DOCSLIB.ORG

Intestininės Enzimopatijos

Total Page：16

File Type：pdf, Size：1020Kb

Download full-text PDF Read full-text
Intestininės Enzimopatijos VILNIAUS UNIVERSITETAS DANUTĖ KALIBATIENĖ INTESTININĖS ENZIMOPATIJOS Mokslinė monografija VILNIAUS UNIVERSITETO LEIDYKLA 2014 PRATARMĖ 1 Monografijos leidyba patvirtinta Vilniaus universiteto Medicinos fakulteto tarybos posėdyje 2014 m. balandžio 23 d. Recenzavo: Prof. Limas KUPČINSKAS Prof. Laimas JONAITIS Prof. Algimantas IRNIUS ISBN 978-609-459-321-5 © Danutė Kalibatienė, 2014 © Vilniaus universitetas, 2014 TURINYS PRATARMĖ ....................................................... 5 1. VIRŠKINIMO TRAKTO FERMENTAI IR INTESTININĖS ENZIMOPATIJOS .............................. 8 1.1. Virškinimo trakto fermentai ...................................... 8 1.2. Disacharidazių nepakankamumas ................................. 9 1.3. Sukrazės-izomaltazės nepakankamumas .......................... 11 1.4. Trehalazės nepakankamumas . 14 2. LAKTAZĖS NEPAKANKAMUMAS ................................ 19 2.1. Laktozės metabolizmas ......................................... 19 2.2. Plonosios žarnos gleivinės fermentas laktazė ...................... 20 2.3. Pieno ir laktozės netoleravimas: istorinė apžvalga .................. 24 2.4. Laktazės aktyvumo pokyčių klasifikacija ir terminija ............... 26 2.5. Pirminės suaugusiųjų hipolaktazijos genetika ..................... 28 2.6. Laktazės nepakankamumo fenotipo paplitimas . 38 2.7. Laktazės aktyvumo persistavimo / nepersistavimo genotipų paplitimo tyrimai ....................... 44 2.8. Laktazės nepakankamumo paplitimas Lietuvoje .................... 47 2.9. Netolygaus laktazės persistavimo / nepersistavimo genotipų paplitimo teorijos ........................ 62 2.10. Laktazės nepakankamumo diagnostikos metodai .................. 75 2.11. Laktazės nepakankamumo poveikis sveikatai ..................... 94 2.12. Antrinis laktazės nepakankamumas ............................. 107 2.13. Laktazės nepakankamumo sąsajos su lėtinėmis ligomis ............ 111 2.14. Laktazės nepakankamumo gydymas ............................ 114 3. CELIAKINĖ LIGA ............................................... 124 3.1. Celiakinės ligos apibrėžtis ...................................... 124 3.2. Celiakinės ligos paplitimas ..................................... 124 3.3. Etiologija ir patogenezė ........................................ 126 3.4. Celiakinės ligos diagnostikos kriterijai ........................... 134 3.5. Celiakinės ligos klinikiniai fenotipai ............................. 134 3.6. Celiakinės ligos klinikinės asociacijos ............................ 137 3.7. Celiakinės ligos gydymas ....................................... 140 LITERATŪRA ................................................... 145 AUTORĖS DARBAI IR PUBLIKACIJOS MONOGRAFIJOS TEMA ..................... 176 SANTRUMPOS .................................................. 177 SANTRAUKA .................................................... 178 ABSTRACT ...................................................... 182 4 PRATARMĖ ntestininės enzimopatijos – tai organizmo būklė, kai sumažėja arba visiškai išnyksta Iplonosios žarnos fermentų (enzimų), dalyvaujančių virškinimo procese, aktyvumas ir dėl to sutrinka tam tikrų maisto medžiagų pasisavinimas. Intestininės enzimopatijos gali būti pirminės, nulemtos genetiškai, ir antrinės, susijusios su ligomis, kuriomis ser- gant pažeidžiama plonosios žarnos gleivinė. Prieš keliasdešimt metų pasirodė pirmosios mokslinės publikacijos, gvildenančios plonosios žarnos fermentų aktyvumo sumažėjimo problemą ir svarbą medicinos prak- tikoje. Nuo to laiko daugiausia tyrinėta dažniausiai pasitaikanti intestininė enzimopa- tija – laktazės nepakankamumas. Pasirodė daug straipsnių, atspindinčių įvairius pieno cukraus (laktozės) pasisavinimo aspektus, tačiau problema lieka nepakankamai aiški iki šiol. Daugelis mokslininkų sutaria, kad pieno cukraus pasisavinimo sutrikimų pa- plitimas įvairių šalių gyventojų populiacijose priklauso nuo rasės ir istorinių bei kul- tūrinių veiksnių. Atlikus epidemiologinius tyrimus paaiškėjo, kad didesnioji žmonijos dalis negali pasisavinti pieno cukraus. Tokiems žmonėms suvalgius pieno sutrinka fi- ziologiniai virškinimo procesai plonojoje žarnoje, atsiranda pieno netoleravimo klini- kinių požymių. Matyt, dėl to daugelis pasaulio tautų, išskyrus europiečius ir išeivius iš Europos kituose žemynuose, neturi šviežio pieno vartojimo maistui tradicijų. Netolygaus laktazės nepakankamumo paplitimo atskirose gyventojų populiacijose reiškiniui paaiškinti buvo pasiūlyta keletas teorijų: dietinio slopinimo, indukcijos, is- torinė-kultūrinė, nišos konstrukcijos ir kitos. Šios teorijos aptariamos monografijoje. Daugiausia autorių palaiko istorinę-kultūrinę teoriją, tačiau jai galutinai įrodyti trūks- ta kai kurių šalių, tarp jų ir Rytų Europos gyventojų, tyrimų rezultatų. Kadangi didelė suaugusių žmonių dalis negali vartoti pieno, nes jų žarnyne yra sumažėjęs laktazės fermentinis aktyvumas ir todėl nehidrolizuojamas pieno cukrus, iškyla keletas pro- blemų. Keičiasi požiūris į sveikų suaugusių žmonių mitybą. Atsiranda poreikis plėsti belaktozių pieno produktų ir sintetinės laktazės gamybą. Iškyla klausimas dėl kalcio papildų vartojimo. Teoriškai tiriant plonosios žarnos fermento laktazės aktyvumo svarbą žmogui, at- siranda naujų klausimų: ar gali šio fermento funkciją pakeisti kiti veiksniai, pavyz- džiui, storosios žarnos mikroflora; ar sumažėjus laktazės aktyvumui ir sutrikus pieno cukraus hidrolizei pablogėja kitų maisto medžiagų pasisavinimas; ar europiečiams, mažiau vartojantiems pieno produktų, atsiranda kalcio ir D vitamino trūkumas; kokie PRATARMĖ 5 metodai yra geriausi šiai enzimopatijai diagnozuoti ir poreikis surasti naujų lengviau pritaikomų klinikinėje praktikoje būdų; pagaliau, ar pirminis laktazės nepakankamu- mas galėtų būti vienas iš genetinių žymenų žmonių etninei ir rasinei priklausomybei diferencijuoti. Analizuojant šią problemą, kyla dar vienas įdomus klausimas: gal kie- kvienos tautos tam tikros mitybos tradicijos apskritai yra genetiškai determinuotos? Pasitvirtinus šiam faktui išeitų, kad negalima be išlygų perimti tolimų mums tautų maitinimosi įpročių, prieš tai neišsiaiškinus savųjų plonosios žarnos fermentų spektro ir galimybių. Antra vertus, pagal istorinę-kultūrinę teoriją, aplinkos veiksniai, tarp jų ir mitybos ypatumai, ilgainiui gali pakeisti genotipą (nišos konstrukcijos teorija). Šiam spėjimui pasitvirtinus, atsivertų plačios, labai įdomios perspektyvos dietologijai ir aps- kritai medicinai. Monografijoje apžvelgta pasaulinė mokslinė literatūra apie plonosios žarnos enzi- mopatijas, daugiausia dėmesio skiriant dažniausioms iš jų – laktazės nepakankamu- mui ir celiakinei ligai, kuriai būdingas antrinis disacharidazių nepakankamumas. Mo- nografijoje pateikiama ne tik kitų autorių publikacijų apžvalga, bet ir savo mokslinių tyrimų šiais klausimais medžiaga. Knygoje pateikta šiuolaikinė intesininių enzimopa- tijų apibrėžtis, terminija ir klasifikacija, išanalizuoti etiologijos ir patogenezės aspektai, amžiaus ir lyties ypatumai, klinikiniai simptomai, komplikacijos, sąsajos su kitomis ligomis, diagnostikos metodai ir gydymas. Monografijoje pateikti autorės vienos ir kartu su doktorantais atliktų originalių lak- tazės nepakankamumo ir celiakinės ligos paplitimo lietuvių populiacijoje, patogenezės ir klinikinės raiškos tyrimų rezultatai. Autorė atliko epidemiologinius ir klinikinius laktazės nepakankamumo tyrimus suaugusių Lietuvos gyventojų populiacijoje. Nusta- tė, kad šios intestininės enzimopatijos dažnis visoje populiacijoje yra 34 % ir jis dides- nis vakarinėje Lietuvos dalyje. Laktazės nepakankamumo paplitimo tarp šalies gyven- tojų duomenys papildo šios enzimopatijos paplitimo regione žemėlapį. Autorė atliko epidemiologinius vaikų hipolaktazijos tyrimus, kurie atskleidė, kad pirminis laktazės nepakankamumas lietuvių populiacijoje pradeda atsirasti 6–7 metų amžiuje (0–8 %), o jo dažnis 17 metų amžiaus grupėje (32 %) statistiškai patikimai nesiskiria nuo šio rodiklio suaugusių Lietuvos gyventojų populiacijoje (34 %). Ši enzimopatija pradeda atsirasti Lietuvos vaikams kiek vėliau negu kituose pasaulio regionuose, suaugusiųjų laktazės aktyvumo dydis susiformuoja paauglystėje, o daugelyje kitų tautų tai atsitinka ikimokykliniame ir jaunesniame mokykliniame amžiuje. Ištyrėme pirminio laktazės nepakankamumo genetinių žymenų dažnį gyventojų grupėje. Tyrimas atskleidė, kad genotipų, siejamų su hipolaktazija, dažniai lietuvių pogrupyje buvo C/C-13910 – 42,5 % ir G/G-22018 – 37,5 %, rusų pogrupyje C/C-13910 – 44,7 % ir G/G-22018 – 39,5 %, lenkų pogrupyje C/C-13910 – 68,0 % ir G/G-22018 – 60,0 %. 6 Šios enzimopatijos genotipo dažnis nesutampa su laktazės aktyvumo sumažėjimo fe- notipu. Netoleruojančių laktozės asmenų grupėje pirminį laktazės nepakankamumą atspindinčių C/C-13910 ir G/G-22018 genotipų dažnis buvo tik 73,6 % ir 64,2%, o genotipų C/T-13910, T/T-13910 ir G/A-22018, A/A-22018, siejamų su pakankamu laktazės aktyvumu, dažnis buvo atitinkamai 20,8 %, 5,7 % ir 30,2 %, 5,7 %. Nustatėme, kad genetinių me- todų jautrumo reikšmės svyravo nuo 64 % (G/A-22018 varianto genotipavimas) iki 74 % (C/T-13910 varianto genotipavimas), o specifiškumas abiem atvejais buvo panašus – ati- tinkamai 63 % iki 64 %. Teigiamo rezultato prognozinės vertės buvo 50–51 %, o nei- giamo rezultato – svyravo nuo 79,3 % iki 83,1 %. Celiakinė liga yra viena iš dažniausių ligų, sukeliančių antrinį plonosios žarnos fer- mentų, tarp jų ir laktazės, aktyvumo sumažėjimą, dėl to sutrinka maisto medžiagų ab- sorbavimas. Disacharidazių aktyvumo sumažėjimas ir celiakinė liga priklauso maisto medžiagų pasisavinimą plonojoje
Load more

Recommended publications
  • List of Approved Ndas for Biological Products That Were Deemed to Be Blas on March 23, 2020
    List of Approved NDAs for Biological Products That Were Deemed to be BLAs on March 23, 2020 On March 23, 2020, an approved application for a biological product under section 505 of the Federal Food, Drug, and Cosmetic Act (FD&C Act) was deemed to be a license for the biological product under section 351 of the Public Health Service Act (PHS Act) (see section 7002(e)(4)(A) of the Biologics Price Competition and Innovation Act of 2009). To enhance transparency and facilitate planning for the March 23, 2020, transition date, FDA compiled a preliminary list of approved applications for biological products under the FD&C Act that were listed in FDA’s Approved Drug Products with Therapeutic Equivalence Evaluations (the Orange Book) and that would be affected by this transition provision. FDA posted this list on the FDA website in December 2018, and periodically updated this list before the March 23, 2020, transition date. The September 2019 update to this preliminary list added certain administratively closed applications related to approved applications for biological products that were on the December 2018 version of this list. The January 2020 update to the preliminary list reflected a change to the definition of “biological product” made by the Further Consolidated Appropriations Act, 2020, which was enacted on December 20, 2019. Section 605 of this Act further amended the definition of a “biological product” in section 351(i) of the PHS Act to remove the parenthetical “(except any chemically synthesized polypeptide)” from the statutory category of “protein.” FDA has provided below a list of each approved application for a biological product under the FD&C Act that was deemed to be a license (i.e., an approved biologics license application (BLA)) for the biological product on March 23, 2020.
    [Show full text]
  • September 2017 ~ Resource #330909
    −This Clinical Resource gives subscribers additional insight related to the Recommendations published in− September 2017 ~ Resource #330909 Medications Stored in the Refrigerator (Information below comes from current U.S. and Canadian product labeling and is current as of date of publication) Proper medication storage is important to ensure medication shelf life until the manufacturer expiration date and to reduce waste. Many meds are recommended to be stored at controlled-room temperature. However, several meds require storage in the refrigerator or freezer to ensure stability. See our toolbox, Medication Storage: Maintaining the Cold Chain, for helpful storage tips and other resources. Though most meds requiring storage at temperatures colder than room temperature should be stored in the refrigerator, expect to see a few meds require storage in the freezer. Some examples of medications requiring frozen storage conditions include: anthrax immune globulin (Anthrasil [U.S. only]), carmustine wafer (Gliadel [U.S. only]), cholera (live) vaccine (Vaxchora), dinoprostone vaginal insert (Cervidil), dinoprostone vaginal suppository (Prostin E2 [U.S.]), varicella vaccine (Varivax [U.S.]; Varivax III [Canada] can be stored in the refrigerator or freezer), zoster vaccine (Zostavax [U.S.]; Zostavax II [Canada] can be stored in the refrigerator or freezer). Use the list below to help identify medications requiring refrigerator storage and become familiar with acceptable temperature excursions from recommended storage conditions. Abbreviations: RT = room temperature Abaloparatide (Tymlos [U.S.]) Aflibercept (Eylea) Amphotericin B (Abelcet, Fungizone) • Once open, may store at RT (68°F to 77°F • May store at RT (77°F [25°C]) for up to Anakinra (Kineret) [20°C to 25°C]) for up to 30 days.
    [Show full text]
  • FDA's Interpretation of the “Deemed to Be a License”
    FDA WATCH FDA’s Interpretation of the “Deemed to be a License” Provision of the Biologics Price Competition and Innovations Act A look at how the FDA will administer the transition of NDAs to BLAs n 2010, Congress passed, and President “deemed to be a license” provision and a Product Time to Approval (months) Aprotinin --- 4 Obama signed, the Patient Protection draft Q&A guidance document providing Beractant --- 1 Iand Affordable Care Act, within which additional details regarding the imple- Calfactant 35 is the Biologics Price Competition and In- mentation of the provision. Choriogonadotropin alfa 10 novations Act (BPCIA)2 that codified the This article provides a summary of how Chorionic gonadotropin* --- ability of the Food and Drug Administra- the FDA will administer the transition of Chymopapain --- Desirudin 33 tion (FDA) to review and approve biosimi- NDAs to BLAs, as described in these two Follitropin alfa 22 lar medicinal products. To harmonize the guidance documents. Follitropin alfa/beta 50 regulatory system for biological products Follicle stimulating hormone --- in the U.S., the BPCIA included Section FDA’s plans for Implementation of the Hyalurionidase 9 7002(e) which requires that all marketing “deemed to be a license” Section of Imiglucerase --- Insulin aspart 21 applications for biological products (as de- the BPCIA Insulin degludec 48 fined in the statute) be submitted as Bio- As written, Section 7002(e)(4) only ap- Insulin detemir 30 logics License Applications (BLA) under plies to approved NDAs and requires their Insulin glargine 12 the Public Health Services Act (PHSA). conversion to BLAs on the date that is 10 Insulin glulisine 10 Furthermore, Section 7002(e)(4) of the years after enactment of the BPCIA.
    [Show full text]
  • Ehealth DSI [Ehdsi V2.2.2-OR] Ehealth DSI – Master Value Set
    MTC eHealth DSI [eHDSI v2.2.2-OR] eHealth DSI – Master Value Set Catalogue Responsible : eHDSI Solution Provider PublishDate : Wed Nov 08 16:16:10 CET 2017 © eHealth DSI eHDSI Solution Provider v2.2.2-OR Wed Nov 08 16:16:10 CET 2017 Page 1 of 490 MTC Table of Contents epSOSActiveIngredient 4 epSOSAdministrativeGender 148 epSOSAdverseEventType 149 epSOSAllergenNoDrugs 150 epSOSBloodGroup 155 epSOSBloodPressure 156 epSOSCodeNoMedication 157 epSOSCodeProb 158 epSOSConfidentiality 159 epSOSCountry 160 epSOSDisplayLabel 167 epSOSDocumentCode 170 epSOSDoseForm 171 epSOSHealthcareProfessionalRoles 184 epSOSIllnessesandDisorders 186 epSOSLanguage 448 epSOSMedicalDevices 458 epSOSNullFavor 461 epSOSPackage 462 © eHealth DSI eHDSI Solution Provider v2.2.2-OR Wed Nov 08 16:16:10 CET 2017 Page 2 of 490 MTC epSOSPersonalRelationship 464 epSOSPregnancyInformation 466 epSOSProcedures 467 epSOSReactionAllergy 470 epSOSResolutionOutcome 472 epSOSRoleClass 473 epSOSRouteofAdministration 474 epSOSSections 477 epSOSSeverity 478 epSOSSocialHistory 479 epSOSStatusCode 480 epSOSSubstitutionCode 481 epSOSTelecomAddress 482 epSOSTimingEvent 483 epSOSUnits 484 epSOSUnknownInformation 487 epSOSVaccine 488 © eHealth DSI eHDSI Solution Provider v2.2.2-OR Wed Nov 08 16:16:10 CET 2017 Page 3 of 490 MTC epSOSActiveIngredient epSOSActiveIngredient Value Set ID 1.3.6.1.4.1.12559.11.10.1.3.1.42.24 TRANSLATIONS Code System ID Code System Version Concept Code Description (FSN) 2.16.840.1.113883.6.73 2017-01 A ALIMENTARY TRACT AND METABOLISM 2.16.840.1.113883.6.73 2017-01
    [Show full text]
  • An Enzyme Is a Form Of
    An Enzyme Is A Form Of Brodie frogmarch her blowball lukewarmly, inobservant and lordless. Obligingly emotional, Matias gyves Quaker and kourbash heptane. Moline Abdel still fossick: parturient and labiovelar Donovan decarbonate quite aloofly but oversleep her knaves meantime. We demonstrate group is recommended rules hold the enzyme is a form an enzyme below shows how you can standard assumption in Many people living with cystic fibrosis and their families face complicated issues related to getting the care they need. These latter part even very rare occasions that form is regulated or significantly at least be degraded. These chemical forms of an instantaneous step in pancreas foundation; scientists should not change tyrosine. The hydrogen bonds with the amount of the enzyme inhibition include breast milk, enzyme is a form an enzyme from solution to measure of the pharmacies identified starting chemicals. Entries for reclassified enzymes transferred from one position also the faucet to fix are followed, for reference, by a comment indicating the fight number. Not restore vision none the enzyme may also quit important roles in conditions such as diabetes lupus and obesity and researchers already are. In reality, the complementarity is rarely perfect, and the interaction of a protein with a ligand often involves changes in the conformation of one or both molecules. Investigate two types of enzyme inhibitors used in regulating enzymatic activity Examine what an enzyme may affect activation energy Introduction Enzymes are. These methods were soon adapted for the production of enzymes. Some enzymes require no chemical groups other be their amino acid residues for activity.
    [Show full text]
  • 2021 Formulary List of Covered Prescription Drugs
    2021 Formulary List of covered prescription drugs This drug list applies to all Individual HMO products and the following Small Group HMO products: Sharp Platinum 90 Performance HMO, Sharp Platinum 90 Performance HMO AI-AN, Sharp Platinum 90 Premier HMO, Sharp Platinum 90 Premier HMO AI-AN, Sharp Gold 80 Performance HMO, Sharp Gold 80 Performance HMO AI-AN, Sharp Gold 80 Premier HMO, Sharp Gold 80 Premier HMO AI-AN, Sharp Silver 70 Performance HMO, Sharp Silver 70 Performance HMO AI-AN, Sharp Silver 70 Premier HMO, Sharp Silver 70 Premier HMO AI-AN, Sharp Silver 73 Performance HMO, Sharp Silver 73 Premier HMO, Sharp Silver 87 Performance HMO, Sharp Silver 87 Premier HMO, Sharp Silver 94 Performance HMO, Sharp Silver 94 Premier HMO, Sharp Bronze 60 Performance HMO, Sharp Bronze 60 Performance HMO AI-AN, Sharp Bronze 60 Premier HDHP HMO, Sharp Bronze 60 Premier HDHP HMO AI-AN, Sharp Minimum Coverage Performance HMO, Sharp $0 Cost Share Performance HMO AI-AN, Sharp $0 Cost Share Premier HMO AI-AN, Sharp Silver 70 Off Exchange Performance HMO, Sharp Silver 70 Off Exchange Premier HMO, Sharp Performance Platinum 90 HMO 0/15 + Child Dental, Sharp Premier Platinum 90 HMO 0/20 + Child Dental, Sharp Performance Gold 80 HMO 350 /25 + Child Dental, Sharp Premier Gold 80 HMO 250/35 + Child Dental, Sharp Performance Silver 70 HMO 2250/50 + Child Dental, Sharp Premier Silver 70 HMO 2250/55 + Child Dental, Sharp Premier Silver 70 HDHP HMO 2500/20% + Child Dental, Sharp Performance Bronze 60 HMO 6300/65 + Child Dental, Sharp Premier Bronze 60 HDHP HMO
    [Show full text]
  • Kaiser Permanente Bernard J. Tyson School of Medicine, Inc. Exclusive Provider Organization (EPO) Student Blanket Health Plan Drug Formulary
    Kaiser Permanente Bernard J. Tyson School of Medicine, Inc. Exclusive Provider Organization (EPO) Student Blanket Health Plan Drug Formulary Effective September 1, 2021 Health Plan Products: Kaiser Permanente Bernard J. Tyson School of Medicine, EPO Student Blanket Health Plan offered by Kaiser Permanente Insurance Company For the most current list of covered medications or for help understanding your KPIC insurance plan benefits, including cost sharing for drugs under the prescription drug benefit and under the medical benefit: Call 1-800-533-1833, TTY 711, Monday through Friday, 7 a.m. to 9 p.m. ET Visit kaiserpermanente.org to: • Find a participating retail pharmacy by ZIP code. • Look up possible lower-cost medication alternatives. • Compare medication pricing and options. • Find an electronic copy of the formulary here. • Get plan coverage information. For cost sharing information for the outpatient prescription drug benefits in your specific plan, please visit kp.org/kpic-websiteTBD The formulary is subject to change and all previous versions of the formulary are no longer in effect. Kaiser Permanente Last updated: September 1, 2021 Table of Contents Informational Section...........................................................................................................................................3 ANTIHISTAMINE DRUGS - Drugs for Allergy.....................................................................................................9 ANTI-INFECTIVE AGENTS - Drugs for Infections...........................................................................................
    [Show full text]
  • Quantum of Effectiveness Evidence in FDA's Approval of Orphan Drugs
    Quantum of Effectiveness Evidence in FDA’s Approval of Orphan Drugs Cataloguing FDA’s Flexibility in Regulating Therapies for Persons with Rare Disorders by Frank J. Sasinowski, M.S., M.P.H., J.D.1 Chairman of the Board National Organization for Rare Disorders One of the key underlying issues facing the development of effective for their intended uses. all drugs, and particularly orphan drugs, is what kind of evi- dence the Food and Drug Administration (FDA) requires for FDA has for many decades acknowledged that there is a need approval. The Federal Food, Drug, and Cosmetic [FD&C] Act for flexibility in applying its standard for approval. For ex- provides that for FDA to grant approval for a new drug, there ample, one of FDA’s regulations states that: “FDA will ap- must be “substantial evidence” of effectiveness derived from prove an application after it determines that the drug meets the “adequate and well-controlled investigations.” This language, statutory standards for safety and effectiveness… While the which dates from 1962, provides leeway for FDA medical re- statutory standards apply to all drugs, the many kinds of drugs viewers to make judgments as to what constitutes “substantial that are subject to the statutory standards and the wide range evidence” of a drug’s effectiveness, that is, of its benefit to of uses for those drugs demand flexibility in applying the stan- patients. dards. Thus FDA is required to exercise its scientific judgment to determine the kind and quantity of data and information an The sole law that applies specifically to orphan drugs, the Or- applicant is required to provide for a particular drug to meet phan Drug Act of 1983, provided financial incentives for drug the statutory standards.” 21 C.F.R.
    [Show full text]
  • Sucraid (Sacrosidase) Oral Solution P&T Approval Date 12/2019, 1/2020, 1/2021 Effective Date 4/1/2021; Oxford Only: 4/1/2021
    UnitedHealthcare Pharmacy Clinical Pharmacy Programs Program Number 2021 P 2179-3 Program Prior Authorization/Medical Necessity Medication Sucraid (sacrosidase) oral solution P&T Approval Date 12/2019, 1/2020, 1/2021 Effective Date 4/1/2021; Oxford only: 4/1/2021 1. Background: Sucraid (sacrosidase) is an oral enzyme replacement therapy indicated for the treatment of genetically determined sucrase deficiency, which is part of congenital sucrase- isomaltase deficiency (CSID). 2. Coverage Criteriona: A. Initial Authorization 1. Sucraid will be approved based on all of the following criteria: a. Diagnosis of congenital sucrase-isomaltase deficiency (CSID) as confirmed by one of the following: i. Duodenal biopsy showing low sucrose activity and normal amounts of other disaccharides -OR- ii. All of the following: a) Stool pH < 6 b) Negative lactose breath test c) Increase in breath hydrogen > 10 ppm when challenged with sucrose after fasting -AND- b. Prescribed by or in consultation with a gastroenterologist or rare disease specialist -AND- c. Will be used with a sucrose-free, low starch diet Authorization will be issued for 3 months. © 2021 UnitedHealthcare Services, Inc. 1 B. Reauthorization 1. Sucraid will be approved based on all of the following criterion: a. Prescribed by or in consultation with a gastroenterologist or rare disease specialist -AND- b. Will be used with a sucrose-free, low starch diet -AND- c. Provider attests that the patient has achieved a clinically meaningful response while on Sucraid therapy, defined as at least a 50% reduction in all of the following: i. Symptoms of abdominal pain, cramps, bloating, gas, vomiting ii. Number of stools per day iii.
    [Show full text]
  • 2020 Aetna Standard Plan
    Plan for your best health Aetna Standard Plan Aetna.com Aetna is the brand name used for products and services provided by one or more of the Aetna group of subsidiary companies, including Aetna Life Insurance Company and its affiliates (Aetna). Aetna Pharmacy Management refers to an internal business unit of Aetna Health Management, LLC. Aetna Pharmacy Management administers, but does not offer, insure or otherwise underwrite the prescription drug benefits portion of your health plan and has no financial responsibility therefor. 2020 Pharmacy Drug Guide - Aetna Standard Plan Table of Contents INFORMATIONAL SECTION..................................................................................................................6 *ADHD/ANTI-NARCOLEPSY/ANTI-OBESITY/ANOREXIANTS* - DRUGS FOR THE NERVOUS SYSTEM.................................................................................................................................16 *ALLERGENIC EXTRACTS/BIOLOGICALS MISC* - BIOLOGICAL AGENTS...............................18 *ALTERNATIVE MEDICINES* - VITAMINS AND MINERALS....................................................... 19 *AMEBICIDES* - DRUGS FOR INFECTIONS.....................................................................................19 *AMINOGLYCOSIDES* - DRUGS FOR INFECTIONS.......................................................................19 *ANALGESICS - ANTI-INFLAMMATORY* - DRUGS FOR PAIN AND FEVER............................19 *ANALGESICS - NONNARCOTIC* - DRUGS FOR PAIN AND FEVER.........................................
    [Show full text]
  • FDA Listing of Established Pharmacologic Class Text Phrases January 2021
    FDA Listing of Established Pharmacologic Class Text Phrases January 2021 FDA EPC Text Phrase PLR regulations require that the following statement is included in the Highlights Indications and Usage heading if a drug is a member of an EPC [see 21 CFR 201.57(a)(6)]: “(Drug) is a (FDA EPC Text Phrase) indicated for Active Moiety Name [indication(s)].” For each listed active moiety, the associated FDA EPC text phrase is included in this document. For more information about how FDA determines the EPC Text Phrase, see the 2009 "Determining EPC for Use in the Highlights" guidance and 2013 "Determining EPC for Use in the Highlights" MAPP 7400.13.
    [Show full text]
  • Enzyme Replacement Therapy - Sucraid® (Sacrosidase Oral Solution)
    Cigna National Formulary Coverage Policy Prior Authorization Enzyme Replacement Therapy - Sucraid® (sacrosidase oral solution) Table of Contents Product Identifer(s) National Formulary Medical Necessity ................ 1 66479 Conditions Not Covered....................................... 2 Background .......................................................... 2 References .......................................................... 2 Revision History ................................................... 2 INSTRUCTIONS FOR USE The following Coverage Policy applies to health benefit plans administered by Cigna Companies. Certain Cigna Companies and/or lines of business only provide utilization review services to clients and do not make coverage determinations. References to standard benefit plan language and coverage determinations do not apply to those clients. Coverage Policies are intended to provide guidance in interpreting certain standard benefit plans administered by Cigna Companies. Please note, the terms of a customer’s particular benefit plan document [Group Service Agreement, Evidence of Coverage, Certificate of Coverage, Summary Plan Description (SPD) or similar plan document] may differ significantly from the standard benefit plans upon which these Coverage Policies are based. For example, a customer’s benefit plan document may contain a specific exclusion related to a topic addressed in a Coverage Policy. In the event of a conflict, a customer’s benefit plan document always supersedes the information in the Coverage Policies. In the absence of a controlling federal or state coverage mandate, benefits are ultimately determined by the terms of the applicable benefit plan document. Coverage determinations in each specific instance require consideration of 1) the terms of the applicable benefit plan document in effect on the date of service; 2) any applicable laws/regulations; 3) any relevant collateral source materials including Coverage Policies and; 4) the specific facts of the particular situation.
    [Show full text]