas strong biochemical control improves wellbeing and restores life expectancy to normal.

Sustained drug delivery optimizes long-term treatment of patients with schizophrenia

2004 ◽  
Vol 16 (6) ◽  
pp. 319-325 ◽  
Author(s):  
Pierre S. Chue ◽  
Peter D'Hoore ◽  
J. Michael Ramstack
Keyword(s):  
Antipsychotic Agents ◽  
Term Treatment ◽  
Treatment Programs ◽  
Sustained Drug Delivery ◽  
Partial Compliance ◽  
Long Term Treatment ◽  
Optimal Maintenance ◽  
Long Acting ◽  
The Impact

Chronic disorders such as schizophrenia require long-term treatment programs in order to maintain patients at the lowest level of symptomatology, reduce the likelihood of psychotic relapse, and support achievement of remission and recovery. Evidence suggests that treatment with long-acting injectable antipsychotics reduces the impact of partial compliance and provides predictable release of medication, assuring continuous therapeutic coverage. Until recently, only conventional antipsychotic agents were available in long-acting formulations, thereby foregoing the advantages of the atypical class. Atypical agents which are given orally have been shown to provide long-term efficacy and tolerability benefits compared with conventional agents, but are limited by the need for daily administration. The most recent pharmacological strategy to achieve optimal maintenance treatment has been to combine the benefits of an atypical antipsychotic with delivery in a water-based long-acting formulation. The first antipsychotic to achieve this combination – long-acting risperidone – may thus represent an important advance in the optimization of long-term treatment outcomes in patients with schizophrenia.

The Impact of Disability Compensation on Long-term Treatment Outcomes of Patients With Sciatica Due to a Lumbar Disc Herniation

Spine ◽  
2006 ◽  
Vol 31 (26) ◽  
pp. 3061-3069 ◽  
Author(s):  
Steven J. Atlas ◽  
Yuchiao Chang ◽  
Robert B. Keller ◽  
Daniel E. Singer ◽  
Yen A. Wu ◽  
...  
Keyword(s):  
Treatment Outcomes ◽  
Lumbar Disc Herniation ◽  
Disc Herniation ◽  
Lumbar Disc ◽  
Term Treatment ◽  
Long Term Treatment ◽  
The Impact ◽  
Disability Compensation

scholarly journals Endoscopic and Histologic Predictors of Outcomes in Pediatric Ulcerative Colitis—Caveat Emptor

2021 ◽  
Vol 9 ◽  
Author(s):  
Lorraine Stallard ◽  
Séamus Hussey
Keyword(s):  
Ulcerative Colitis ◽  
Mucosal Healing ◽  
Term Treatment ◽  
Clinical Settings ◽  
Adult Disease ◽  
Long Term Treatment ◽  
Acute Severe Colitis ◽  
The Impact ◽  
Severe Colitis

The impact of endoscopic and histological mucosal healing on outcomes in adult settings is impressive. Despite many clinical parallels, pediatric ulcerative colitis (UC) is set apart from adult disease in several respects. Many frequently used indices are not fully validated, especially in pediatric settings, and consensus on precise definitions in clinical settings are lacking. Endoscopic mucosal healing is an acceptable long-term treatment goal in pediatrics, but not histologic normalization. Early prediction of disease course in UC may allow treatment stratification of patients according to risks of relapse, acute severe colitis, and colectomy. Putative endoscopic and histologic predictors of poor clinical outcomes in adults have not held true in pediatric settings, including baseline endoscopic extent, endoscopic severity, and specific histologic characteristics which are less prevalent in pediatrics at diagnosis. In this mini-review we appraise predictive endoscopic and histologic factors in pediatric UC with reference to relapse, severe colitis, and colectomy risks. We recommend that clinicians routinely use endoscopic and histologic sores to improve the quality of clinical and research practice. The review summarizes differences between adult and pediatric prediction data, advises special consideration of those with primary sclerosing cholangitis, and suggests areas for future study in this field.

scholarly journals Analysis of the impact of radiotherapy on long-term treatment results in patients with malignant salivary gland tumors

2016 ◽  
Vol 5 (4) ◽  
pp. 16-27
Author(s):  
V. T. Vayradyan ◽  
A. M. Mudunov ◽  
R. I. Azizyan ◽  
I. A. Zaderenko ◽  
S. B. Alieva ◽  
...  
Keyword(s):  
Salivary Gland ◽  
Term Treatment ◽  
Salivary Gland Tumors ◽  
Treatment Results ◽  
Long Term Treatment ◽  
The Impact ◽  
Malignant Salivary Gland Tumors

Multimethod Clinical Assessment of Patterns and Prevalence of Nonadherence (NA) to Imatinib Treatment (IMRx) in Patients (Pts) with Chronic Myeloid Leukemia (CML): Results from the ADAGIO Study.

Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 5163-5163
Author(s):  
Marie-Anne van Lierde ◽  
Filipo Serra ◽  
Larissa De Rop ◽  
Eric Strobbe ◽  
Karen MacDonald ◽  
...  
Keyword(s):  
Clinical Assessment ◽  
Structured Interview ◽  
Term Treatment ◽  
Pill Count ◽  
Assessment Tools ◽  
Imatinib Treatment ◽  
Open Label ◽  
Long Term Treatment ◽  
The Impact

Abstract BACKGROUND. ImRx for CML is a long-term treatment. Patterns and prevalence of NA to ImRx remain largely unknown. Short-term NA trends may be indicative of long-term NA. Methods for clinical NA assessment vary in reliability. A multimethod approach is indicated. OBJECTIVE. Multimethod estimation of patterns and prevalence of ImRx NA in CML pts at baseline (BL) and follow-up (FU) at 90 days (90d), incl. BL to 90d changes. DESIGN AND PATIENTS. Data subset from prospective, 90d observational, open-label, multicenter study. 169 evaluable pts on ImRx for minimum 30d at enrollment [1]. METHODS OF NA ASSESSMENT. At BL (NA with prior ImRx) and 90d (NA during study): visual analog scale (VAS) for physicians (phs; mVAS), pts (pVAS), cos (cVAS); Basel Assessment of Adherence Scale for pts (pBAAS; structured interview re NA in past 4 weeks [4wks]); pts reported persistence (pPST); % clinic appointments (%CAPPTS) kept (if any scheduled). At 90d also: % of ImRx taken per pill count (%pts@ImRx). RESULTS. See Table 1. CONCLUSIONS. Intuitive adherence ratings (VAS) by phs, pts, and cos are very high and differ from those from structured interview, where about one-third of patients exhibited NA behavior in 4wks prior to BL and FU - despite high persistence. Pill count suggests patterns of under- and overtaking, with only 1 out 7 patients being perfectly adherent. Rate of clinic appointments may be affected by physician scheduling practices and collateral input is a function of availability of collateral person. Consenting to participate in the ADAGIO study did not reduce NA. Though patient self-reports in structured interview (pBAAS) and pill counts have inherent biases, both indices suggest that NA with ImRx may be similar to NA rates in other disease categories. Especially pBAAS and pill count may be useful rapid clinical assessment tools, with pBAAS having the benefit of validated categorical assessment (vs. continuous in other methods). Determinants of NA and the impact of NA on treatment outcomes must be examined. Table 1 - Multimethod Assessment of Non-Adherence with Imatinib Method BL 90d n M±SD/Min-Max M±SD/Min-Max P mVAS 164 95.0±7.6/60–100 94.9±9.9/0–100 ns pVAS 169 95.3±8.5/25–100 95.7±6.1/75–100 ns cVAS 56 97.1±5.1/80–100 97.4±5.1/75–100 ns %pts@ImRx 162 - 91.0±21.1/29.5–2002.2 71.0% @ < 100% ImRx 14.2% @ 100% ImRx n % NA % NA P pBAAS 163 36.2% 32.5% ns %CAPPTS 51 94.1% 88.2% 0.001 pPST 163 98.8% 100.0% ns

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