Kornimate/medical-research-clean huggingface dataset:
long term beta thalassemia treatment: findings from the extension period

Conditions: Fetal Hemoglobin; Beta-Thalassemia; Hemoglobinopathies

Summary: the project, titled "long term beta thalassemia treatment: findings from the extension period of phase 2 clinical trial," aims to compare the efficacy and safety of combination therapy (thalidomide and hydroxyurea) versus thalidomide alone. the study, lasting three years, is a phase 2 single-center, open-label interventional study with a sample size of 30 participants aged 8-35 years. it includes specific inclusion and exclusion criteria for participant selection. data will be collected through clinical interviews and medical records and analyzed using(statistical package for the social sciences. this project aims to enhance beta thalassemia treatment strategies, focusing on reducing transfusion dependency and improving patient quality of life.

study titled long term beta thalassemia treatment: findings from the extension period of phase 2 clinical trial, conducted at the national institute of blood disease \& bone marrow transplantation (nibd \& bmt). this study focuses on the long-term comparison of combination therapy (thalidomide and hydroxyurea) versus thalidomide alone in treating beta thalassemia. the objective is to evaluate the efficacy and safety of the combination therapy compared to thalidomide alone, with the hypothesis that the combination will be more effective. beta thalassemia is defined as an autosomal recessive disorder affecting beta-globin production, influenced by genetic modifiers. key variables include hemoglobin, red blood cells, leukocyte count, reticulocyte count, platelets, lactate dehydrogenase, nucleated red blood cells, ferritin, bilirubin, serum glutamate pyruvate transaminase, creatinine, transfusion frequency, spleen and liver size, hemoglobin subunit beta \[ homo sapiens (human) \] mutation, and certain polymorphism in gamma globin gene . the study took place at nibd hospital over three years, designed as a phase 2 single-center, two-arm open-label interventional study with a sample size of 30 participants using simple randomized sampling. inclusion criteria are beta thalassemia major/intermediate patients aged 8-35 years, while exclusion criteria include patients with liver dysfunction, married patients, lactating mothers, and those with a history of thrombosis and fits. data will be collected through clinical interviews and medical record reviews and analyzed using (statistical package for the social sciences.

Eligibility criteria:
inclusion criteria: * known case of beta thalassemia major/intermediate (transfusion dependent) * willing to give informed consent exclusion criteria * patients with comorbidities such as liver dysfunction * married patients * lactating mothers * history of thrombosis and fits

Keywords: thalidomide safety and efficacy thalassemia fetal hemoglobin inducer hydroxyurea
multistate relative survival model

Conditions: Colorectal Cancer

Summary: colorectal cancer (crc) has high incidence and is associated with high case fatality. in france, the 5-year survival, pooled across all cancer stages at diagnosis, ranges from 57% in men to 60% in women. about one third of patients diagnosed with crc will develop a metachronous recurrence during the following years. it is of paramount importance to accurately identify factors associated with the increased risk of progression and death, in order to develop effective follow-up and treatment strategies. however, to accurately assess the role of patients' specific characteristics in the progression of cancer several methodological challenges need to be overcome. one difficulty, common to prognostic studies of cancer, concerns the need to separate the effects of prognostic factors on different clinical endpoints, such as disease recurrence vs recurrence-free death. another difficulty, encountered in prognostic studies, is that the cause of death is not available or not accurately coded. yet, some patients are likely to die of causes not related to the disease of primary interest, especially in cancers with longer survival and in those that affect older subjects. until recently, the existing statistical methodology was not able to simultaneously, deal with both difficulties, i.e. to account for (i) possibly different effects of prognostic factors on death vs recurrence, and (ii) unknown causes of death. however, this challenge has been addressed by the recent development of the markov relative survival model (mrs) , which extends the markov multi-state model to incorporate relative survival modelling. simulations demonstrate that mrs is able to accurately estimate different effects of prognostic factors on the risk of each of several events, including separate effects on disease-specific vs other causes of death. to date, the mrs had not been applied in clinical or epidemiological studies. the aim of this study was to assess the potential advantages of the new multi-state relative survival model (mrs), proposed by huszti et al. (2012), in a prognostic cancer study. to this end, we compared the mrs results with those obtained with two more conventional analyses, based on cox's proportional hazards model, and the multi-state markov model proposed by alioum and commenges (2001). the three models were applied to explore the impact of prognostic factors on cancer-specific mortality and recurrence, in a large population-based french registry of colorectal cancer, with up to 25 years of follow-up.


Eligibility criteria:
inclusion criteria: patient with primary digestive cancer between 1985 and 2000, tnm i-iii, resected for cure, residing in burgundy or calvados, france exclusion criteria: metastatic cancer, non adenocarcinoma, recurrence within 6 month
m-mode ultrasonography in the assessment and treatment of pelviperineal dysfunctions (perineum)

Conditions: Pelvic Floor Disorders

Summary: objectives: to analyze the mode m ultrasound use in the assessment of pelvic floor musculature (pfm) and in the rehabilitation of motor control in pelviperineal dysfunctions that affect the support of pelvic organs, urinary and fecal continence and reproductive and sexual functions. methodology: a cross-sectional descriptive study will be followed by a randomized clinical trial. there will be two samples: a sample of volunteers without pfm dysfunction; and a sample of patients with pelviperineal dysfunction derived from health centers in the region of murcia. an intra- and interobserver reliability study of transabdominal m-mode ultrasound of pfm will be performed. then, a cross-sectional descriptive study of the functional behavior of pfm will be used in different functional situations such as: voluntary contraction at maximum force, fast contraction, valsalva maneuver and forced cough. the following ultrasound variables will be measured: direction and amount of displacement, speed of displacement and time of contraction. correlations between ultrasound variables and sociodemographic and clinical variables will be analyzed. the randomized clinical trial will compare a pfm contraction awareness treatment based on endocavitary digital contact with a motor learning program based on transabdominal mode m ultrasound biofeedback.


Eligibility criteria:
inclusion criteria: * patients with pelvic floor dysfunction derived from the pelvic floor unit of hla la vega in the city of murcia diagnosed by the doctor and without previous training in pelvic floor muscles contraction. exclusion criteria: * pregnancy, a history of pelvic floor surgery in the previous year, neurological injury with affectation of the sacral reflex from s2 to s4, previous delivery in a period of less than 6 weeks or known or suspected urinary tract infection.
a phase 2, dose finding study of pf-03187207 in patients with primary open angle glaucoma or ocular hypertension.

Conditions: Primary Open Angle Glaucoma; Ocular Hypertension

Summary: to investigate dose response, safety and efficacy of pf-03187207 in patients with primary open-angle glaucoma or ocular hypertension


Eligibility criteria:
inclusion criteria: * man or woman at least 20 years of age * diagnosis of primary open-angle glaucoma or ocular hypertension in one or both eyes. exclusion criteria: * closed/barely open anterior chamber angle or a history of acute angle closure in either eye * contraindications to latanoprost and nitric oxide treatment * known latanoprost non-responders
use of pet-ct in the management of crohn's disease

Conditions: Crohn's Disease

Summary: crohn's disease (cd) is an autoimmune disorder which affects over 700,000 people in the u.s. the disease can vary in severity and multiple drug classes are available to the disease depending on disease severity and complications. our purpose will be to study how pet-ct scans can be used to better manage crohn's disease.

crohn's disease (cd) is a gastrointestinal autoimmune disease thought to result from exposure to luminal antigen. this exposure then results in a robust th1 mediated immune response which is not downregulated, resulting in intestinal tissue injury. crohn's disease most commonly involves the distal small intestine, a site that can be difficult to visualize given its central location within the gastrointestinal tract. a substantial proportion of patients do not respond to any of the standard therapies. over 40% of patients do not respond to an initial anti-tnf (biologic) agent and approximately 10% per year lose their response to these drugs. there is no standard definition of what constitutes a patient who has primary biologic failure. disease activity is measured by indices such as the crohn's disease activity index (cdai). the cdai includes a combination of clinical criteria and lab values. however, the cdai is limited by reliance on subjective criteria, limiting the score's use as a valid measure of studying response to medical therapy. clinically, reliance on cdai can result in inappropriate continuation of an ineffective therapy resulting in worsening of underlying disease, and increasing risk of adverse reactions. small studies have demonstrated that pet scans may accurately quantify disease activity in crohn's. we propose to study cd patients with active flares to assess for a correlation between pet activity and cdai. additionally, we will determine whether pet signal intensity is predictive of clinical response to therapy with biologic agents. the study proposes to test the hypothesis that pet can be used to determine which cd patients with small bowel disease will be true responders (or nonresponders) to biologic therapy at week 12. the use of pet to determine response to biologic therapy in small bowel crohn's disease would be novel.

Eligibility criteria:
inclusion criteria: * diagnosis of crohn's disease * cdai1\>220 * identification of small bowel disease based on an imaging study (ct scan, abdominal ultrasound, mri), colonoscopy, or histopathology 4. need for initiation of biologic therapy to control disease process. exclusion criteria: * abdominal surgery within 8 wks of study entry * change in dose of steroids or immunomodulators within 2wks of study entry * pregnancy * active bacterial infection or undrained abscess * any contraindications to initiation of antitnf therapy (i.e. latent tuberculosis).

Keywords: inflammatory bowel disease, pet scan, crohn's disease
vitamin d and chronic obstructive lung disease

Conditions: Chronic Obstructive Lung Disease

Summary: the purpose of this study is to examine the effect of vitamin d intervention in patients with chronic obstructive pulmonary disease (copd) undergoing rehabilitation.


Eligibility criteria:
inclusion criteria: * age ≥ 50 years * diagnosed copd with forced expiratory volume in 1 second (fev1) \< 50 % of predicted * breathlessness relative to mrc dyspnoea grade 3 or more exclusion criteria: * patients with known diseases of bone metabolism apart from osteoporosis and patients with granulomatous disease * patients with hyper- or hypocalcemia at inclusion * patients with cardiovascular or musculoskeletal disease that impacts significantly on walking based on the investigator's judgement * patients with dementia or other mental conditions rendering them unable to understand the study information and thus provide informed consent * patients with terminal disease or known cancer with bone metastases, increasing their risk of hypercalcemia * patients taking more than 20 mcg vitamin d on a daily basis. if a patient takes vitamin d below this limit, dose should have been stable 6 months prior to inclusion in order to achieve steady-state. * pregnancy

Keywords: copd vitamin d pulmonary rehabilitation
assessment of home tonometry in glaucoma

Conditions: Glaucoma; Intraocular Pressure

Summary: worldwide, glaucoma is the most common cause of irreversible blindness. the major risk factor is raised pressure within the eye (intraocular pressure, iop). iop is typically measured using goldman applanation tonometry (gat). this involves instilling anaesthetic eye drops and using a probe to contact the cornea and obtain a pressure measurement. a major disadvantage of gat is that only limited numbers of pressure measurements can be obtained, whereas iop is known to vary throughout the day and in ¾ of people is highest during the night. the result is that clinicians have poor understanding of patient's 24 hour iops and the effect that this might have on their glaucoma. this is a serious limitation as the treatment of glaucoma depends on lowering iop. recently a new device has become available, designed specifically for home iop monitoring. the icare home tonometer is a ce marked commercial device (http://www.icaretonometer.com/products/icare-home-tonometer/) that can be used to gain insight into 24-hour iop fluctuations. the investigators wish to determine the feasibility of home iop monitoring using this device. the icare home tonometer depends on the patient performing self-tonometry. testing one's own iop may be technically difficult and it is likely that large numbers of patients will struggle with this.


Eligibility criteria:
inclusion criteria: * glaucoma * at least 4 previous visual field tests (to enable rate of change to be calculated). * age 16 years and above * age less than 100 exclusion criteria: * corneal disease (potentially affects pressure measurements) * neovascular glaucoma * uveitis * secondary glaucomas * inability to give informed consent. * less than 16 years of age. * pregnancy. * unable to hold icare home® tonometer due to upper limb weakness or disability.
optimization of skin preparation to reduce cutibacterium acnes colonization in superficial and deep samples during prosthetic shoulder surgery in male patients

Conditions: Cutibacterium Acnes Infection

Summary: infections on joint replacements remain one of the most serious complications of orthopaedic surgery. despite improvements in skin preparation and antibiotic prophylaxis procedures, the risk of infection of joint prosthesis is still high, particularly for shoulder prostheses, especially in men. one of the bacteria most often involved in post-operative infections for shoulder prosthesis is cutibacterium acnes (ca). this bacterium is one of the predominant bacteria in pilosebaceous units. eradication of this micro-organism remains difficult despite the techniques used in the preoperative phase. the objective of this protocol is to evaluate the efficacy of applying a 5% benzoyl peroxide topical during the 5 days preceding the procedure in the surgical skin incision area, which is based on local acne treatment, on the reduction of the ca bacterial load in the dermis of the approach to shoulder arthroplasty in men.


Eligibility criteria:
inclusion criteria: * male patients, adult patients, * patient without a history of surgical intervention on the operated shoulder, * patient managed for glenohumeral, partial (cup or anatomical hemi- arthroplasty) or total (anatomical or inverted) glenohumeral arthroplasty placement for primary osteoarthritis, or secondary to necrosis, massive rotator cuff tear or old fracture. * patients affiliated to a social security system * patients who have given their informed and written consent exclusion criteria: * patient with arthroplasty for resumption of previous shoulder surgery or failure of arthroplasty * patient with arthroplasty for acute trauma * patient on immunosuppressantsimmunosuppressive drugs * patient with inflammatory rheumatism * patient with progressive cancer pathology * allergies or intolerances concerning the modalities of selected skin preparations (povidone iodine, hypersensitivity to benzoyl peroxide) * dermatological pathologies in the area to be treated * acne treatment in the area to be treated within four weeks before inclusion * patient without possible help from a third party for the application of cutacnyl® 5% in the intervention group if necessary * major under guardianship * patient under the protection of justice * patient not covered by social security
the aging brain answers program

Conditions: Traumatic Brain Injury; Alzheimer Disease; Veterans; Family Caregivers; Caregiver Burden; Quality of Life

Summary: this study will test the effectiveness of an intervention for veterans diagnosed with alzheimer's disease (ad) or traumatic brain injury (tbi) and the burden on their informal (family/friend) caregiver.

the majority of people with alzheimer's (ad) and traumatic brain injury (tbi) are cared for in primary care settings and receive significant amounts of care and assistance in activities of daily living from family members or friends. the abc answers intervention will incorporate and integrate the common features of an evidence-based collaborative care model for brain care, while also attending to the implementation barriers of delivering care and skills to dyads of patients with ad and tbi and their family caregivers. the study team will conduct a 1-year randomized control trial (rct) designed to improve the quality of life (qol) of veterans with ad and tbi, and their family caregivers, and decrease their family caregivers' burden. the dyads in the intervention group will receive primary care plus abc answers. the control group will receive primary care.

Eligibility criteria:
inclusion criteria: * veterans: 1)diagnosis of ad or tbi 2) receives primary care from veterans administration medical center (vamc) * caregivers: 1) primary caregiver (family or friend) of a veteran with ad or tbi 2) plans to provide care for 1 year or longer * both: 1) over 18 years of age 2) speak and understand english 3) access to telephone exclusion criteria: * veterans: 1) resides in nursing home or long term care facility 2) currently receiving care in grace clinic 3) enrolled in answers-va research study * caregivers: 1) does not consider him/herself a caregiver for veteran 2) serious medical illness limiting ability to participate * both: unable to contact within 10 calls age \< 18 years either caregiver or veteran denies that they have ad or a tbi either the caregiver or veteran does not want to participate not fluent in the english language difficulty hearing or talking by telephone has a terminal illness (e.g., cancer, end of life condition with decreased life expectancy, renal failure requiring dialysis) history of hospitalization for alcohol or drug abuse history of severe mental illness (e.g., suicidal tendencies, severe untreated depression or bipolar disorder, or schizophrenia) prisoner or on house arrest
thoracic paravertebral block versus rhomboid block for postoperative pain control in thoracic surgeries

Conditions: Postoperative Pain

Summary: the main aim of this study is to compare thoracic paravertebral block and rhomboid block for postoperative analgesia and analgesic usage in thoracic surgeries.

patients with scheduled thoracic surgery are going to be enrolled for this study. patients will be randomized and separated in two groups. group i will receive thoracic paravertebral block, group ii will receive rhomboid block. each group will receive patient controlled analgesia with intravenous fentanyl. patients will be monitored at postoperative 1st, 3rd, 6th, 12th, 24th hours and their vas scores, adverse reactions like nausea and vomiting, total analgesic usage, additional analgesic need and time of the first report of pain will be filed. then the two groups will be compared and evaluated.

Eligibility criteria:
inclusion criteria: * patients scheduled for general thoracic surgery * asa i-iii exclusion criteria: * patients without consent * emergency surgeries * pregnancy * coagulopathies and bleeding diathesis * allergy to local anesthetics * local skin infection at the puncture site

Keywords: thoracic surgery postoperative pain rhomboid block paravertebral block
study of proton radiation to the brain and spinal cord for patients with leptomeningeal metastases

Conditions: Leptomeningeal Metastases

Summary: the purpose of this study is to find the safest and most effective dose for delivering proton beam to the space containing csf, brain, and spinal cord, in treating leptomeningeal metastases. the researchers think that using proton beam radiation to treat the space containing csf, brain, and spinal cord, instead of treating only the areas where the metastasized tumor cells are causing symptoms, would improve the treatment of this disease.


Eligibility criteria:
inclusion criteria: * female subjects must either be of non-reproductive potential (i.e. post-menopausal by history: \>/= 60 years old and no menses for 1\> year without an alternative medical cause; or history of hysterectomy, or history of bilateral tubal ligation, or history of bilateral oophorectomy) or must have a negative serum pregnancy test within 2 weeks prior to starting treatment. * patient with solid tumor malignancy with leptomeningeal metastases established radiographically and/or through csf cytology. * kps ≥ 60. * age ≥ 10 years. * for adult patient, the patient is able to provide informed consent. for pediatric patient, a parent is able to provide informed consent. * patient at reproductive potential must agree to practice an effective contraceptive method. * adequate bone marrow function: * hemoglobin ≥ 8g/dl * absolute neutrophil count ≥500/mm\^3 * platelet count ≥ 100,000/mm\^3 exclusion criteria: * patient with multiple, serious major neurologic deficits including encephalopathy. * patient with extensive systemic disease and without reasonable systemic treatment options. * patient who is unable to undergo mri brain and spine with gadolinium contrast. * previous radiotherapy to the intended treatment site that precludes developing a treatment plan that respects normal tissue tolerances. * pregnant or lactating women.

Keywords: proton radiation craniospinal irradiation (csi) 18-205
role of vitamin d in innate immunity to tuberculosis

Conditions: Vitamin D Deficiency; Tuberculosis

Summary: the investigators proposed a pilot study preparatory to developing a randomized trial of vitamin d for the prevention of tb infection. the specific aims are presented below: 1. to recruit subjects, obtain consent, distribute vitamin d supplements to the children in the schools, obtain blood samples and transport them to the united states. 2. to test the hypothesis that daily vitamin d supplementation will increase plasma levels of 25(oh)d, and restore tlr-induced antimicrobial activity in monocytes/macrophages tested in vitro.


Eligibility criteria:
inclusion criteria: * eligible subjects will be children age 12 to 15 years inclusive in public school, located in songino-khairhan district, who are residents of ulaanbaatar, and whose parents have given informed consent and who accent to participate in the study. exclusion criteria: * individuals with pre-existing calcium, parathyroid conditions, or type i diabetes, sarcoidosis, or who require chronic diuretic therapy including calcium channel blockers, or who are cognitively impaired.

Keywords: vitamin d deficiency tuberculin sin test immunity tuberculosis