Normal Quality Of Life Research Articles

Patient-centered benefit-risk analysis of transcatheter aortic valve replacement

Background: Aortic stenosis (AS) treatments include surgical aortic valve replacement (SAVR) and transcatheter aortic valve replacement (TAVR). Choosing between SAVR and TAVR requires patients to trade-off benefits and risks. The objective of this research was to determine which TAVR and SAVR outcomes patients consider important, collect quantitative data about how patients weigh benefits and risks, and evaluate patients’ preferences for SAVR or TAVR. Methods: Patients were recruited from advocacy organization databases. Patients self-reported as being diagnosed with AS, and as either having received AS treatment or as experiencing AS-related physical activity limitations. An online adapted swing weighting (ASW) method – a pairwise comparison of attributes – was used to elicit attribute tradeoffs from 93 patients. Survey data were used to estimate patients’ weights for AS treatment attributes, which were incorporated into a quantitative benefit-risk analysis (BRA) to evaluate patients’ preferences for TAVR and SAVR. Results: On average, patients put greater value on attributes that favored TAVR than SAVR. Patients’ valuation of the lower mortality rate, reduced procedural invasiveness, and quicker time to return to normal quality of life associated with TAVR, offset their valuation ofthe time over which SAVR has been proven to work. There was substantial heterogeneity in patients’ preferences. This was partly explained by age, with differences in preference observed between patients <60 years to those ≥60 years. A Monte Carlo Simulation found that 75.1% of patients prefer TAVR. Conclusions: Most AS patients are willing to tolerate sizable increases in clinical risk in exchange for the benefits of TAVR, resulting in a large proportion of patients preferring TAVR to SAVR. Further work should be undertaken to characterize the heterogeneity in preferences for AS treatment attributes. Shared decision-making tools based on attributes important to patients can support patients’ selection of the procedure that best meets their needs.

EPISPADIAS IN GIRLS

Purpose. To study the clinical picture, diagnosis and treatment of epispadias in girls. Material and methods. 22 girls with epispadias of various forms were under supervision for 50 years. During diagnostics the following issues were used: anamnesis, examination, catheterization and uroflowmetry, cystoscopy of the bladder, ultrasound and X-ray examination. Results and discussion. Out of 22 girls with epispadias, clitoral epispadia (partial ) was in 10 patients; sub-symphisal (subtotal) - in 4; symphisal (total) - in 8 girls. In 10 girls with the clitoral form, urological examination was made because of changes in the urine. Girls with sub-symphisal epispadia complained of irritation and itching in the vulva. All had vulvitis, cystitis, chronic pyelonephritis. In 2 patients, renal doubling was diagnosed; in other 2 patients ureterohydronephrosis and kidney dystopia were diagnosed. Uroflowmetry revealed hyperactive bladder in all. 8 girls with the total (symphisial) form of epispadia and urinary incontinence were operated by the Derzhavin technique; after the surgery the function of bladder sphincter was restored and the patients could have a normal quality of life. Conclusion. Epispadia in girls is a rare case . There are clitoral, sub-symphisal and symphisal (total) forms of epispadias. Cluster and sub-symphisial forms do not require surgical treatment. While in the symphisial (total) form, plastic surgery on the bladder neck by the Derzhavin technique is recommended. It is a good option for recovery.

Patient-centered benefit-risk analysis of transcatheter aortic valve replacement

Background: Aortic stenosis (AS) treatments include surgical aortic valve replacement (SAVR) and transcatheter aortic valve replacement (TAVR). Choosing between SAVR and TAVR requires patients to trade-off benefits and risks. The objective of this research was to determine which TAVR and SAVR outcomes patients consider important, collect quantitative data about how patients weigh benefits and risks, and evaluate patients’ preferences for SAVR or TAVR. Methods: Patients were recruited from advocacy organization databases. Patients self-reported as being diagnosed with AS, and as either having received AS treatment or as experiencing AS-related physical activity limitations. An online adapted swing weighting (ASW) method – a pairwise comparison of attributes – was used to elicit attribute tradeoffs from 93 patients. Survey data were used to estimate patients’ weights for AS treatment attributes, which were incorporated into a quantitative benefit-risk analysis (BRA) to evaluate patients’ preferences for TAVR and SAVR. Results: On average, patients put greater value on attributes that favored TAVR than SAVR. Patients’ valuation of the lower mortality rate, reduced procedural invasiveness, and quicker time to return to normal quality of life associated with TAVR, offset their valuation ofthe time over which SAVR has been proven to work. There was substantial heterogeneity in patients’ preferences. This was partly explained by age, with differences in preference observed between patients <60 years to those ≥60 years. A Monte Carlo Simulation found that 75.1% of patients prefer TAVR. Conclusions: Most AS patients are willing to tolerate sizable increases in clinical risk in exchange for the benefits of TAVR, resulting in a large proportion of patients preferring TAVR to SAVR. Further work should be undertaken to characterize the heterogeneity in preferences for AS treatment attributes. Shared decision-making tools based on attributes important to patients can support patients’ selection of the procedure that best meets their needs.

Physical Activity, Exercise Capacity and Quality of Life in Adolescent Heart Transplant Patients

Purpose The primary goal of pediatric heart transplantation (PHTx) is to improve the quality of life (QoL) of patients. National physical activity guidelines for adolescents recommend at least 60-90 minutes of moderate to vigorous physical activity per day. The Physical Activity Questionnaire (PAQ-C/A) for children and adolescents provides general estimates of physical activity levels. Measurement of peak aerobic capacity (VO2peak) using cardiopulmonary exercise testing (CPX) is the gold standard for assessing exercise capacity. Both physical activity and exercise capacity may be important factors that influence reporting a normal QoL. Our aim was to determine if there was a relationship between physical activity, exercise capacity, and QoL in a PHTx adolescent population. Methods This is a single centre retrospective chart review of adolescent (11-18 yrs) PHTx patients. Those who completed a maximal CPX test (RER>1.0), self-report physical activity (PAQ-C or PAQ-A) and QoL (PedsQL and PedsQL-Transplant) questionnaires, as part of their annual assessment, were eligible for inclusion. Results were compared to published age- and sex-matched healthy control data used in our laboratory. VO2peak and QoL were deemed to be normal if results were within two standard deviations of the mean. Pearson correlation coefficients were calculated. Medians and interquartile ranges are reported. Results Thirteen PHTx (10 males) completed a CPX and both QoL questionnaires. The median age was 14.9 yrs (12.3-17.5 yrs). VO2peak=34.5 mL/min/kg (30.2-43.4 mL/min/kg. Only 1 of 13 had an abnormal VO2peak. Total Peds QL score=84.8 (68.5-92.4) with 10 out 13 reporting a normal QoL. The Peds QL-Transplant score=90.5 (84.8-95.1). There was no significant correlation between VO2peak and QoL. The PAQ-C/PAQ-A was completed in 10 HTx. PAQ scores=2.38 (1.59-3.01). Only 2 of 13 met the cut-off point (>2.87) for adequate physical activity. There was a significant correlation between the Peds QL-Transplant QoL and the PAQ (r=0.69; p=0.04) Conclusion PHTx adolescents do not meet their required physical activity recommendations. Despite this, they have low normal exercise capacity and report a normal QoL. Efforts to engage adolescents to increase their physical activity should be encouraged.

The frequency of remission and low disease activity in patients with rheumatoid arthritis, and their ability to identify people with low disability and normal quality of life

ObjectiveTreat-to-target in rheumatoid arthritis (RA) recommends targeting remission, with low disease activity (LDA) being an alternative goal. When deciding to target remission or LDA, important considerations are the likelihood of attaining them, and their impacts on function and health-related quality of life (HRQoL). We have addressed this by studying: (a) the frequency of remission and LDA/remission; (b) DAS28-ESR trends after remission; (c) ability of remission vs. LDA to identify patients with normal function (HAQ ≤ 0.5) and HRQoL (EQ-5D ≥ the normal population). MethodsWe studied 571 patients in two clinical trials, and 1693 patients in a 10-year routine care cohort. We assessed the frequency and sustainability of remission and LDA/remission, variability in DAS28-ESR after remission, and sensitivity/specificity of remission and LDA/remission at identifying patients with low disability levels and normal HRQoL using Receiver Operator Characteristic (ROC) curves. Results:Point remission and remission/LDA were common (achieved by 35–58% and 49–74% of patients, respectively), but were rarely sustained (sustained remission and remission/LDA achieved by 5–9% and 9–16% of patients, respectively). Following attaining remission, DAS28-ESR levels varied substantially. Despite this, of those patients attaining point remission, the majority (53–61%) were in remission at study end-points. Whilst remission was highly specific at identifying patients with low disability (85–91%) it lacked sensitivity (51–57%); similar findings were seen for normal HRQoL (specificity 78–86%; sensitivity 52–59%). The optimal DAS28-cut-off to identify individuals with low disability and normal HRQoL was around the LDA threshold. ConclusionsOur findings support both the treat-to-target goals. Attaining remission is highly specific for attaining low disability and normal HRQoL, although many patients with more active disease also have good function and HRQoL. Attaining a DAS28-ESR ≤ 3.2 has a better balance of specificity and sensitivity for attaining these outcomes, with the benefit of being more readily achievable. Although sustaining these targets over time is rare, even attaining them on a one-off basis leads to better function and HRQoL outcomes for patients.

Decreased quality of life (QoL) in hypopituitary patients: involvement of glucocorticoid replacement and radiation therapy

Hypopituitary patients are assumed to have decreased QoL due to GHD. However, in placebo controlled trials, the effects of GH replacement are no different from placebo. Hydrocortisone dose > 20mg/day and pituitary radiation are independently associated with poorer QoL. We assessed QoL in panhypopituitary GH- deficient patients never treated with GH. Study group was divided into: (a) surgery followed by radiation (n = 21) and (b) surgery alone (n = 32). Mean duration of GHD was 71.4 ± 7.8 months and mean daily hydrocortisone dose was 15 ± 0.7mg. Control group had transnasal surgery for benign sinus conditions (n = 54). AGHDA scores were significantly worse in the entire study group compared to controls (8.1 ± 1.0 vs. 5.1 ± 0.9, p = 0.03). In patients with history of radiation therapy AGHDA scores were significantly worse than in controls (9.1 ± 1.5, p = 0.02) and SNOT-22 (Sino-Nasal Outcome Test) scores were also significantly worse (15.8 ± 2.0 vs. 23.2 ± 3.5, p = 0.04). However, AGHDA scores in patients without history of radiation and on "physiological" dose of hydrocortisone were similar to those in controls (5.1 ± 0.9 vs. 7.3 ± 1.3, p = 0.17). Replacement with hydrocortisone doses not exceeding 20mg/day and avoidance of radiation therapy was accompanied by normal QoL in patients not replaced with GH. Thus, we suggest that the decreased QoL in hypopituitary patients may not be due to GH deficiency per se, but rather to high hydrocortisone doses and to aftereffects of cranial radiation.

Long-Term Health Outcome and Quality of Life Post-HSCT for IL7Rα-, Artemis-, RAG1- and RAG2-Deficient Severe Combined Immunodeficiency: a Single Center Report.

Hematopoietic stem cell transplantation (HSCT) is curative for severe combined immunodeficiency (SCID), but data on long-term impact of pre-HSCT chemotherapy, immune reconstitution and quality of life (QoL) of specific SCID genotypes are limited. We evaluated the long-term immune-reconstitution, health outcome and QoL in IL7Rα SCID, Artemis and RAG1 and 2 SCID survivors > 2years post-HSCT in our center. Clinical data and immune reconstitution parameters were collated, and patients/families answered PedsQL generic core scale v4.0 questionnaires. Thirty-nine patients with a diagnosis of IL7Rα SCID (17 patients), Artemis SCID (8 patients) and RAG1/2 SCID (13 patients) had undergone HSCT with median age at last follow up for IL7Rα SCID, 14years (range 4-27) and Artemis and RAG1/2 SCID, 10years (range 2-18). Many patients have ongoing medical issues at latest follow-up [IL7Rα (73%), Artemis (85%), RAG1/2 (55%)]. Artemis SCID patients experienced more sequela than RAG1/2 SCID. Conditioned recipients with Artemis and RAG SCID had more CD4+ naïve lymphocytes compared to unconditioned recipients. All patients except those of IL7Rα SCID reported lower QoL; further subset group analysis showed parents and Artemis and RAG1/2 survivors without ongoing medical issues reported normal QoL. Conditioned recipients have superior long-term thymopoiesis, chimerism and immunoglobulin-independence. QoL was normal in those who did not have medical issues at long-term follow-up.

The Relationship Between Dialysis Patients' Quality of Life and Caregivers' Quality of Life.

Patients on dialysis require caregiving and assistance in their daily lives from family members and/or others for hospital visitation and supervised administration. This places a considerable burden on caregivers, which can in turn influence caregivers' quality of life (QOL). We recruited dialysis patients and their caregivers to elucidate how the QOL of patients relates to that of their caregivers'. Patients completed the EuroQol 5-Dimension scale (EQ-5D) and Kidney Disease Quality of Life-Short Form. Caregivers completed the EQ-5D and Medical Outcomes Study 36-Item Short-Form Health Survey (SF-36). We calculated utility index values for the EQ-5D, and physical, mental (MCS), and role-social component summary scores for the SF-36. Compared to national norms, the caregivers of dialysis patients tended to have poor physical health-related QOL but normal mental health-related QOL, as also found with patients. The multivariate analysis revealed that ≥ median dialysis period and ≥ average burden of kidney disease were significantly related to caregiver MCS score (odds ratios; 6.79 and 9.89, respectively). Caregivers tended to have lower physical health-related QOL if their patients had high social QOL, and lower mental health-related QOL during the early stage of the patient's dialysis treatment, and when patients experienced low disease-targeted QOL.

Continuous Clinical Response Is Associated With a Change of Disease Course in Patients With Moderate to Severe Ulcerative Colitis Treated With Golimumab

Responders to induction treatment sustain continuous clinical response (CCR) through 1 year in about 50% of patients in PURSUIT-M trial with golimumab maintenance in ulcerative colitis (UC). This post hoc analysis of PURSUIT-M describes the 1-year clinical, endoscopic, quality of life (QoL), and biomarker and 4-year clinical outcome in patients with sustained response to golimumab therapy for UC. We compared clinical, endoscopic, QoL, and calprotectin outcomes in CCR and non-CCR patients through 54 weeks in PURSUIT-M. Persistence on golimumab therapy and clinical response at 4 years was assessed for CCR and non-CCR patients. The relationship of colectomy with CCR status was determined. Among patients receiving golimumab maintenance, greater proportions of patients with vs without CCR at week 54 achieved clinical remission (67.1% vs 1.9%), corticosteroid-free remission (61.6% vs 1.9%), endoscopic remission (Mayo endoscopy score 0 [47.9% vs 1.3%]), and normal QoL (inflammatory bowel disease questionnaire score ≥170 [75.0% vs 24.4%]). CCR but not non-CCR patients maintained normalized calprotectin levels during maintenance. Among patients who entered the long-term extension study, a greater proportion of patients with vs without CCR maintained PGA 0 through week 216 (58% vs 42%). Colectomy was performed in 47 induction nonresponders and in 13 induction responders. None of the patients going onto colectomy achieved CCR through 54 weeks in PURSUIT-M. Continuous clinical response is associated with favorable short- and long-term clinical, endoscopic, QoL, and biomarker responses that may result in changing the course of disease and may prevent colectomy in patients with moderate to severe UC treated with golimumab. 10.1093/ibd/izy229_video1izy229.video15806022773001.

Quality of Life and Psychosocial Functioning 2 Years Following Facial Transplantation

Face transplantation is a novel option for patients with severe facial disfigurement. Quality of life (QoL) outcomes of face transplantation remain poorly understood. We sought to evaluate psychosocial functioning among 6 patients undergoing facial transplantation. We prospectively assessed depressive symptoms, health status, mental and physical QoL, and self-esteem at 3-month intervals for 2 years. Social desirability was assessed pretransplant. On average, before transplantation, patients generally reported minimal to subthreshold depressive symptoms, normal to high health status, normal mental-health QoL, slightly below normal physical-health QoL, and normal to high self-esteem. Most endorsed high social desirability. As patients recovered from surgery, hospitalization, and immunosuppression induction, physical-health QoL generally deteriorated 3 months posttransplantation. Posttransplant trajectories show that perceived health state improved; health status and mental and physical health-related QoL slightly improved; self-esteem remained stable and high; and overall depressive symptoms remained stable but 3 patients experienced a depressive episode. Pretransplant ceiling effects may render improvements difficult to quantify. Future research should use mixed methods including population-specific measures with demonstrated sensitivity to change.

Identification of Endpoints for Development of Antifibrosis Drugs for Treatment of Crohn’s Disease

Intestinal fibrosis is a challenge to management of patients with Crohn's disease (CD); there is an urgent need to expedite development of antifibrosis drugs for this disease. The International Organization for the Study of Inflammatory Bowel Disease (IOIBD) aimed to identify a set of endpoints that can be used to determine efficacy of antifibrosis agents tested in clinical trials of patients with CD. We conducted a systematic review to identify clinical, radiologic, biochemical, endoscopic, and composite endpoints used in assessing activity of fibrostenosing CD and response to treatment, and determined their operational properties. A panel of IOIBD experts performed a consensus process to identify the best endpoints for inclusion in clinical trials, through a 2-round, Delphi-style online survey. A total of 36 potentially relevant endpoints for intestinal fibrosis were selected and assessed. Forty-eight physicians with expertise in inflammatory bowel disease, from 5 regions (North America, Europe, Middle East, Asia/Pacific, and Latin America), participated in the Delphi consensus process. A core set of 13 endpoints (complete clinical response, long-term efficacy, sustained clinical benefit, treatment failure, radiological remission, normal quality of life, clinical remission without steroids, therapeutic failure, deep remission, complete absence of occlusive symptoms, symptom-free survival, bowel damage progression, and no disability) were rated as critical. Agreement was high among the experts. Members of the IOIBD reached expert consensus on a set of endpoints that can be used to assess antifibrosis agents in trials of patients with CD. Studies are needed to clarify methods for measuring these outcomes and validate measurement instruments.

Hypothalamic abnormalities: Growth failure due to defects of the GHRH receptor

Several acquired or congenital hypothalamic abnormalities may cause growth failure (GF). We described two of these congenital abnormalities. First, a case of CHARGE syndrome, an epigenetic disorder mostly caused by heterozygous mutations in the gene encoding CHD7, a chromatin remodeling protein, causing several malformations, some life-threatening, with additional secondary hypothalamus-hypophyseal dysfunction, including GF. Second, a cohort of individuals with genetic isolated severe GH deficiency (IGHD), due to a homozygous mutation in the GH-releasing hormone (GHRH) receptor gene described in Itabaianinha County, in northeast Brazil. In this IGHD, with marked reduction of serum concentrations of IGF-I, and an up regulation of IGF-II, GF is the principal finding in otherwise normal subjects, with normal quality of life and longevity. This IGHD may unveil the effects of GHRH, pituitary GH and IGF-I, IGF-II and local GH and growth factor on the size and function of body and several systems. For instance, anterior pituitary hypoplasia, and impairment of the non-REM sleep may be due to GHRH resistance. Proportionate short stature, doll facies, high-pitched pre-pubertal voice, and reduced muscle mass reflect the lack of the synergistic effect of pituitary GH and IGF-I in bones and muscles. Central adiposity may be due to a direct effect of the lack of GH. Brain, eyes and immune system may also involve IGF-II and local GH or growth factors. A concept of physiological hierarchy controlling body size and function by each component of the GH system may be drawn from this model.

Impact of Sleep-Disordered Breathing Treatment on Quality of Life Measures in a Large Clinic-Based Cohort.

This study aims to report the effect of positive airway pressure (PAP) therapy on quality of life (QoL) measures in the clinical sleep-disordered breathing (SDB) population. We examined general QoL measures assessed by European Quality of Life-5D (EQ-5D) and sleep-specific QoL by examining Functional Outcomes of Sleep Questionnaire (FOSQ) scores before and after PAP therapy retrospectively in a clinical SDB population using paired and two-sample t tests. Age and socioeconomic status (SES) effect modification on pre-PAP QoL measures were investigated utilizing the interaction terms. A total of 2,027 patients with SDB initiated PAP therapy between January 1, 2010 and December 31, 2014. The mean age of the cohort was 56.2 years (standard deviation = 13.2), with 45.8% female and 76.9% Caucasians. EQ-5D change after exclusion of those with normal QoL was 0.042 (0.152) in all patients, 0.051 (0.150) in patients who were PAP adherent by self-report, and 0.050 (0.132) in patients who were objectively PAP adherent (n = 704 of 1,011 with available objective adherence data, 69.6%). Change in FOSQ after excluding those with normal FOSQ was 1.9 (2.9) in all patients, 2.2 (2.9) in patients who were PAP adherent by self-report, and 2.3 (2.9) in patients who were objectively PAP adherent. Those with (1) worse QoL at baseline and younger age and (2) worse QoL at baseline and residing in lower SES strata had worse outcomes after PAP therapy (P < .05). We found consistent improvement in global and sleep-specific QoL measures after PAP therapy, hence providing evidence of PAP benefit in the clinical population and rationale for targeted efforts to optimize QoL in younger and lower SES subgroups.

Body composition and clinical symptoms in patients with hip or knee osteoarthritis: Results from the KHOALA cohort.

To analyze the associations between body composition, notably low lean mass, and clinical symptoms [pain, physical function, quality of life (QoL)] in patients with symptomatic hip and/or knee OA. Cross-sectional study using data from the 3-year follow-up visit of the Knee and Hip OsteoArthritis Long-term assessment (KHOALA) cohort. Skeletal muscle and fat mass were measured by dual X-ray absorptiometry (DXA). Fat mass index (FMI) was defined as total fat mass/height2. Appendicular lean mass was adjusted on body mass index (ALM/BMI), and low lean mass was defined according to the definition of FNIH Sarcopenia Project recommendations. Pain and function were measured by the WOMAC index and QoL by the SF-36. In total, 358 patients underwent DXA (67% women, mean [SD] age 63.4 [8.4] years, mean BMI 29.5 [5.6]kg/m2). The visual analog scale (0-100) pain score was 38.0 [24.7] and 25.4% had hip and 74.6% knee OA. Low lean mass and ALM/BMI were associated with impaired QoL and WOMAC scores on bivariate analysis (all p ≤ 0.001) but not on multivariate analysis after adjustment for FMI. For patients with normal BMI, mean [SD] WOMAC scores were higher (greater impairment) with low lean mass than normal body composition (WOMAC function 33.4 [23.3] and 24.0 [17.4], p = 0.02), and mean SF-36 physical component score was lower (greater impairment) 40.3 [10.2] and (44.3 [8.4], p = 0.04). Among patients with obesity, low lean mass had no additional effect. For patients with OA and normal BMI, QoL and function were more impaired for those with than without low lean mass. Conserving muscle mass in people with OA could have functional and antalgic benefits especially for those with normal BMI.

Surgical options in the treatment of ulcerative colitis

Children and young adults with ulcerative colitis tend to present with more extensive colonic disease than an adult population. The need for surgical intervention in the pediatric population with ulcerative colitis occurs earlier after diagnosis and has a greater incidence than a comparably matched adult population with an estimated need for colectomy at 5 years following diagnosis of 14–20%. Perhaps, even more than the adult population, there is a desire to restore intestinal continuity for the pediatric patient to achieve as healthy and normal quality of life as possible. With surgery playing such a prominent role in the treatment of ulcerative colitis in this age group, an understanding of the surgical treatment options that are available is important. The surgeon’s awareness of the complexities of the different operations associated with proctocolectomy and reestablishing intestinal continuity may help to avoid early complications and minimize the risk of less than ideal long-term outcomes.

Is it possible to preserve the blood supply of erectile organs by anastomosis using the hypogastric artery in kidney transplantation?

IntroductionThree modalities for treating chronic kidney failure are peritoneal dialysis, hemodialysis, and kidney transplantation. Among them kidney transplantation is cost-efficient and leads to a somewhat normal quality of life. In this approach, most often the external iliac artery is selected for anastomosis, but this could be disastrous if anastomosis leads to a complication. The traditional end-to-end approach for anastomosis of the kidney artery to the internal iliac artery leads to pelvic organ ischemia. However, if the end-to-end anastomosis is replaced by an end-to-side approach, it is safer. This report discusses some cases of end-to-side anastomosis using the internal iliac artery.MethodIn ten cases of chronic kidney failure, we anastomosed the kidney artery to the internal iliac artery with an end-to-side approach.ResultsAfter vessels were unclamped, all patients had diuresis. Their creatinine was in normal range and was blood flow in the internal iliac artery, based on color Doppler ultrasound.ConclusionEnd-to-side anastomosis can be done in some chronic kidney failure patients if their internal iliac arteries are large enough. This approach is safer than anastomosis using the external iliac artery.

Menopause-related symptoms and quality of life in peri- and postmenopausal women in Thailand: a multicenter study

Background: Menopausal symptoms can affect the quality of life (QoL) of women. Objectives: To determine menopause-related symptoms and QoL in peri- and postmenopausal women in Thailand undergoing various treatment. Methods: A prospective cohort study was conducted at 9 menopause clinics in 4 regions of Thailand. Peri- and postmenopausal women newly registered at the clinics were recruited and followed up for 12 months. A standardized medical record form was used to collect data regarding demographic characteristics, menopause-related symptoms, and treatment. QoL was evaluated using a specific questionnaire. Results: We included 870 women who were 51.8 ± 5.6 years old. We compared demographic characteristics and baseline health profiles of women with normal QoL and impaired QoL. Women with impaired QoL were significantly younger (51.2 ± 4.4 vs. 52.0 ± 6.0 y, P = 0.035) and were disproportionately from northeast Thailand (11.4% vs. 6.6%, P = 0.022). The 3 most common menopause-related symptoms were psychoemotional distress, musculoskeletal pain, and hot flushes. All QoL scores improved with time in both users and nonusers of postmenopausal hormone therapy (pHT), and the scores of both groups converged during months 3 to 12 of the follow-up period. Conclusions: The most common menopause-related symptom in peri- and postmenopausal women in Thailand is psychoemotional distress. QoL scores of women with moderate-to-severe menopause-related symptoms at baseline improve over time, whether or not they use of pHT. The pHT users had poorer QoL at baseline than nonusers, but their QoL improves and matches that of their peers after 3 months. Keywords: Hormonal therapy, menopause-related symptoms, quality of life

Extrahepatic Portal Venous Obstruction: What Should be the Mainstay of Treatment?

The two cornerstones of management for Extrahepatic portal vein obstruction (EHPVO) are endotherapy and surgery [Porto-systemic shunts (PSS)/Mesorex bypass (MRB)]. Endotherapy is the mainstay of treatment for acute variceal bleed control and has also been used extensively for secondary prophylaxis till variceal eradication is achieved. However, long-term follow-up beyond endoscopic eradication of esophageal varices (EEEV) indicates that there are numerous delayed bleed and non bleed sequelae of EHPVO, which merit surgery as a definitive procedure to decompress the hypertensive portal venous system. While endotherapy obliterates natural porto-systemic collaterals in the gastroesophageal region, persistently raised portal pressures manifest as an increase in secondary isolated gastric varices, ectopic varices, portal hypertensive vasculopathy, issues related to massive splenomegaly, portal biliopathy, growth retardation and hence impaired quality of life (QOL). An ideal management strategy should address both bleed and non-bleed consequences of EHPVO and translate into a near normal QOL. Further, MRB has opened up new dimensions to the management philosophy of EHPVO. This review article critically evaluates the role of surgery and endotherapy based on available literature and authors' own experience.Surgery and endotherapy are complementary. However, with increasing duration of follow-up post EEEV, it is evident that there is resurgence in the role of surgery (PSS/MRB) as a single one time definitive procedure for alleviating all bleed and delayed non bleed sequelae of EHPVO.Surgery for EHPVO (PSS/MRB) should not be allowed to become a dying art and future generations of surgeons should continue to receive training in this specialized area of surgery.

Altered sleep patterns in patients with non-functional GHRH receptor.

GH-releasing hormone (GHRH) exerts hypnotic actions increasing the non-rapid eye movement (NREM) sleep. Conversely, GH stimulates the REM sleep. GH deficiency (GHD) often leads to sleep problems, daytime fatigue and reduced quality of life (QoL). GHD may be due to lack of hypothalamic GHRH or destruction of somatotroph cells. We have described a cohort with isolated GHD (IGHD) due to GHRH resistance caused by a homozygous null mutation (c.57 + 1G > A) in the GHRH receptor gene. They have normal QoL and no obvious complaints of chronic tiredness. The aim of this study was to determine the sleep quality in these subjects. A cross-sectional study was carried out in 21 adult IGHD subjects, and 21 age- and gender-matched controls. Objective sleep assessment included polygraphic records of the awake, stages NREM [N1 (drowsiness), N2 and N3 (already sleeping)] and REM (R). Subjective evaluation included the Pittsburgh Sleep Quality Index, the Insomnia Severity Index and the Epworth Sleepiness Scale. IGHD subjects showed a reduction in sleep efficiency (P = 0.007), total sleep time (P = 0.028), duration of N2 and R in minutes (P = 0.026 and P = 0.046 respectively), but had increased duration and percentage of N1 stage (P = 0.029 and P = 0.022 respectively), wake (P = 0.007) and wake-time after sleep onset (P = 0.017). There was no difference in N3 or in sleep quality questionnaire scores. Patients with IGHD due to GHRH resistance exhibit objective reduction in the sleep quality, with changes in NREM and REM sleep, with no detectable subjective consequences. GHRH resistance seems to have a preponderant role over GHD in the sleep quality of these subjects.

Long-term outcome of hematopoietic stem cell transplantation for IL2RG/JAK3 SCID: a cohort report

AbstractHematopoietic stem cell transplantation (HSCT) cures the T-lymphocyte, B-lymphocyte, and natural killer (NK)–cell differentiation defect in interleukin-2 γ-chain receptor (IL2RG)/JAK3 severe combined immunodeficiency (SCID). We evaluated long-term clinical features, longitudinal immunoreconstitution, donor chimerism, and quality of life (QoL) of IL2RG/JAK3 SCID patients >2 years post-HSCT at our center. Clinical data were collated and patients/families answered PedsQL Generic Core Scale v4.0 questionnaires. We performed longitudinal analyses of CD3+, CD4+ naive T-lymphocyte, CD19+, and NK-cell numbers from pretransplant until 15 years posttransplant. Thirty-one of 43 patients (72%) survived. Median age at last follow-up was 10 years (range, 2-25 years). Twenty-one (68%) had persistent medical issues, mainly ongoing immunoglobulin replacement (14; 45%), cutaneous viral warts (7; 24%), short stature (4; 14%), limb lymphoedema (3; 10%), and bronchiectasis (2; 7%). Lung function was available and normal for 6 patients. Longitudinal analysis demonstrated sustained CD3+, CD19+, and NK-cell output 15 years post-HSCT. CD4+ naive lymphocyte numbers were better in conditioned vs unconditioned recipients (P, .06). B-lymphocyte and myeloid chimerism were highly correlated (ρ, 0.98; P < .001). Low-toxicity myeloablative conditioning recipients have better B-lymphocyte/myeloid chimerism and are free from immunoglobulin replacement therapy. IL2RG/JAK3 SCID survivors free from immunoglobulin replacement have normal QoL.