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Inborn disorder of purine or pyrimidine metabolism

MedGen UID:
48263
Concept ID:
C0034139
Disease or Syndrome
Synonym: Purine-Pyrimidine Metabolism, Inborn Errors
SNOMED CT: Purine and pyrimidine metabolism disorder (238006008); Disorder of purine and pyrimidine metabolism (238006008)
 
Monarch Initiative: MONDO:0019254
Orphanet: ORPHA79224

Definition

Dysfunctions in the metabolism of PURINES or PYRIMIDINES resulting from inborn genetic mutations that are inherited or acquired in utero. [from MeSH]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVInborn disorder of purine or pyrimidine metabolism
Follow this link to review classifications for Inborn disorder of purine or pyrimidine metabolism in Orphanet.

Professional guidelines

PubMed

Sookoian S, Pirola CJ
Cell 2024 Jun 6;187(12):2897-2897.e1. doi: 10.1016/j.cell.2024.05.009. PMID: 38848671
Rausch CR, Jabbour EJ, Kantarjian HM, Kadia TM
Cancer 2020 Mar 15;126(6):1152-1160. Epub 2019 Dec 3 doi: 10.1002/cncr.32606. PMID: 31794095
Chu CM, Lowder JL
Am J Obstet Gynecol 2018 Jul;219(1):40-51. Epub 2018 Jan 2 doi: 10.1016/j.ajog.2017.12.231. PMID: 29305250

Recent clinical studies

Etiology

Luukkonen PK, Sakuma I, Gaspar RC, Mooring M, Nasiri A, Kahn M, Zhang XM, Zhang D, Sammalkorpi H, Penttilä AK, Orho-Melander M, Arola J, Juuti A, Zhang X, Yimlamai D, Yki-Järvinen H, Petersen KF, Shulman GI
Proc Natl Acad Sci U S A 2023 Jan 24;120(4):e2217543120. Epub 2023 Jan 20 doi: 10.1073/pnas.2217543120. PMID: 36669104Free PMC Article
Mercuri E, Baranello G, Boespflug-Tanguy O, De Waele L, Goemans N, Kirschner J, Masson R, Mazzone ES, Pechmann A, Pera MC, Vuillerot C, Bader-Weder S, Gerber M, Gorni K, Hoffart J, Kletzl H, Martin C, McIver T, Scalco RS, Yeung WY, Servais L; SUNFISH Working Group
Eur J Neurol 2023 Jul;30(7):1945-1956. Epub 2022 Aug 1 doi: 10.1111/ene.15499. PMID: 35837793
Ghiboub M, Penny S, Verburgt CM, Boneh RS, Wine E, Cohen A, Dunn KA, Pinto DM, Benninga MA, de Jonge WJ, Levine A, Van Limbergen JE
Gastroenterology 2022 Oct;163(4):922-936.e15. Epub 2022 Jun 7 doi: 10.1053/j.gastro.2022.05.050. PMID: 35679949
Jurecka A, Tylki-Szymanska A
Mol Genet Metab 2022 Jul;136(3):164-176. Epub 2022 Feb 19 doi: 10.1016/j.ymgme.2022.02.007. PMID: 35216884
Sun S, Fu J
Bioorg Med Chem Lett 2018 Nov 1;28(20):3283-3289. Epub 2018 Sep 14 doi: 10.1016/j.bmcl.2018.09.016. PMID: 30243589

Diagnosis

Spanogiannopoulos P, Kyaw TS, Guthrie BGH, Bradley PH, Lee JV, Melamed J, Malig YNA, Lam KN, Gempis D, Sandy M, Kidder W, Van Blarigan EL, Atreya CE, Venook A, Gerona RR, Goga A, Pollard KS, Turnbaugh PJ
Nat Microbiol 2022 Oct;7(10):1605-1620. Epub 2022 Sep 22 doi: 10.1038/s41564-022-01226-5. PMID: 36138165Free PMC Article
Ghiboub M, Penny S, Verburgt CM, Boneh RS, Wine E, Cohen A, Dunn KA, Pinto DM, Benninga MA, de Jonge WJ, Levine A, Van Limbergen JE
Gastroenterology 2022 Oct;163(4):922-936.e15. Epub 2022 Jun 7 doi: 10.1053/j.gastro.2022.05.050. PMID: 35679949
Jurecka A, Tylki-Szymanska A
Mol Genet Metab 2022 Jul;136(3):164-176. Epub 2022 Feb 19 doi: 10.1016/j.ymgme.2022.02.007. PMID: 35216884
Cantor JR, Abu-Remaileh M, Kanarek N, Freinkman E, Gao X, Louissaint A Jr, Lewis CA, Sabatini DM
Cell 2017 Apr 6;169(2):258-272.e17. doi: 10.1016/j.cell.2017.03.023. PMID: 28388410Free PMC Article
Peng B, Lloyd P, Schran H
Clin Pharmacokinet 2005;44(9):879-94. doi: 10.2165/00003088-200544090-00001. PMID: 16122278

Therapy

Chifotides HT, Bose P, Verstovsek S
J Hematol Oncol 2022 Jan 19;15(1):7. doi: 10.1186/s13045-021-01157-4. PMID: 35045875Free PMC Article
Markham A, Duggan S
Drugs 2021 Apr;81(6):721-726. doi: 10.1007/s40265-021-01496-z. PMID: 33770393
Kaur Gill A, Bansal Y, Bhandari R, Kaur S, Kaur J, Singh R, Kuhad A, Kuhad A
Drugs Today (Barc) 2019 Jul;55(7):423-437. doi: 10.1358/dot.2019.55.7.2958474. PMID: 31347611
Zhou L, Jiang Y, Luo Q, Li L, Jia L
Mol Cancer 2019 Apr 3;18(1):77. doi: 10.1186/s12943-019-0979-1. PMID: 30943988Free PMC Article
Weissman SM
JAMA 1966 Jan 3;195(1):27-30. PMID: 5323048

Prognosis

Luukkonen PK, Sakuma I, Gaspar RC, Mooring M, Nasiri A, Kahn M, Zhang XM, Zhang D, Sammalkorpi H, Penttilä AK, Orho-Melander M, Arola J, Juuti A, Zhang X, Yimlamai D, Yki-Järvinen H, Petersen KF, Shulman GI
Proc Natl Acad Sci U S A 2023 Jan 24;120(4):e2217543120. Epub 2023 Jan 20 doi: 10.1073/pnas.2217543120. PMID: 36669104Free PMC Article
Armstrong PW, Pieske B, Anstrom KJ, Ezekowitz J, Hernandez AF, Butler J, Lam CSP, Ponikowski P, Voors AA, Jia G, McNulty SE, Patel MJ, Roessig L, Koglin J, O'Connor CM; VICTORIA Study Group
N Engl J Med 2020 May 14;382(20):1883-1893. Epub 2020 Mar 28 doi: 10.1056/NEJMoa1915928. PMID: 32222134
Shukla SK, Purohit V, Mehla K, Gunda V, Chaika NV, Vernucci E, King RJ, Abrego J, Goode GD, Dasgupta A, Illies AL, Gebregiworgis T, Dai B, Augustine JJ, Murthy D, Attri KS, Mashadova O, Grandgenett PM, Powers R, Ly QP, Lazenby AJ, Grem JL, Yu F, Matés JM, Asara JM, Kim JW, Hankins JH, Weekes C, Hollingsworth MA, Serkova NJ, Sasson AR, Fleming JB, Oliveto JM, Lyssiotis CA, Cantley LC, Berim L, Singh PK
Cancer Cell 2017 Jul 10;32(1):71-87.e7. doi: 10.1016/j.ccell.2017.06.004. PMID: 28697344Free PMC Article
Theuretzbacher U, Ihle F, Derendorf H
Clin Pharmacokinet 2006;45(7):649-63. doi: 10.2165/00003088-200645070-00002. PMID: 16802848
Peng B, Lloyd P, Schran H
Clin Pharmacokinet 2005;44(9):879-94. doi: 10.2165/00003088-200544090-00001. PMID: 16122278

Clinical prediction guides

Vandekeere S, Dubois C, Kalucka J, Sullivan MR, García-Caballero M, Goveia J, Chen R, Diehl FF, Bar-Lev L, Souffreau J, Pircher A, Kumar S, Vinckier S, Hirabayashi Y, Furuya S, Schoonjans L, Eelen G, Ghesquière B, Keshet E, Li X, Vander Heiden MG, Dewerchin M, Carmeliet P
Cell Metab 2018 Oct 2;28(4):573-587.e13. Epub 2018 Jul 12 doi: 10.1016/j.cmet.2018.06.009. PMID: 30017355
Jurecka A, Zikanova M, Kmoch S, Tylki-Szymańska A
J Inherit Metab Dis 2015 Mar;38(2):231-42. Epub 2014 Aug 12 doi: 10.1007/s10545-014-9755-y. PMID: 25112391Free PMC Article
Korashy HM, Rahman AF, Kassem MG
Profiles Drug Subst Excip Relat Methodol 2014;39:205-37. doi: 10.1016/B978-0-12-800173-8.00004-0. PMID: 24794907
Theuretzbacher U, Ihle F, Derendorf H
Clin Pharmacokinet 2006;45(7):649-63. doi: 10.2165/00003088-200645070-00002. PMID: 16802848
Peng B, Lloyd P, Schran H
Clin Pharmacokinet 2005;44(9):879-94. doi: 10.2165/00003088-200544090-00001. PMID: 16122278

Recent systematic reviews

Abiramalatha T, Thanigainathan S, Ramaswamy VV, Pressler R, Brigo F, Hartmann H
Cochrane Database Syst Rev 2023 Oct 24;10(10):CD014967. doi: 10.1002/14651858.CD014967.pub2. PMID: 37873971Free PMC Article
Mateos-Haro M, Novoa-Candia M, Sánchez Vanegas G, Correa-Pérez A, Gaetano Gil A, Fernández-García S, Ortega-Quijano D, Urueña Rodriguez MG, Saceda-Corralo D, Bennouna-Dalero T, Giraldo L, Tomlinson J, Vaño-Galván S, Zamora J
Cochrane Database Syst Rev 2023 Oct 23;10(10):CD013719. doi: 10.1002/14651858.CD013719.pub2. PMID: 37870096Free PMC Article
Bahardoust M, Eghbali F, Shahmiri SS, Alijanpour A, Yarigholi F, Valizadeh R, Madankan A, Pouraskari AB, Ashtarinezhad B, Farokhi H, Sarafraz H, Khanafshar E
Obes Surg 2022 Sep;32(9):3104-3112. Epub 2022 Jul 1 doi: 10.1007/s11695-022-06178-7. PMID: 35776243
Jasielski P, Piędel F, Piwek M, Rocka A, Petit V, Rejdak K
Nutrients 2020 Oct 12;12(10) doi: 10.3390/nu12103113. PMID: 33053828Free PMC Article
Amstutz U, Henricks LM, Offer SM, Barbarino J, Schellens JHM, Swen JJ, Klein TE, McLeod HL, Caudle KE, Diasio RB, Schwab M
Clin Pharmacol Ther 2018 Feb;103(2):210-216. Epub 2017 Nov 20 doi: 10.1002/cpt.911. PMID: 29152729Free PMC Article

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