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Alpha-1 antitrypsin deficiency

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Search Period All yearsLast 12 monthsLast 5 years Topic All topicsGeneralNutrition and dietAlternative treatment methods Type of research Any type of researchHighest Level of Evidence Only (35)Meta-analysis (8)Treatment guideline (5)Phase 3 study (8)Randomized trial (8)Systematic review (6)Clinical Trial (169)Review article (176)Early study (phase 1 or 2) (18)Case report (114)Laboratory or animal study (27)Commentary or editorial (90)Retracted publication (1) Only studies currently recruiting participants (28)

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Publications and studies (1173)

• Danish children with ZZ-homozygous alpha-1 antitrypsin deficiency are more affected on liver parameters than children with heterozygosity. (2024/03/01) ♡
• Alpha-1 Antitrypsin Augmentation and the Liver Phenotype of Adults With Alpha-1 Antitrypsin Deficiency (Genotype Pi∗ZZ). (2024/02/01) ♡
• Sirtuin3 promotes the degradation of hepatic Z alpha-1 antitrypsin through lipophagy. (2024/01/29) ♡
• Personalised indication of augmentation therapy for emphysema associated with severe alpha-1 antitrypsin deficiency: a case series. (2024/01/01) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Study of DCR-A1AT in Healthy Adult Volunteers (2024-11-06) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Long-Term Follow-up Study of ADVM-043 (2024-09-20) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. An Extension Study of Belcesiran in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATLD) (2024-09-19) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. FibroScan™ in Pediatric Cholestatic Liver Disease (FORCE) (2024-08-28) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Alvelestat (MPH966) for the Treatment of ALpha-1 ANTitrypsin Deficiency (2024-08-20) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. COPD Exacerbation Blood and Urine Biomarkers Study (2024-06-04) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Exploring the Utility of Hyperpolarized 129Xe MRI in Healthy Volunteers and Patients With Lung Disease (2024-05-21) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Phase 1, First-in-human Study of VX-668 (2024-03-15) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Phase 1, First-in-human Study of VX-634 (2024-03-12) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Alpha-1 Research Registry (2024-01-30) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. Provider Education in Alpha 1 Antitrypsin Deficiency: Try Again, Fail Again, Fail Better. (2023/12/28) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Vanishing Lung Syndrome in a Young Male With Chronic Marijuana Use: A Case Report. (2023/12/28) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Testing Alpha-1 Antitrypsin Deficiency in Black Populations. (2023/12/19) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Targeting the Liver with Nucleic Acid Therapeutics for the Treatment of Systemic Diseases of Liver Origin. (2023/12/15) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Unusual case of bullous emphysema with superimposed pneumonia. (2023/12/15) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Dipeptidyl peptidase 1 inhibition as a potential therapeutic approach in neutrophil-mediated inflammatory disease. (2023/12/14) ♡
• Probing of the reactive center loop region of alpha-1-antitrypsin by mutagenesis predicts new type-2 dysfunctional variants. (2023/12/13) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Primary biliary cholangitis with features of autoimmune hepatitis in a 19-year-old adolescent with 14q24.1q24.2 deletion: a case report. (2023/12/12) ♡
• Alpha-defensins inhibit ERK/STAT3 signaling during monocyte-macrophage differentiation and impede macrophage function. (2023/12/11) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. Alpha-1 antitrypsin deficiency: Relevance in haematology. (2023/12/07) ♡
• Neonatal cholestasis in children with Alpha-1-AT deficiency is a risk for earlier severe liver disease with male predominance. (2023/12/07) ♡
• Molecular confirmation of alpha 1-antitrypsin deficiency in liver transplant setting: A province-wide experience. (2023/12/07) ♡
• Nine controversial questions about augmentation therapy for alpha-1 antitrypsin deficiency: a viewpoint. (2023/12/06) ♡
• Plasma levels of α(1)-antitrypsin-derived C-terminal peptides in PiMM and PiZZ COPD patients. (2023/12/04) ♡
• A test of automated use of electronic health records to aid in diagnosis of genetic disease. (2023/12/01) ♡
• Randomized researchiParticipants were divided into groups by lottery and compared with each other. This reduces the chance that a difference is due to something other than the treatment. Governmental Non-Pharmaceutical Interventions during the COVID-19 Pandemic and the COPD Exacerbation and Respiratory Infection Rate in Patients with Alpha-1 Antitrypsin Deficiency. (2023/12/01) ♡
• Evaluation of body weight-based dosing, alternative dosing regimens, and treatment interruptions for α1-proteinase inhibitors and implications on biochemical efficacy in patients with α1-antitrypsin deficiency. (2023/12/01) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Intrahepatic cholestasis of pregnancy and theory of inheritance of the disease. Literature review. (2023/12/01) ♡
• Risk factors of prolonged diarrhea in children under 2 years old. (2023/12/01) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. Are clinical trials into emerging drugs for the treatment of alpha-1 antitrypsin deficiency providing promising results? (2023/12/01) ♡
• European pathways of care in Alpha-1 Antitrypsin deficiency. (2023/12/01) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. An Alpha-1 Antitrypsin Deficiency Screening Study in Patients with Chronic Obstructive Pulmonary Disease, Bronchiectasis, or Asthma in Turkey. (2023/11/28) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Case report: Self-administration of alpha-1 antitrypsin therapy: a report of two cases. (2023/11/21) ♡
• Baseline characteristics from a 3-year longitudinal study to phenotype subjects with COPD: the FOOTPRINTS study. (2023/11/17) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. Health-related Quality of Life in Alpha-1 Antitrypsin Deficiency-associated Chronic Obstructive Pulmonary Disease. (2023/11/15) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. AATD as a genetic risk factor for aneurysmal disease. (2023/11/04) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. AATD as a genetic risk factor for aneurysmal disease - Authors' reply. (2023/11/04) ♡
• Correction to: Cigarette smoke exposed airway epithelial cells-derived EVs promote pro-inflammatory macrophage activation in alpha-1 antitrypsin deficiency. (2023/11/04) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Transplant with MZ genotype liver: what is the clinical pulmonary picture after 30 years? a case report and review of the literature. (2023/11/02) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. PI*ZQ0(Attikon) genotype discovery in severe alpha-1 antitrypsin deficiency. (2023/11/01) ♡
• Evaluating Novel Protein Phosphatase 2A Activators as Therapeutics for Emphysema. (2023/11/01) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Augmentation Therapy for Severe Alpha-1 Antitrypsin Deficiency Improves Survival and Is Decoupled from Spirometric Decline-A Multinational Registry Analysis. (2023/11/01) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Liver disease progression in patients with alpha-1 antitrypsin deficiency and protease inhibitor ZZ genotype with or without lung disease. (2023/11/01) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. Treatment for Alpha-1 Antitrypsin Deficiency: Does Augmentation Therapy Work? (2023/11/01) ♡
• Meta-analysisiAll studies on one question combined and calculated together. This is the strongest form of research that exists: a single loose study can be coincidence, dozens together much less so. The label says something about the design, not about the outcome — which can also be that something does NOT work. Single vs Bilateral Lung Transplant in the Management of Patients With Chronic Obstructive Pulmonary Disease: A Systematic Review and Meta-Analysis. (2023/11/01) ♡
• Sarcopenia is associated with osteopenia and impaired quality of life in children with genetic intrahepatic cholestatic liver disease. (2023/10/31) ♡

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