Kornimate/medical-research-clean huggingface dataset:
simvastatin treatment of pachyonychia congenita

Conditions: Pachyonychia Congenita

Summary: simvastatin and related statins, such as compactin, are able to inhibit the constitutive expression of inducible/repressible keratin genes such as k6a and k17. this effect is due to the reported ability of statins to inhibit stat1 expression (lee et al., 2007). the constitutive k6a promoter activity is dependent on stat1 and blocked by simvastatin or sirna against stat1. this stat1-dependent constitutive expression of k6a, is independent of jak (janus family of kinases). thus simvastatin is capable of down-regulating both the constitutive and interferon-inducible expression of pc-related keratins such as k6a and k17. therefore, this class of molecule has potential for the treatment of pc or related inherited disorders where the causative mutation resides in an inducible/repressible keratin gene such as k6a or k17. simvastatin or other statins approved for human use might be delivered either orally, as is currently the case for cholesterol-lowering treatment or, if higher therapeutic doses are required in skin for reduction of hyperkeratosis in pc or in related keratinizing disorders, this might be achieved by topical formulations.


Eligibility criteria:
inclusion criteria: * patients with pachyonychia congenita with known mutations in keratin 6a. - exclusion criteria: * the use of concomitant medications known to interact with simvastatin. * this would include itraconazole, ketoconazole, fluconazole, gemfibrozil, verapamil, diltiazem, mibefradil, erythromycin, clarithromycin, telithromycin, cyclosporine, ritonavir, nefazodone, danazol, amiodarone, rifampin, and carbamazepine. * during the study subjects must agree to avoid grapefruit juice. * also excluded are patients with a past history of myopathy, or impaired liver function.
efficacy of omalizumab as add on therapy for minority patients with moderate to severe asthma

Conditions: Asthma

Summary: this study will be a randomized, placebo controlled, double blind study to measure the safety and efficacy of a new, injectable asthma medication, omalizumab, in a group of minority with moderate to severe asthma who are not adequately controlled with use of inhaled or oral steroids. primary endpont will be change in asthma symptom utility index. secondary endpoints will be changes in asthma quality of life, asthma exacerbation rate, and lung function over the 12 week treatment period.


Eligibility criteria:
inclusion criteria: * age 18-30 * minority patients * moderate or severe allergic asthma (as defined by the national asthma education and prevention guidelines(7)\]. allergic asthma will be defined as elevated ige level (\> 100 iu/ml) and skin test sensitivity to at least one common aeroallergen * asthma control score greater than 1.5 (5) at randomization visit with use of equivalent of at least 1000 mcg fluticasone daily or systemic steroid use * fev1 50-90% predicted at screening and randomization visit exclusion criteria: * active smoking within one year and/or greater than 10-pack year history of smoking * women of childbearing age must be using effective contraception * malignancy diagnosed within the past 5 years * underlying lung disease other than asthma * inability to comply with study protocol

Keywords: omalizumab severe asthma moderate asthma injectable asthma medication xolair
central sensitization and alteration of circulating neurosteroid

Conditions: Arthroplasty, Replacement, Knee; Pain, Postoperative

Summary: for patients who underwent two-stage replacement of both knee joints (one knee surgery - one week term- the other knee surgery), postoperative pain and analgesic usage with patient-controlled analgesia (pca) increased at the second stage, which suggests that central sensitization occurs within a short period (one week) in patients who undergo total knee arthroplasty (tka). neurosteroids act on nmda and ampa receptors, gabaa receptors, and voltage-dependent ca2+ or k+ channels of sensory neurons to increase invasive or neuropathic pain and, conversely, to exhibit analgesic and anticonvulsant effects. these actions mean that the neurosteroid acts as an endogenous regulator of pain control and central sensitization. the purpose of this study is to confirm that the hypothalamus-pituitary-adrenal (hpa) axis, which is the main body of endocrine neurosteroid, is associated with increased pain sensitivity after tka. the concentrations of cortisol and dehydroepiandrosterone (dhea) in the saliva of patients who undergo two-staged bilateral knee replacement surgery (one knee surgery - one week term- the other knee surgery) will be measured at each stage and analyzed for correlation between concentration-related changes of hpa and postoperative knee pain variations.


Eligibility criteria:
inclusion criteria: * two-staged bilateral total knee arthroplasty (one knee - one week - the other knee) * american society of anesthesiologists (asa) class i, ii * postmenopausal women aged under 71 exclusion criteria: * patients who have used analgesics before surgery, such as chronic pain patients * ≥ asa class iii, including patients with renal impairment or symptomatic cardiovascular disease * patients who refuse to participate in the study or from whom receive informed consent cannot be received.

Keywords: central nervous system sensitization neuropeptides
observational registry of novoseven® used as on-demand treatment of bleeds in patients with haemophilia a and b with inhibitors

Conditions: Congenital Bleeding Disorder; Haemophilia A With Inhibitors; Haemophilia B With Inhibitors

Summary: this study was conducted in africa, europe, the middle-east and south america. the primary objective of this registry was to observe the use of single dose and multi-dose use of activated recombinant human factor vii and to compare short-term outcomes, including effectiveness, safety, quality of life and treatment satisfaction with the approved treatments.


Eligibility criteria:
inclusion criteria: * diagnosed with haemophilia a or b with inhibitors * experience mild to moderate spontaneous bleeds which require on-demand treatment and who are currently prescribed activated recombinant human factor vii * be able and willing to provide informed consent (or proxy consent by caregiver, if applicable), as required by local research ethics committee, governmental or regulatory authorities * be willing to provide information on at least one alternate contact person in the event that the patient be somehow lost-to-follow-up over the course of registry participation (not applicable if patient is withdrawn) exclusion criteria: * known hypersensitivity to the active substance or the excipients in the formulation of activated recombinant human factor vii, or to mouse, hamster or bovine protein
a feasibility study of octreotide infusion during liver transplant.

Conditions: Liver Transplantation; Renal Failure

Summary: the purpose of the study is to determine whether an octreotide infusion during liver transplantation improves renal outcomes, intraoperative blood pressure and reduces haemorrhage and transfusion requirement.

common and serious complications of liver transplantation surgery include renal failure, haemorrhage and blood transfusion. these complications prolong post-operative recovery, increase the risk of liver graft failure, mortality and the need for long-term renal dialysis. the drug octreotide is a synthetic analogue of somatostatin with comparable physiological effects and a good side-effect profile. existing evidence in liver transplantation supports octreotide efficacy in improving renal function, reducing bleeding and enhancing blood pressure. however, there is no robust randomised controlled trial evidence for octreotide in liver transplantation and limited safety data regarding its use in this setting. this is a multi centre, prospective double-blind, randomised, placebo-controlled trial of octreotide infusion during liver transplantation. the patients will be randomised in a 2:1 ratio to either octreotide or placebo groups. stratified randomisation of patients is by donation type (dcd vs. dbd). patients will be randomised in the anaesthetic room and study medication given as an initial bolus of 5ml (100mcg octreotide or saline) prior to surgical incision and then continued throughout surgery at 5ml/hr (100mcg/hour octreotide or saline).

Eligibility criteria:
inclusion criteria: * adults aged 18 years and over undergoing primary liver transplantation of a whole or partial liver graft from a cardiac or brain dead donor. * provision of written informed consent. exclusion criteria: * previous solid organ transplant. * acute liver failure. * fulminant hepatic failure. * patients receiving a living donor liver graft. * patients currently admitted to icu prior to transplantation. * requirement of haemodialysis or cvvhf pre-operatively. * known allergy or adverse reaction to octreotide. * pre-operative decision to use intra-operative cvvhf. * a positive pregnancy test.
facilitating access to specialty treatment

Conditions: Mild Traumatic Brain Injury; Concussion, Mild; Concussion, Severe; Concussion, Intermediate

Summary: the goal of this hybrid implementation-effectiveness study is to evaluate the effectiveness (hastened recovery times) and feasibility (fidelity in connecting to concussion specialty care) of a novel mobile health intervention, designed to reduce disparities in access to specialty care through the use of remote patient monitoring (rpm) to facilitate care hand-off from the emergency department (ed) to concussion specialty care. participants will report their symptoms and activity once daily through rpm chat technology that is linked to their electronic health record and prompts referral to specialty care.


Eligibility criteria:
inclusion criteria for concussed subjects: * males and females age 13 - 18 * present to the children's hospital of philadelphia (chop) emergency department (ed) within 72 hours of head injury * meet criteria for concussion as defined by the most recent international consensus statement on concussion * own a smartphone * meet criteria for moderate-to-high risk for persistent post-concussion symptoms according to 5p rule (score \>3/12) exclusion criteria for concussed subjects: * glasgow coma scale score \<13 * lower extremity trauma * a prior concussion within 1 month * non-english speaking * admission to the hospital at the initial head injury visit * previously enrolled in the study * inability to complete study procedures. inclusion criteria for parents: * child meets the study eligibility criteria exclusion criteria for parents: * non-english speaking inclusion criteria for providers: * ed or specialty provider caring for at least one patient via the mobile health (mhealth)-facilitated care handoff strategy exclusion criteria for providers: * non-english speaking

Keywords: mobile health emergency department persistent post-concussion symptoms remote patient monitoring
rapid kidney function decline in older adults

Conditions: Kidney Function Decline; Glomerular Filtration Rate; Urine Albumin-creatinine Ratio; Healthy Aging; Comorbidity; Chronic Kidney Disease

Summary: this observational study investigates whether a single set of gfr and urine albumin-to-creatinine ratio (acr) thresholds can accurately screen for rapid kidney function decline in different elderly groups.


Eligibility criteria:
inclusion criteria: 1. age greater than 70 years; 2. gfr data measured at least twice (two years apart) and acr data measured once (at baseline); 3. a clear record of the cause of the previous visit; from this, people diagnosed with kidney disease were selected, and kidney disease was defined as various primary nephritis, secondary nephropathy, acute kidney injury (aki), chronic kidney disease, kidney tumors, or a diagnosis of proteinuria alone. 4. signed informed consent form. exclusion criteria: 1. people with missing baseline biochemical data; 2. outliers in the population indicators, i.e., data deviating from the mean ± three times the standard deviation; 3. data with significantly abnormal baseline renal function follow-up (abnormally elevated gfr by more than 10% within 2 years or abnormal acr value as determined by consultation with a senior nephrologist).
cardiovascular events in patients with cad during emergent endoscopy for upper gastrointestinal bleeding

Conditions: Coronary Disease

Summary: background: upper gastrointestinal (gi) bleeding, a common disorder encountered at emergency room, may cause hypotension and tachycardia that may in turn result in myocardial ischemia in patients with coronary artery disease (cad). an emergent endoscopy with hemostasis is the mainstay of management. however, endoscopy itself may result in myocardial ischemia. whether myocardial ischemia and arrhythmia occurs more frequently during emergent endoscopy in patients with cad remains unknown. objective: to determine whether the risk of myocardial ischemia and arrhythmia is increased during emergent endoscopy in patients with cad. method: adult patients with documented cad undergoing emergent endoscopy due to ugi bleeding are included. adult patients without cad undergoing emergent endoscopy due to ugi bleeding were included as the control group. the expected case numbers were 50 patients in each group. patients with terminal illness, pregnancy, active lung disease requiring ventilator support are excluded. before endoscopy, symptoms of myocardial ischemia, blood pressure, heart rate, o2 saturation, hemogram, baseline 12-lead ekg, and cardiac enzyme are obtained. all patients are monitored with holter ekg since 10 minutes before endoscopy to 2 hours after the procedure. blood pressure, heart rate, and o2 saturation are closely monitored during the procedure. endoscopy is performed by experienced endoscopist and endoscopic hemostasis is done according to the types of lesions. the duration of endoscopy, types of endoscopic hemostasis, and blood pressure are recorded. after endoscopy, symptoms of myocardial ischemia, blood pressure, heart rate, o2 saturation, hemogram, baseline 12-lead ekg, and cardiac enzyme are checked again. keywords: emergent endoscopy, coronary artery disease, cardiovascular events

expected result and implication if myocardial ischemia is not increased in cad patients undergoing emergent endoscopy, then this technique can be used safely in this group of patients. if myocardial ischemia occurs more frequently in cad patients undergoing emergent endoscopy, we can try to analyze factors associated with increased risk, such as duration of endoscopy, severity of anemia, or drugs used for endoscopic hemostasis. then we can try to avoid these predisposing factors and increase the safety of emergent endoscopy.

Eligibility criteria:
inclusion criteria: * adult patients with documented cad undergoing emergent endoscopy due to ugi bleeding are included. * adult patients without cad undergoing emergent endoscopy due to ugi bleeding were included as the control group. exclusion criteria: * acute coronary syndrome before enrollment * unable to receive upper endoscopy
open-label study of efficacy and safety of recombinant human n-acetylgalactosamine 4-sulfatase in patients with mps vi

Conditions: Mucopolysaccharidosis VI

Summary: the purpose of the study is evaluate the efficacy, safety, and pharmacokinetics of weekly intravenous infusions of 1 mg/kg recombinant human n-acetylgalactosamine 4-sulfatase (rhasb) in patients diagnosed with mucopolysaccharidosis vi (mps vi)


Eligibility criteria:
inclusion criteria: * patient consent * patient must be five years of age or older * patient must have documented biochemical or genetic proof of mps vi * patient must walk at least 1 meter, but less than 250 meters, in the first 6 minutes of the baseline 12-minute walk test * if female of childbearing potential, patient must have a negative pregnancy test exclusion criteria: * patient is under consideration for or has undergone a successful bone marrow transplant (bmt). * pregnant or lactating patient * patient has received an investigational drug within 30 days prior to study enrollment * patient has been previously treated with rhasb * patient has a medical condition, serious intercurrent illness, or other extenuating circumstance that may significantly confound study results or decrease study compliance * patient has a known hypersensitivity to rhasb or to components of the study drug * history of cancer (except low grade and fully resolved skin malignancy)
beta-blockers in pulmonary arterial hypertension

Conditions: Pulmonary Arterial Hypertension

Summary: the investigators will conduct a, randomized, phase 2, placebo-controlled, double-blinded, crossover trial of carvedilol in 26 pah patients with world health organization functional class ii or iii symptoms and rv ejection fraction (ef) \< 45% for 6 months.

adult pah patients on a stable dose of an approved pah medication will undergo the following baseline assessments: cardiac magnetic resonance imaging (mri), right heart catheterization (rhc), echocardiogram, 6-minute walk test (6-mwt), plasma nt-probnp (biomarkers of rv function) and serum catecholamine (measure of sympathetic activation), and quality of life. patients will be randomized to carvedilol (3.125 mg bid and escalated to 9.375 mg bid, as tolerated, over 3 months) or placebo in a 1:1 fashion. after 6 months, testing is repeated and patients are crossed over to the alternate treatment. testing is repeated at the end of the study (month 13).

Eligibility criteria:
inclusion criteria: * age \> 18 years * who category 1 pulmonary arterial hypertension (nice 2013) * who functional class ii-iii * rvef by cardiac mri \< 45% * stable on pah-specific therapy as defined by no change in pah-specific treatment and functional class in the past 3 months. patient can be on either mono or combination pah-specific therapy exclusion criteria: * subjects will be excluded from participation in the study if any of the following conditions exist: * significant persistent bradycardia (resting heart rate \< 60 bpm) without a permanent pacemaker * second or third degree av block without a permanent pacemaker * significant sinus tachycardia (resting heart rate \> 110 bpm) * use of anti-arrhythmic drugs * hypotension defined as systolic blood pressure \< 100 mmhg at the time of enrollment * significant illness in the past 30 days requiring hospitalization * acute decompensated right heart failure within past 30 days * known allergy or intolerance to carvedilol or other β blockers * cardiac index \ 15 mm hg by right heart catheterization within last 3 months * asthma * positive pregnancy test in patients of child bearing-potential
effects of olive oil and bran oil on antioxidant levels, glycemic control, and lipid profile in patient type 2 dm

Conditions: Diabetes Mellitus, Type 2; Oxidative Stress; Glucose, High Blood; Lipid Disorder

Summary: this study determines the effect of olive oil and bran oil on antioxidant levels, and glycemic control in patients with type 2 diabetes mellitus (dm) intervention: patient type 2 dm will receive olive oil and bran oil with cross over study

diabetes mellitus (dm) has become one of the global public health problems. the chronic complications of type 2 dm can be macrovascular and microvascular complications that can decrease the quality of life of the patient. the main goal of sustainable dm nutrition therapy is to keep glucose in the blood close to normal levels to stop hyperglycemic and hyperlipidemic events that may inhibit further complications this study was a clinical trial with cross over study design, random allocation, and double blindness to compare changes in antioxidant, glucose, cholesterol and triglyceride levels supplemented with 15 ml / day of olive oil (kz) with supplementation of 15 ml / day of bran oil (kb). provision of oil for 4 weeks in a row. during the 2-week interval not being treated (wash out). after the wash out period cross-over by exchanging olive oil supplementation to rice bran and vice versa for 4 consecutive weeks. the research will be conducted at family clinic clinic fkui kayu putih, jakarta.

Eligibility criteria:
inclusion criteria: * patients with type 2 diabetes aged 30-60 years. * diagnosed type 2 dm \<3 years. * body mass index of 20-30 kg / m2 * subjects willing to participate in research and sign informed consent. * drinking oho (oral hyperglycemic medication). exclusion criteria: * there are acute and chronic complications. * subject is pregnant. * get cholesterol-lowering drugs, steroids and other drugs that affect the metabolism of fat. * smoking more than 10 cigarettes / day. * eat regular supplements that contain phytosterols or other antioxidants that are known by anamnesis. * has a disorder or gastrointestinal disease, thyroid, heart, liver, cancer, stroke and kidney are known with medical record data.

Keywords: olive oil bran oil type 2 diabetes mellitus antioxidant glycemic control lipid profile
donafenib monotherapy for previously treated metastatic nasopharyngeal carcinoma

Conditions: Nasopharyngeal Carcinoma

Summary: the investigators do the clinical trial (patients with metastatic nasopharyngeal carcinoma treated with donafenib after failure of standard therapy) to assess safety and efficacy of donafenib in patients with metastatic nasopharyngeal carcinoma, progressing after all approved standard therapies.

the study is an open-label, phase 1b study recruiting 18 patients. patients were eligible to participate when they have histological or cytological documentation of the nasopharyngeal carcinoma. they must have received locally and currently approved standard therapies and to have disease progression after the last administration of the last standard therapy or to have stopped standard therapy because of unacceptable toxic effects. the available standard therapies have to include one or more drugs of the following as were licensed: a fluoropyrimidine,taxane,ddp, gemcitabine. patients have to be aged 18 years or older and have an eastern cooperative oncology group (ecog) performance status of 0 or 1; life expectancy of at least 3 months;and adequate bone-marrow, liver, and renal function at the start of the trial. patients could not participate if they had previously received sorafenib or had uncontrolled medical disorders. all patients receive best supportive care, excluding other investigational antitumour agents or antineoplastic chemotherapy, hormonal therapy, or immunotherapy.patients receive oral donafenib 200mg (cm4307) of each 4 week cycle until disease progression,death, unacceptable toxic effects, withdrawal of consent by the patient,or decision by the treating physician that discontinuation would be in the patient's best interest.the primary endpoint is safety.the second endpoint is progression-free survival.

Eligibility criteria:
inclusion criteria: * have histological or cytological documentationof the nasopharyngeal carcinoma; * have received system standard therapies and to have disease progression(recist1.1) or to have stopped standard therapy because of unacceptable toxic effects. * standard therapies including as many of the following as were licensed: a fluoropyrimidine,taxol,ddp,or gemcitabine; * have an eastern cooperative oncology group (ecog) performance status of 0 or 1; * life expectancy of at least 3 months; * have adequate bone-marrow, liver, and renal function at the start of the trial. • prothrombin time international normalized ratio≤2；or prothrombin time≤16 seconds;or activated partial thromboplastin time(aptt) ≤43 seconds;or tt≤21 seconds. exclusion criteria: * patients have prior treatment with sorafenib; * patients have central nervous system(cns) involvement; * patients have uncontrolled medical disorders.

Keywords: nasopharyngeal carcinoma,donafeinib