Kornimate/medical-research-clean huggingface dataset:
allogeneic transplantation for severe osteopetrosis

Conditions: Severe Osteopetrosis

Summary: the purpose of this research is to explore what we believe may be a safer and more effective means of performing stem cell transplantation in patients with osteopetrosis, using chemotherapy and radiation designed to bring about engraftment and lessen transplant mortality. prior multi-institutional data in past studies found that approximately 30% of osteopetrosis patients do not engraft. therefore, in this study, we utilize a reduced intensity design of pre-transplant drugs to try to make transplants safer for this disease, as well as to provide a second infusion of stem cells in patients with matched related or unrelated donors.

this revised transplant protocol will test the following: 1) the ability to achieve engraftment with the reduced intensity protocol and a second infusion of stem cells on day 42, 2) the mortality associated with transplant by day 100, 3) patient outcomes, based on differential imaging and biologic evaluations prior to transplantation and at designated points after transplantation (day 100, 6 months, 1, 2 and 5 years). additional biologic studies will include microarray analysis, and evaluation of blood parameters and genes that may be important in the disease process. in older patients, studies to evaluation osteoclast differentiation and function will also be offered.

Eligibility criteria:
inclusion criteria: * patients eligible for transplantation under this protocol will be \< or = 45 years of age, and will be diagnosed with severe osteopetrosis. this will be defined as having the following manifestations of the disease. 1. bones that are uniformly markedly dense based on skeletal survey 2. no history that would suggest autosomal dominant inheritance 3. evidence of hematologic changes that are attributed to the underlying disease, including * the need for ongoing transfusions, or * the presence of progressive anemia or thrombocytopenia, or * a white blood cell differential with a predominance of immature forms and evidence of extramedullary hematopoiesis, or * persistence of serious infectious complications that are thought to be due to the abnormal architecture of the bone that are resistant to surgical and medical interventions. exclusion criteria: * patients \>45 years of age * evidence of hepatic failure * pulmonary dysfunction sufficient to significantly increase the risk of transplant. * renal dysfunction with glomerular filtration rate (gfr) \<30% of predicted. * cardiac compromise sufficient to substantially increase the risk of transplantation * severe, stable neurologic impairment. * human immunodeficiency virus (hiv) positivity. * pregnant or lactating females

Keywords: osteopetrosis
effects of traditional chinese medicine on bronchiectasis patients

Conditions: Bronchiectasis

Summary: the aim of this study is to evaluate the effectiveness of traditional chinese medicine (tcm) on patients with clinically stable bronchiectasis by a multi-center, randomized, double-blind, controlled trial: one, tcm treatments based on syndrome differentiation; the other, a placebo of tcm treatment.

bronchiectasis is a common and frequently occurring respiratory disease, with more hospitalization rate and mortality, poor quality of life and heavy financial burden. the main clinical manifestations are persistent or recurrent cough, expectoration, sometimes accompanied by hemoptysis, which can cause respiratory dysfunction and chronic pulmonary heart disease. there are some randomized controlled trials of tcm for bronchiectasis, and have the effect in the treatment of bronchiectasis, reducing the number of acute aggravation and alleviating dyspnea. however, some tcm interventions lack comprehensive syndrome differentiation and the placebo control. this is a multi-center, randomized, double-blind, controlled trial to compare the efficacy to evaluate the effectiveness of tcm on patients with clinically stable bronchiectasis. 216 subjects will be randomly assigned to one therapies (tcm treatments based on syndrome differentiation) and the other therapy (placebo of tcm treatment) for 24 weeks. after the treatment period, the subjects will be followed up for 24 weeks. the primary outcomes include the frequency of bronchiectasis exacerbation, and the secondary outcomes include quality of life (sgrq, lcq, qol-b), pulmonary function, safety observation index.

Eligibility criteria:
inclusion criteria: * a confirmed diagnosis of clinically stable bronchiectasis * age between 18 and 80 years * syndrome differentiation meets criteria of syndrome of qi deficiency of lung and phlegm-turbidity obstructing the lung, syndrome of qi and yin deficiency of the lung and the phlegm-heat obstructing the lung * without participations in other interventional trials in the previous one month * with the informed consent signed exclusion criteria: * current respiratory disorders other than bronchiectasis (e.g. copd,bronchial asthma, lung cancer,active tuberculosis, interstitial lung disease, pulmonary thromboembolic, or other patients who have an impact on the observation of disease) * patients with severe cardiovascular disease (e.g. acute myocardial infarction,acute heart failure) * severe liver disease(e.g.active phase of hepatitis, cirrhosis, severe impairment of liver function by portal hypertension) * severe renal disease(e.g. renal transplant, dialysis) * patients with large hemoptysis in the last month * pregnant and lactating women * insanity, dementia, and all kinds of psychosis * smoking patients (years of smoking10/ package year) * participating in other clinical trials with other interventions; * allergic to the used medicine.

Keywords: bronchiectasis traditional chinese medicine
pemetrexed +/- carboplatin as second line treatment in nsclc

Conditions: Advanced Non-Small Cell Lung Cancer

Summary: the aim of this study is to compare time to progression between the combination pemetrexed-carboplatin and pemetrexed alone in previously treated patients with locally advanced or metastatic nsclc.

in spite of the superiority of single agent over best supportive care in second-line nsclc, the prognosis of these patients remains poor with a median survival of 6-7 months, justifying the evaluation of new regimens in this setting. an open question in the second-line treatment of nsclc remains the possible superiority of combination chemotherapy over single agent, as it has been clearly demonstrated in first-line. particularly, the addition of platinum to either docetaxel or pemetrexed should be further investigated especially in patients with prior response/stable disease to platinum-based first-line chemotherapy. pemetrexed has shown anti-tumor activity in combination with other chemotherapy agents, including gemcitabine, vinorelbine, taxanes, cisplatin and carboplatin. in this multicenter, open label, randomized phase ii trial, pemetrexed 500 mg/m2 and carboplatin auc 5 will be administered on day 1 every 3 weeks until progression of disease or for a maximum of 4 courses in the experimental arm. the control arm will be pemetrexed as single agent at 500 mg/m2 on day 1 every 3 weeks until progression of disease or for a maximum of 4 courses.

Eligibility criteria:
inclusion criteria: * written informed consent * histologically or cytologically confirmed non-small-cell lung cancer * unresectable stage iiib, stage iv and unresectable local relapse or metastatic disease, with evidence of disease progression after first line chemotherapy which should have included a platinum agent. * ecog performance status lower than or equal to 2 * adequate hematological, hepatic and renal functions * life expectancy greater than or equal to 12 weeks * prior treatment with only 1 chemotherapy regimen for the treatment of advanced disease which should have included a platinum agent * at baseline, presence of at least one measurable target lesion as per recist criteria exclusion criteria: * prior treatment with pemetrexed. * patients who are pregnant or lactating * patients with any underlying medical condition that might be aggravated by treatment or which cannot be controlled. * symptomatic brain metastases * history of another malignancy within the past five years except basal cell carcinoma of the skin or carcinoma in situ of the cervix. * concomitant treatment with any other anticancer drug.

Keywords: second line treatment pemetrexed carboplatin
novel mri techniques in the evaluation of pulmonary vascular disease

Conditions: Pulmonary Hypertension

Summary: the diagnosis of a patient with pulmonary hypertension (ph) requires many investigations. at present cardiac catheterisation is the cornerstone investigation in these patients where it is used to establish disease severity and estimate prognosis. it is an invasive procedure which is expensive and not without risk to the patient. despite the multitude of tests performed, identifying those patients with ph who have a poor diagnosis can be difficult. the aim of this study is to improve the assessment of patients with ph using novel magnetic resonance techniques.


Eligibility criteria:
inclusion criteria: * all cases referred to the clinic for assessment for possible pulmonary hypertension. exclusion criteria: * patients with a cardiac pacemaker or retained temporary pacing wire * patients with aneurysm clip * non mri compatible heart valve prosthesis * intra orbital metalic foreign body * pregnancy * metal prosthesis/spinal rods * retained shrapnel * cochlear implants/bladder stimulator