Section 1 of 6
Terms and abbreviations
1 evidence topic · 0 sources
| Abbreviation | Term |
|---|---|
| 3PL | Third-party logistics provider |
| ACD | Anemia of chronic disease |
| ACVR1 | Activin A receptor type I |
| ADR | American depositary receipt |
| AE | Adverse event |
| Ae0-96h | Amount of drug excreted in urine from 0 to 96 hours |
| AHFS | American Hospital Formulary Service |
| ALK2 | Activin receptor-like kinase 2 |
| ANOVA | Analysis of variance |
| AP-HP | Assistance Publique-Hôpitaux de Paris |
| ASP | Average sales price |
| AUC | Area under the plasma concentration-time curve |
| AUC0-12h | Area under the plasma concentration-time curve from time 0 to 12 hours |
| AUC0-24h | Area under the plasma concentration-time curve from time 0 to 24 hours |
| AUC0-72 | Area under the plasma concentration-time curve from time 0 to 72 hours |
| AUC0-inf | Area under the plasma concentration-time curve from time 0 extrapolated to infinity |
| AUC0-t | Area under the plasma concentration-time curve from time 0 to the last measurable concentration |
| AUC0-tau | Area under the plasma concentration-time curve over the dosing interval |
| BCRP | Breast cancer resistance protein |
| BIA | Budget impact analysis |
| BID | Twice daily |
| BL | Baseline |
| BMI | Body mass index |
| BMP | Bone morphogenetic protein |
| BNDMR | Banque Nationale de Données Maladies Rares (French National Rare Diseases Registry) |
| BOIN | Bayesian optimal interval |
| C-QTc | Concentration-QTc |
| C1D1 | Cycle 1 Day 1 |
| CA | California |
| CADTH | Canadian Agency for Drugs and Technologies in Health |
| CAJIS | Cumulative Analogue Joint Involvement Scale |
| CCI | Charlson Comorbidity Index |
| CD34 | Cluster of differentiation 34 |
| CEO | Chief executive officer |
| CHMP | Committee for Medicinal Products for Human Use |
| CI | Confidence interval |
| CKD-EPI | Chronic Kidney Disease Epidemiology Collaboration |
| CL/F | Apparent oral clearance |
| CLR | Renal clearance |
| Cmax | Maximum plasma concentration |
| CMML | Chronic myelomonocytic leukemia |
| COVID-19 | Coronavirus disease 2019 |
| COX-2 | Cyclooxygenase-2 |
| CT | Computed tomography |
| Ctrough | Trough plasma concentration |
| CV | Coefficient of variation |
| CYP | Cytochrome P450 |
| DB | Double-blind |
| DE | Delaware |
| DLT | Dose-limiting toxicity |
| DNA | Deoxyribonucleic acid |
| ECG | Electrocardiogram |
| eGFR | Estimated glomerular filtration rate |
| EHR | Electronic health record |
| EMA | European Medicines Agency |
| ENDO | Endocrine Society annual meeting |
| EQ-5D | EuroQol 5 Dimensions |
| EQ-5D-5L | EuroQol 5 Dimensions, 5 Levels |
| EQ-5D-Y | EuroQol 5 Dimensions, Youth version |
| ESA | Erythropoiesis-stimulating agent |
| ESRD | End-stage renal disease |
| EU | European Union |
| EudraCT | European Union Drug Regulating Authorities Clinical Trials Database |
| FAP | Fibroadipogenic progenitor |
| FAS | Full analysis set |
| FDA | U.S. Food and Drug Administration |
| FKBP12 | FK506-binding protein 12 |
| fm | Fraction metabolized |
| FOP | Fibrodysplasia ossificans progressiva |
| FOP-PFQ | FOP Physical Function Questionnaire |
| FOP-PROMPT | Fibrodysplasia Ossificans Progressiva - Patient RepOrted syMPtoms Tool |
| FVC | Forced vital capacity |
| GI | Gastrointestinal |
| GMR | Geometric mean ratio |
| GP | General practitioner |
| GS | Glycine-serine (activation domain) |
| H0 | Null hypothesis |
| Ha | Alternative hypothesis |
| HAMP | Hepcidin antimicrobial peptide gene |
| Hb | Hemoglobin |
| HBV | Hepatitis B virus |
| HCP | Healthcare professional |
| HCV | Hepatitis C virus |
| HD | Hemodialysis |
| Hgb | Hemoglobin |
| HIV | Human immunodeficiency virus |
| HO | Heterotopic ossification |
| HR | Heart rate |
| IA | Interim analysis |
| ICER | Incremental cost-effectiveness ratio |
| ICF | Informed consent form |
| IDMS | Isotope dilution mass spectrometry |
| IEP | Individual Educational Program |
| IFOPA | International Fibrodysplasia Ossificans Progressiva Association |
| IgG4 | Immunoglobulin G4 |
| IL | Illinois |
| IL-1RA | Interleukin-1 receptor antagonist |
| IM | Intramuscular |
| Int-1, Int-2 | Intermediate-1 risk, intermediate-2 risk |
| iPSC | Induced pluripotent stem cell |
| IR | Immediate-release |
| ISR | Incurred sample reanalysis |
| IV | Intravenous |
| JAK | Janus kinase |
| ke | Apparent terminal elimination rate constant |
| L4 | Fourth lumbar vertebra |
| LSM | Least-squares mean |
| LY | Life-year |
| M.D. | Doctor of Medicine (physician) |
| MAA | Marketing authorization application |
| MAD | Multiple ascending dose |
| MAP | Mirum Access Plus |
| MATE1 | Multidrug and toxin extrusion protein 1 |
| MATE2-K | Multidrug and toxin extrusion protein 2-K |
| MDRD | Modification of Diet in Renal Disease |
| MDS | Myelodysplastic syndromes |
| MF | Myelofibrosis |
| MIM | Mendelian Inheritance in Man |
| MM | Multiple myeloma |
| mo | Month |
| MPN | Myeloproliferative neoplasm |
| MRP2 | Multidrug resistance-associated protein 2 |
| MTD | Maximum tolerated dose |
| N/A | Not applicable |
| NA | Not available |
| NCT | National Clinical Trial (ClinicalTrials.gov identifier) |
| NDA | New Drug Application |
| NDC | National Drug Code |
| NHS | Natural History Study |
| NICE | National Institute for Health and Care Excellence |
| NSAID | Nonsteroidal anti-inflammatory drug |
| NTD | Non-transfusion-dependent |
| OAT | Organic anion transporter |
| OATP | Organic anion transporting polypeptide |
| OCT | Organic cation transporter |
| OLE | Open-label extension |
| OT | Occupational therapy |
| P-gp | P-glycoprotein |
| PBO | Placebo |
| PD | Pharmacodynamics |
| PDGFRα | Platelet-derived growth factor receptor alpha |
| PDUFA | Prescription Drug User Fee Act |
| PET | Positron emission tomography |
| PICC | Peripherally inserted central catheter |
| PK | Pharmacokinetics |
| po | By mouth (per os) |
| PPAR-γ | Peroxisome proliferator-activated receptor gamma |
| PPC | Premature physeal closure |
| PRMA | Patient-Reported Mobility Assessment |
| PROMIS | Patient-Reported Outcomes Measurement Information System |
| PRV | Priority Review Voucher |
| pts | Patients |
| Q2, Q3 | Second quarter, third quarter |
| QALY | Quality-adjusted life year |
| QD | Once daily |
| QoL | Quality of life |
| QTc | QT interval corrected for heart rate |
| QTcI | QT interval with individualized correction for heart rate |
| R206H | Arginine-to-histidine substitution at codon 206 of ACVR1 |
| RARγ | Retinoic acid receptor gamma |
| RBC | Red blood cell |
| RDE | Recommended dose for expansion |
| RUX | Ruxolitinib |
| SAD | Single ascending dose |
| SAE | Serious adverse event |
| SAP | Statistical analysis plan |
| Sca1 | Stem cell antigen-1 |
| SD | Standard deviation |
| SE | Standard error |
| SF-36 | 36-Item Short Form Health Survey |
| SLR | Systematic literature review |
| SMAD | Small mothers against decapentaplegic (signaling protein family) |
| SoC | Standard of care |
| STI | Signal transduction inhibitor |
| T4 | Fourth thoracic vertebra |
| TD | Transfusion-dependent |
| TEAE | Treatment-emergent adverse event |
| TGA, TGB, TGC | Treatment group A, treatment group B, treatment group C |
| TGF-β | Transforming growth factor beta |
| Tie2 | Tyrosine kinase with immunoglobulin-like and EGF-like domains 2 |
| TLC | Total lung capacity |
| tmax | Time to maximum plasma concentration |
| TNF-α | Tumor necrosis factor alpha |
| TRAE | Treatment-related adverse event |
| t½ | Elimination half-life |
| UAE | United Arab Emirates |
| UMC | University Medical Center |
| US, U.S., USA | United States |
| USD | U.S. dollars |
| USP | United States Pharmacopeia |
| VUmc | VU University Medical Center |
| Vz/F | Apparent volume of distribution during the terminal phase |
| WAC | Wholesale acquisition cost |
| WBCT | Whole-body computed tomography |
| WHO | World Health Organization |
| wk | Week |
| wLME | Weighted linear mixed effects |
| WTP | Willingness to pay |
| yr | Year |
| ZBI | Zarit Burden Interview |
| ZGB | Zilurgisertib |