A BRIEF REVIEW OF ANALYTICAL METHODS FOR THE ESTIMATION OF TTR KINETIC STABILIZERS IN PHARMACEUTICAL FORMULATIONS AND BIOLOGICAL MATRICES

MIGLENA SMERIKAROVA; STANISLAV BOZHANOV; VANIA MASLARSKA

doi:10.22159/ijap.2022v14i5.45612

Authors

MIGLENA SMERIKAROVA Department of Chemistry, Faculty of Pharmacy, Medical University Sofia, Dunav Str. No. 2, Sofia 1000, Bulgaria https://orcid.org/0000-0002-5106-1036
STANISLAV BOZHANOV Department of Chemistry, Faculty of Pharmacy, Medical University Sofia, Dunav Str. No. 2, Sofia 1000, Bulgaria https://orcid.org/0000-0002-6543-9087
VANIA MASLARSKA Department of Chemistry, Faculty of Pharmacy, Medical University Sofia, Dunav Str. No. 2, Sofia 1000, Bulgaria https://orcid.org/0000-0002-5057-4403

DOI:

https://doi.org/10.22159/ijap.2022v14i5.45612

Keywords:

Tafamidis, Diflunisal, Biological matrix, Review, HPLC, Spectrometry

Abstract

Transthyretin kinetic stabilizers are used as first-line drug therapy for transthyretin amyloid polyneuropathy mostly in patients unsuitable for liver transplantation. The two drugs prescribed in clinical practice are Tafamidis and Diflunisal. The European Medicines Agency approved Tafamidis for this prescription in 2011 and 2019 American Food and Drug Association also registered it for the same use. Diflunisal is a non-steroidal anti-inflammatory drug but its structural similarities to Tafamidis determine its “off-label” use for such clinical conditions. This review article represents the various analytical methods available in published literature for the determination of Tafamidis and Diflunisal in bulk drugs, pharmaceutical formulations, and biological matrices. Detailed information about all developed quantitative methods consisting of spectrophotometry, spectrofluorimetry, high-performance liquid chromatography with ultraviolet, fluorescence or diode array detection, liquid chromatography-tandem mass spectrometry, and voltammetry is provided and can be effectively used in the development of new analytical procedures and routine drug manufacturing or clinical practice.

Downloads

Download data is not yet available.

References

Connors LH, Lim A, Prokaeva T, Roskens VA, Costello CE. Tabulation of human transthyretin (TTR) variants, 2003. Amyloid. 2003 Sep;10(3):160-84. doi: 10.3109/13506120308998998, PMID 14640030.

Westermark P, Benson MD, Buxbaum JN, Cohen AS, Frangione B, Ikeda S. A primer of amyloid nomenclature. Amyloid. 2007 Sep;14(3):179-83. doi: 10.1080/13506120701460923, PMID 17701465.

Cakar A, Durmus Tekçe H, Parman Y. Familial amyloid polyneuropathy. [Noro psikiyatr Ars]. Noro Psikiyatr Ars. 2019 May 6;56(2):150-6. doi: 10.29399/npa.23502, PMID 31223250.

Luigetti M, Romano A, Di Paolantonio A, Bisogni G, Sabatelli M. Diagnosis and treatment of hereditary transthyretin amyloidosis (hATTR) polyneuropathy: current perspectives on improving patient care. Ther Clin Risk Manag. 2020 Feb 21;16:109-23. doi: 10.2147/TCRM.S219979. PMID 32110029, PMCID PMC7041433.

Ando Y, Coelho T, Berk JL, Cruz MW, Ericzon BG, Ikeda S. Guideline of transthyretin-related hereditary amyloidosis for clinicians. Orphanet J Rare Dis. 2013 Feb 20;8:31. doi: 10.1186/1750-1172-8-31, PMID 23425518, PMCID PMC3584981.

PubChem, National Library of Medicine [internet]. National library of medicine, National Center for Biotechnology Information, USA; c2022. Available from: https://pubchem.ncbi.nlm.nih.gov. [Last accessed on 21 Jun 2022]

DrugBank online database [Internet]. Database for drug and drug target information, USA; c2022. Available from: https://go.drugbank.com [Last accessed on 21 Jun 2022]

Coelho T, Merlini G, Bulawa CE, Fleming JA, Judge DP, Kelly JW. Mechanism of action and clinical application of tafamidis in hereditary transthyretin amyloidosis. Neurol Ther. 2016 Jun;5(1):1-25. doi: 10.1007/s40120-016-0040-x. PMID 26894299, PMCID PMC4919130.

Adams D. Recent advances in the treatment of familial amyloid polyneuropathy. Ther Adv Neurol Disord. 2013 Mar;6(2):129-39. doi: 10.1177/1756285612470192, PMID 23483184, PMCID PMC3582309.

Sekijima Y. Recent progress in the understanding and treatment of transthyretin amyloidosis. J Clin Pharm Ther. 2014 Jun;39(3):225-33. doi: 10.1111/jcpt.12145, PMID 24749898.

Snetkov P, Morozkina S, Olekhnovich R, Uspenskaya M. Diflunisal targeted delivery systems: a review. Materials (Basel). 2021 Nov 6;14(21):6687. doi: 10.3390/ma14216687, PMID 34772213, PMCID PMC8588122.

Obici L, Merlini G. An overview of drugs currently under investigation for the treatment of transthyretin-related hereditary amyloidosis. Expert Opin Investig Drugs. 2014 Sep;23(9):1239-51. doi: 10.1517/13543784.2014.922541. PMID 25003808.

Burton A, Castano A, Bruno M, Riley S, Schumacher J, Sultan MB. Drug discovery and development in rare diseases: taking a closer look at the tafamidis story. Drug Des Devel Ther. 2021;15:1225-43. doi: 10.2147/DDDT.S289772. PMID 33776421.

Gundapaneni BK, Sultan MB, Keohane DJ, Schwartz JH. Tafamidis delays neurological progression comparably across Val30Met and non-Val30Met genotypes in transthyretin familial amyloid polyneuropathy. Eur J Neurol. 2018 Mar;25(3):464-8. doi: 10.1111/ene.13510. PMID 29115008, PMCID PMC5838526.

Klamerus KJ, Watsky E, Moller R, Wang R, Riley S. The effect of tafamidis on the QTc interval in healthy subjects. Br J Clin Pharmacol. 2015 Jun;79(6):918-25. doi: 10.1111/bcp.12561, PMID 25546001, PMCID PMC4456124.

McKeage K, Lyseng-Williamson KA, Scott LJ. Tafamidis in transthyretin amyloidosis: a guide to its use in delaying peripheral neurological impairment in patients with stage 1 polyneuropathy. Drugs Ther Perspect. 2017;33(2):47-53, doi: 10.1007/s40267-016-0368-4.

Kerschen P, Plante Bordeneuve V. Current and future treatment approaches in transthyretin familial amyloid polyneuropathy. Curr Treat Options Neurol. 2016 Dec;18(12):53. doi: 10.1007/s11940-016-0436-z, PMID 27873215.

Sekijima Y, Tojo K, Morita H, Koyama J, Ikeda S. Safety and efficacy of long-term diflunisal administration in hereditary transthyretin (ATTR) amyloidosis. Amyloid. 2015;22(2):79-83. doi: 10.3109/13506129.2014.997872. PMID 26017328.

Gertz MA, Mauermann ML, Grogan M, Coelho T. Advances in the treatment of hereditary transthyretin amyloidosis: a review. Brain Behav. 2019 Sep;9(9):e01371. doi: 10.1002/brb3.1371. PMID 31368669, PMCID PMC6749475.

Miller SR, Sekijima Y, Kelly JW. Native state stabilization by NSAIDs inhibits transthyretin amyloidogenesis from the most common familial disease variants. Lab Invest. 2004 May;84(5):545-52. doi: 10.1038/labinvest.3700059, PMID 14968122.

Tojo K, Sekijima Y, Kelly JW, Ikeda S. Diflunisal stabilizes familial amyloid polyneuropathy-associated transthyretin variant tetramers in serum against dissociation required for amyloidogenesis. Neurosci Res. 2006 Dec;56(4):441-9. doi: 10.1016/j.neures.2006.08.014. PMID 17028027.

Tempero KF, Cirillo VJ, Steelman SL. Diflunisal: a review of pharmacokinetic and pharmacodynamic properties, drug interactions, and special tolerability studies in humans. Br J Clin Pharmacol. 1977 Feb;4 Suppl 1:31S-6S. doi: 10.1111/j.1365-2125.1977.tb04511.x. PMID 328032, PMCID PMC1428837.

Coelho T, Maia LF, Martins da Silva A, Waddington Cruz M, Plante Bordeneuve V, Lozeron P. Tafamidis for transthyretin familial amyloid polyneuropathy: a randomized, controlled trial. Neurology. 2012 Aug 21;79(8):785-92. doi: 10.1212/WNL.0b013e3182661eb1. PMID 22843282, PMCID PMC4098875.

Coelho T, Maia LF, da Silva AM, Cruz MW, Plante Bordeneuve V, Suhr OB. Long-term effects of tafamidis for the treatment of transthyretin familial amyloid polyneuropathy. J Neurol. 2013 Nov;260(11):2802-14. doi: 10.1007/s00415-013-7051-7. PMID 23974642, PMCID PMC3825212.

Lozeron P, Theaudin M, Mincheva Z, Ducot B, Lacroix C, Adams D. Effect on disability and safety of tafamidis in late onset of Met30 transthyretin familial amyloid polyneuropathy. Eur J Neurol. 2013 Dec;20(12):1539-45. doi: 10.1111/ene.12225. PMID 23834402.

Merlini G, Plante Bordeneuve V, Judge DP, Schmidt H, Obici L, Perlini S. Effects of tafamidis on transthyretin stabilization and clinical outcomes in patients with non-Val30Met transthyretin amyloidosis. J Cardiovasc Transl Res. 2013 Dec;6(6):1011-20. doi: 10.1007/s12265-013-9512-x. PMID 24101373, PMCID PMC3838581.

Barroso FA, Judge DP, Ebede B, Li H, Stewart M, Amass L. Long-term safety and efficacy of tafamidis for the treatment of hereditary transthyretin amyloid polyneuropathy: results up to 6 years. Amyloid. 2017 Sep;24(3):194-204. doi: 10.1080/13506129.2017.1357545. PMID 28758793.

Cortese A, Vita G, Luigetti M, Russo M, Bisogni G, Sabatelli M. Erratum to: monitoring effectiveness and safety of Tafamidis in transthyretin amyloidosis in Italy: a longitudinal multicenter study in a non-endemic area. J NeurolJ Neurol. 2016;263(5):925-6. doi: 10.1007/s00415-016-8116-1. PMID 27098978.

Plante Bordeneuve V, Gorram F, Salhi H, Nordine T, Ayache SS, Le Corvoisier P. Long-term treatment of transthyretin familial amyloid polyneuropathy with tafamidis: a clinical and neurophysiological study. J Neurol. 2017 Feb;264(2):268-76. doi: 10.1007/s00415-016-8337-3. PMID 27878441.

Maurer MS, Grogan DR, Judge DP, Mundayat R, Packman J, Lombardo I. Tafamidis in transthyretin amyloid cardiomyopathy: effects on transthyretin stabilization and clinical outcomes. Circ Heart Fail. 2015 May;8(3):519-26. doi: 10.1161/CIRCHEARTFAILURE.113.000890. PMID 25872787.

Maurer MS, Schwartz JH, Gundapaneni B, Elliott PM, Merlini G, Waddington-Cruz M. Tafamidis treatment for patients with transthyretin amyloid cardiomyopathy. N Engl J Med. 2018 Sep 13;379(11):1007-16. doi: 10.1056/NEJMoa1805689. PMID 30145929.

Damy T, Judge DP, Kristen AV, Berthet K, Li H, Aarts J. Cardiac findings and events observed in an open-label clinical trial of tafamidis in patients with non-Val30Met and non-Val122Ile hereditary transthyretin amyloidosis. J Cardiovasc Transl Res. 2015 Mar;8(2):117-27. doi: 10.1007/s12265-015-9613-9. PMID 25743445, PMCID PMC4382536.

Rapezzi C, Elliott P, Damy T, Nativi Nicolau J, Berk JL, Velazquez EJ. Efficacy of tafamidis in patients with hereditary and wild-type transthyretin amyloid cardiomyopathy: further analyses from ATTR-ACT. JACC Heart Fail. 2021 Feb;9(2):115-23. doi: 10.1016/j.jchf.2020.09.011. PMID 33309574.

Ishii T, Hirano Y, Matsumoto N, Takata A, Sekijima Y, Ueda M. Characteristics of patients with hereditary transthyretin amyloidosis and an evaluation of the safety of tafamidis meglumine in Japan: an interim analysis of an all-case postmarketing surveillance. Clin Ther. 2020 Sep;42(9):1728-37.e6. doi: 10.1016/j.clinthera.2020.07.001. PMID 32800381.

Huber P, Flynn A, Sultan MB, Li H, Rill D, Ebede B. A comprehensive safety profile of tafamidis in patients with transthyretin amyloid polyneuropathy. Amyloid. 2019 Dec;26(4):203-9. doi: 10.1080/13506129.2019.1643714. PMID 31353964.

Suhr OB, Conceicao IM, Karayal ON, Mandel FS, Huertas PE, Ericzon BG. Post hoc analysis of nutritional status in patients with transthyretin familial amyloid polyneuropathy: impact of tafamidis. Neurol Ther. 2014 Dec 11;3(2):101-12. doi: 10.1007/s40120-014-0023-8, PMID 26000226, PMCID PMC4386428.

Amass L, Li H, Gundapaneni BK, Schwartz JH, Keohane DJ. Influence of baseline neurologic severity on disease progression and the associated disease-modifying effects of tafamidis in patients with transthyretin amyloid polyneuropathy. Orphanet J Rare Dis. 2018 Dec 17;13(1):225. doi: 10.1186/s13023-018-0947-7, PMID 30558645, PMCID PMC6296038.

Merlini G, Coelho T, Waddington Cruz M, Li H, Stewart M, Ebede B. Evaluation of mortality during long-term treatment with tafamidis for transthyretin amyloidosis with polyneuropathy: clinical trial results up to 8.5 Years. Neurol Ther. 2020 Jun;9(1):105-15. doi: 10.1007/s40120-020-00180-w. PMID 32107748, PMCID PMC7229124.

Keohane D, Schwartz J, Gundapaneni B, Stewart M, Amass L. Tafamidis delays disease progression in patients with early stage transthyretin familial amyloid polyneuropathy: additional supportive analyses from the pivotal trial. Amyloid. 2017 Mar;24(1):30-6. doi: 10.1080/13506129.2017.1301419. PMID 28393570.

Russo M, Gentile L, Di Stefano V, Di Bella G, Minutoli F, Toscano A. Use of drugs for ATTRv amyloidosis in the real world: how therapy is changing survival in a non-endemic area. Brain Sci. 2021 Apr 27;11(5):545. doi: 10.3390/brainsci11050545, PMID 33925301, PMCID PMC8146901.

Müller ML, Butler J, Heidecker B. Emerging therapies in transthyretin amyloidosis-a new wave of hope after years of stagnancy? Eur J Heart Fail. 2020 Jan;22(1):39-53. doi: 10.1002/ejhf.1695. PMID 31912620.

Abdel Hamid ME, Najib NM, Suleiman MS, El-Sayed YM. Differential spectrophotometric, fluorimetric and high-performance liquid chromatographic determination of diflunisal and its tablets. Analyst. 1987 Nov;112(11):1527-30. doi: 10.1039/an9871201527, PMID 3439602.

Ioannou PC, Lianidou ES, Konstantianos DG. Simple, rapid and sensitive spectrofluorimetric determination of diflunisal in serum and urine based on its ternary complex with terbium and EDTA. Anal Chim Acta. 1995;300(1-3):237-41. doi: 10.1016/0003-2670(94)00415-I.

Perez Ruiz T, Martinez Lozano CM, Tomas V, Carpena J. Sensitive synchronous spectrofluorimetric methods for the determination of naproxen and diflunisal in serum. Fresenius J Anal Chem. 1998;361(5):492-5. doi: 10.1007/s002160050931.

Maher HM. Simultaneous determination of naproxen and diflunisal using synchronous luminescence spectrometry. J Fluoresc. 2008 Sep;18(5):909-17. doi: 10.1007/s10895-008-0322-5. PMID 18256908.

Rappley I, Monteiro C, Novais M, Baranczak A, Solis G, Wiseman RL. Quantification of transthyretin kinetic stability in human plasma using subunit exchange. Biochemistry. 2014 Apr 1;53(12):1993-2006. doi: 10.1021/bi500171j. PMID 24661308, PMCID PMC3977577.

Smerikarova M, Bozhanov S, Maslarska V, Tournev I. Determination of tafamidis plasma concentrations in amyloidosis patients with Glu89Gln mutation by HPLC-UV detection. J Chromatogr Sci. 2021 Dec 11:bmab132. doi: 10.1093/chromsci/bmab132. PMID 34897414.

Lockwood PA, Le VH, O’Gorman MT, Patterson TA, Sultan MB, Tankisheva E. The bioequivalence of tafamidis 61-mg free acid capsules and tafamidis meglumine 4 × 20-mg capsules in healthy volunteers. Clin Pharmacol Drug Dev. 2020 Oct;9(7):849-54. doi: 10.1002/cpdd.789. PMID: 32196976, PMC7754314.

Hyun HC, Jeong JW, Kim HR, Oh JH, Lee JH, Choi S, Kim YS, Koo TS. Development and validation of a liquid chromatography-tandem mass spectrometry method for the assay of tafamidis in rat plasma: application to a pharmacokinetic study in rats. J Pharm Biomed Anal. 2017 Apr 15;137:90-5. doi: 10.1016/j.jpba.2017.01.020. PMID: 28107689.

Gao L, Xing J, Liu W. Determination of diflunisal in plasma by RP-HPLC after solid-liquid extraction. Yao Xue Xue Bao. 1998;33(4):286-9. PMID: 11939069.

Van Loenhout JW, Ketelaars HC, Gribnau FW, Van Ginneken CA, Tan Y. Rapid high-performance liquid chromatographic method for the quantitative determination of diflunisal in plasma. J Chromatogr. 1980 Jun 13;182(3-4):487-91. doi: 10.1016/s0378-4347(00)81506-6, PMID: 7391195.

Schwartz M, Chiou R, Stubbs RJ, Bayne WF. Determination of diflunisal in human plasma and urine by fast high-performance liquid chromatography. J Chromatogr. 1986 Aug 2;380(2):420-4. doi: 10.1016/s0378-4347(00)83673-7, PMID: 3760071.

Ray JE, Day RO. High-performance liquid chromatographic analysis of diflunisal in plasma and urine: application to pharmacokinetic studies in two normal volunteers. J Pharm Sci. 1983 Dec;72(12):1403-5. doi: 10.1002/jps.2600721209, PMID: 6663474.

Shaalan RA, Belal TS. Validated stability-indicating HPLC-DAD method for the simultaneous determination of diclofenac sodium and diflunisal in their combined dosage form. Sci Pharm. 2013 Mar 17;81(3):713-31. doi: 10.3797/scipharm.1301-24, PMID: 24106669, PMCID: PMC3791935.

Maher HM. Development and validation of a stability-indicating HPLC-DAD method with ANN optimization for the determination of diflunisal and naproxen in pharmaceutical tablets. J Liq Chromatogr Relat Technol. 2014;37(5):634-52. doi: 10.1080/10826076.2012.758134.

Magdy G, Abdel Hakiem AF, Belal F, Abdel Megied AM. A novel quality by design approach for development and validation of a green reversed-phase HPLC method with fluorescence detection for the simultaneous determination of lesinurad, febuxostat, and diflunisal: application to human plasma. J Sep Sci. 2021 Jun;44(11):2177-88. doi: 10.1002/jssc.202100016. PMID: 33773042.

Waahlin Boll E, Brantmark B, Hanson A, Melander A, Nilsson C. High-pressure liquid chromatographic determination of acetylsalicylic acid, salicylic acid, diflunisal, indomethacin, indoprofen and indobufen. Eur J Clin Pharmacol. 1981;20(5):375-8. doi: 10.1007/BF00615408, PMID: 7286048.

Hansen Moøller J, Dalgaard L, Hansen SH. Reversed-phase high-performance liquid chromatographic assay for the simultaneous determination of diflunisal and its glucuronides in serum and urine. Rearrangement of the 1-O-acylglucuronide. J Chromatogr. 1987 Sep 4;420(1):99-109. doi: 10.1016/0378-4347(87)80159-7, PMID: 3667833.

Farid NF, Naguib IA, Moatamed RS, El Ghobashy MR. Separation and determination of diflunisal and its impurity by two chromatographic methods: TLC-densitometry and HPLC. J AOAC Int. 2021 Dec 11;104(6):1719-25. doi: 10.1093/jaoacint/qsab076, PMID: 34051091.

Patel DS, Sharma N, Patel MC, Patel BN, Shrivastav PS, Sanyal M. Sensitive and selective determination of diflunisal in human plasma by LC-MS. J Chromatogr Sci. 2013 Oct;51(9):872-82. doi: 10.1093/chromsci/bms181. PMID: 23144359.

Vidya D, Siva Prasad M, Vishnu Priya M, Roja K, Sreedhar NY. Voltammetric determination of desloratadine in pharmaceutical and human urine samples using glassy carbon electrode. Sri Int J Pharm Pharm Sci. 2014 Oct;6(10):119-22.

Jg MJg M. A new electrochemical sensor based on modified carbon nanotube-graphite mixture paste electrode for voltammetric determination of resorcinol. Asian J Pharm Clin Res. 2017 Dec 1;10(12):295-300. doi: 10.22159/ajpcr.2017.v10i12.21028.

Hanabaratti RM, Gowda JI, Tuwar SM. Development of a sensor by electro-polymerization of erichrome black-t on glassy carbon electrode and determination of an anti-inflammatory drug diclofenac. Int J Pharm Pharm Sci. 2019 Feb 1;11(2):81-7. doi: 10.22159/ijpps.2019v11i2.30648.

Beltagi AM. Utilization of a montmorillonite-Ca-modified carbon paste electrode for the stripping voltammetric determination of diflunisal in its pharmaceutical formulations and human blood. J Appl Electrochem. 2009;39(12):2375-84. doi: 10.1007/s10800-009-9924-0.

Shaikh T, Uddin S, Talpur FN, Khaskeli AR, Agheem MH, Shah MR. Ultrasensitive determination of piroxicam at diflunisal-derived gold nanoparticle-modified glassy carbon electrode. J Electron Mater. 2017;46(10):5957-66. doi: 10.1007/s11664-017-5573-y.