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PIK3R1 Deficiency

MedGen UID:
1784355
Concept ID:
C5447337
Disease or Syndrome

Definition

A deficiency caused by mutation(s) in the PIK3R1 gene, encoding phosphatidylinositol 3-kinase regulatory subunit alpha. It is associated with autosomal recessive agammaglobulinemia 7, immunodeficiency 36 and SHORT (short stature, hyperextensibility of joints or hernia, ocular depression, Rieger anomaly, teething delay) syndrome. [from NCI]

Term Hierarchy

Professional guidelines

PubMed

Soyak Aytekin E, Keskin A, Tan C, Yalçın E, Dogru D, Ozcelik U, Kiper N, Tezcan I, Cagdas D
Scand J Immunol 2021 Oct;94(4):e13084. Epub 2021 Jul 28 doi: 10.1111/sji.13084. PMID: 34780073

Recent clinical studies

Etiology

Maccari ME, Wolkewitz M, Schwab C, Lorenzini T, Leiding JW, Aladjdi N, Abolhassani H, Abou-Chahla W, Aiuti A, Azarnoush S, Baris S, Barlogis V, Barzaghi F, Baumann U, Bloomfield M, Bohynikova N, Bodet D, Boutboul D, Bucciol G, Buckland MS, Burns SO, Cancrini C, Cathébras P, Cavazzana M, Cheminant M, Chinello M, Ciznar P, Coulter TI, D'Aveni M, Ekwall O, Eric Z, Eren E, Fasth A, Frange P, Fournier B, Garcia-Prat M, Gardembas M, Geier C, Ghosh S, Goda V, Hammarström L, Hauck F, Heeg M, Heropolitanska-Pliszka E, Hilfanova A, Jolles S, Karakoc-Aydiner E, Kindle GR, Kiykim A, Klemann C, Koletsi P, Koltan S, Kondratenko I, Körholz J, Krüger R, Jeziorski E, Levy R, Le Guenno G, Lefevre G, Lougaris V, Marzollo A, Mahlaoui N, Malphettes M, Meinhardt A, Merlin E, Meyts I, Milota T, Moreira F, Moshous D, Mukhina A, Neth O, Neubert J, Neven B, Nieters A, Nove-Josserand R, Oksenhendler E, Ozen A, Olbrich P, Perlat A, Pac M, Schmid JP, Pacillo L, Parra-Martinez A, Paschenko O, Pellier I, Sefer AP, Plebani A, Plantaz D, Prader S, Raffray L, Ritterbusch H, Riviere JG, Rivalta B, Rusch S, Sakovich I, Savic S, Scheible R, Schleinitz N, Schuetz C, Schulz A, Sediva A, Semeraro M, Sharapova SO, Shcherbina A, Slatter MA, Sogkas G, Soler-Palacin P, Speckmann C, Stephan JL, Suarez F, Tommasini A, Trück J, Uhlmann A, van Aerde KJ, van Montfrans J, von Bernuth H, Warnatz K, Williams T, Worth AJJ, Ip W, Picard C, Catherinot E, Nademi Z, Grimbacher B, Forbes Satter LR, Kracker S, Chandra A, Condliffe AM, Ehl S; European Society for Immunodeficiencies Registry Working Party
J Allergy Clin Immunol 2023 Oct;152(4):984-996.e10. Epub 2023 Jun 28 doi: 10.1016/j.jaci.2023.06.015. PMID: 37390899
Wang J, Xiu J, Farrell A, Baca Y, Arai H, Battaglin F, Kawanishi N, Soni S, Zhang W, Millstein J, Shields AF, Grothey A, Weinberg BA, Marshall JL, Lou E, Khushman M, Sohal DPS, Hall MJ, Liu T, Oberley M, Spetzler D, Korn WM, Shen L, Lenz HJ
Lancet Oncol 2023 Feb;24(2):151-161. Epub 2023 Jan 18 doi: 10.1016/S1470-2045(22)00783-5. PMID: 36681091Free PMC Article
López-Nevado M, González-Granado LI, Ruiz-García R, Pleguezuelo D, Cabrera-Marante O, Salmón N, Blanco-Lobo P, Domínguez-Pinilla N, Rodríguez-Pena R, Sebastián E, Cruz-Rojo J, Olbrich P, Ruiz-Contreras J, Paz-Artal E, Neth O, Allende LM
Front Immunol 2021;12:671755. Epub 2021 Aug 10 doi: 10.3389/fimmu.2021.671755. PMID: 34447369Free PMC Article
Nunes-Santos CJ, Uzel G, Rosenzweig SD
J Allergy Clin Immunol 2019 May;143(5):1676-1687. doi: 10.1016/j.jaci.2019.03.017. PMID: 31060715
Cancer Genome Atlas Research Network
Nature 2008 Oct 23;455(7216):1061-8. Epub 2008 Sep 4 doi: 10.1038/nature07385. PMID: 18772890Free PMC Article

Diagnosis

Moriya K, Mitsui-Sekinaka K, Sekinaka Y, Endo A, Kanegane H, Morio T, Imai K, Nonoyama S
Immunol Med 2023 Dec;46(4):153-157. Epub 2023 May 13 doi: 10.1080/25785826.2023.2210366. PMID: 37178059
López-Nevado M, González-Granado LI, Ruiz-García R, Pleguezuelo D, Cabrera-Marante O, Salmón N, Blanco-Lobo P, Domínguez-Pinilla N, Rodríguez-Pena R, Sebastián E, Cruz-Rojo J, Olbrich P, Ruiz-Contreras J, Paz-Artal E, Neth O, Allende LM
Front Immunol 2021;12:671755. Epub 2021 Aug 10 doi: 10.3389/fimmu.2021.671755. PMID: 34447369Free PMC Article
Coulter TI, Cant AJ
Front Immunol 2018;9:2043. Epub 2018 Sep 7 doi: 10.3389/fimmu.2018.02043. PMID: 30245694Free PMC Article
Oda K, Hamanishi J, Matsuo K, Hasegawa K
Gynecol Oncol 2018 Nov;151(2):381-389. Epub 2018 Sep 12 doi: 10.1016/j.ygyno.2018.09.001. PMID: 30217369Free PMC Article
Domené HM, Fierro-Carrión G
Growth Horm IGF Res 2018 Feb;38:19-23. Epub 2017 Dec 12 doi: 10.1016/j.ghir.2017.12.004. PMID: 29249625

Therapy

Moriya K, Mitsui-Sekinaka K, Sekinaka Y, Endo A, Kanegane H, Morio T, Imai K, Nonoyama S
Immunol Med 2023 Dec;46(4):153-157. Epub 2023 May 13 doi: 10.1080/25785826.2023.2210366. PMID: 37178059
Wang J, Xiu J, Farrell A, Baca Y, Arai H, Battaglin F, Kawanishi N, Soni S, Zhang W, Millstein J, Shields AF, Grothey A, Weinberg BA, Marshall JL, Lou E, Khushman M, Sohal DPS, Hall MJ, Liu T, Oberley M, Spetzler D, Korn WM, Shen L, Lenz HJ
Lancet Oncol 2023 Feb;24(2):151-161. Epub 2023 Jan 18 doi: 10.1016/S1470-2045(22)00783-5. PMID: 36681091Free PMC Article
Coulter TI, Cant AJ
Front Immunol 2018;9:2043. Epub 2018 Sep 7 doi: 10.3389/fimmu.2018.02043. PMID: 30245694Free PMC Article
Cohen JI
Front Immunol 2018;9:237. Epub 2018 Feb 23 doi: 10.3389/fimmu.2018.00237. PMID: 29599765Free PMC Article
Condliffe AM, Chandra A
Front Immunol 2018;9:338. Epub 2018 Mar 5 doi: 10.3389/fimmu.2018.00338. PMID: 29556229Free PMC Article

Prognosis

Maccari ME, Wolkewitz M, Schwab C, Lorenzini T, Leiding JW, Aladjdi N, Abolhassani H, Abou-Chahla W, Aiuti A, Azarnoush S, Baris S, Barlogis V, Barzaghi F, Baumann U, Bloomfield M, Bohynikova N, Bodet D, Boutboul D, Bucciol G, Buckland MS, Burns SO, Cancrini C, Cathébras P, Cavazzana M, Cheminant M, Chinello M, Ciznar P, Coulter TI, D'Aveni M, Ekwall O, Eric Z, Eren E, Fasth A, Frange P, Fournier B, Garcia-Prat M, Gardembas M, Geier C, Ghosh S, Goda V, Hammarström L, Hauck F, Heeg M, Heropolitanska-Pliszka E, Hilfanova A, Jolles S, Karakoc-Aydiner E, Kindle GR, Kiykim A, Klemann C, Koletsi P, Koltan S, Kondratenko I, Körholz J, Krüger R, Jeziorski E, Levy R, Le Guenno G, Lefevre G, Lougaris V, Marzollo A, Mahlaoui N, Malphettes M, Meinhardt A, Merlin E, Meyts I, Milota T, Moreira F, Moshous D, Mukhina A, Neth O, Neubert J, Neven B, Nieters A, Nove-Josserand R, Oksenhendler E, Ozen A, Olbrich P, Perlat A, Pac M, Schmid JP, Pacillo L, Parra-Martinez A, Paschenko O, Pellier I, Sefer AP, Plebani A, Plantaz D, Prader S, Raffray L, Ritterbusch H, Riviere JG, Rivalta B, Rusch S, Sakovich I, Savic S, Scheible R, Schleinitz N, Schuetz C, Schulz A, Sediva A, Semeraro M, Sharapova SO, Shcherbina A, Slatter MA, Sogkas G, Soler-Palacin P, Speckmann C, Stephan JL, Suarez F, Tommasini A, Trück J, Uhlmann A, van Aerde KJ, van Montfrans J, von Bernuth H, Warnatz K, Williams T, Worth AJJ, Ip W, Picard C, Catherinot E, Nademi Z, Grimbacher B, Forbes Satter LR, Kracker S, Chandra A, Condliffe AM, Ehl S; European Society for Immunodeficiencies Registry Working Party
J Allergy Clin Immunol 2023 Oct;152(4):984-996.e10. Epub 2023 Jun 28 doi: 10.1016/j.jaci.2023.06.015. PMID: 37390899
Wang J, Xiu J, Farrell A, Baca Y, Arai H, Battaglin F, Kawanishi N, Soni S, Zhang W, Millstein J, Shields AF, Grothey A, Weinberg BA, Marshall JL, Lou E, Khushman M, Sohal DPS, Hall MJ, Liu T, Oberley M, Spetzler D, Korn WM, Shen L, Lenz HJ
Lancet Oncol 2023 Feb;24(2):151-161. Epub 2023 Jan 18 doi: 10.1016/S1470-2045(22)00783-5. PMID: 36681091Free PMC Article
Lougaris V, Cancrini C, Rivalta B, Castagnoli R, Giardino G, Volpi S, Leonardi L, La Torre F, Federici S, Corrente S, Cinicola BL, Soresina A, Marseglia GL, Cardinale F
Pediatr Allergy Immunol 2022 Jan;33 Suppl 27(Suppl 27):69-72. doi: 10.1111/pai.13634. PMID: 35080319Free PMC Article
Hu Y, Chen C, Tong X, Chen S, Hu X, Pan B, Sun X, Chen Z, Shi X, Hu Y, Shen X, Xue X, Lu M
Cell Death Dis 2021 Sep 9;12(9):842. doi: 10.1038/s41419-021-04127-3. PMID: 34504059Free PMC Article
López-Nevado M, González-Granado LI, Ruiz-García R, Pleguezuelo D, Cabrera-Marante O, Salmón N, Blanco-Lobo P, Domínguez-Pinilla N, Rodríguez-Pena R, Sebastián E, Cruz-Rojo J, Olbrich P, Ruiz-Contreras J, Paz-Artal E, Neth O, Allende LM
Front Immunol 2021;12:671755. Epub 2021 Aug 10 doi: 10.3389/fimmu.2021.671755. PMID: 34447369Free PMC Article

Clinical prediction guides

Maccari ME, Wolkewitz M, Schwab C, Lorenzini T, Leiding JW, Aladjdi N, Abolhassani H, Abou-Chahla W, Aiuti A, Azarnoush S, Baris S, Barlogis V, Barzaghi F, Baumann U, Bloomfield M, Bohynikova N, Bodet D, Boutboul D, Bucciol G, Buckland MS, Burns SO, Cancrini C, Cathébras P, Cavazzana M, Cheminant M, Chinello M, Ciznar P, Coulter TI, D'Aveni M, Ekwall O, Eric Z, Eren E, Fasth A, Frange P, Fournier B, Garcia-Prat M, Gardembas M, Geier C, Ghosh S, Goda V, Hammarström L, Hauck F, Heeg M, Heropolitanska-Pliszka E, Hilfanova A, Jolles S, Karakoc-Aydiner E, Kindle GR, Kiykim A, Klemann C, Koletsi P, Koltan S, Kondratenko I, Körholz J, Krüger R, Jeziorski E, Levy R, Le Guenno G, Lefevre G, Lougaris V, Marzollo A, Mahlaoui N, Malphettes M, Meinhardt A, Merlin E, Meyts I, Milota T, Moreira F, Moshous D, Mukhina A, Neth O, Neubert J, Neven B, Nieters A, Nove-Josserand R, Oksenhendler E, Ozen A, Olbrich P, Perlat A, Pac M, Schmid JP, Pacillo L, Parra-Martinez A, Paschenko O, Pellier I, Sefer AP, Plebani A, Plantaz D, Prader S, Raffray L, Ritterbusch H, Riviere JG, Rivalta B, Rusch S, Sakovich I, Savic S, Scheible R, Schleinitz N, Schuetz C, Schulz A, Sediva A, Semeraro M, Sharapova SO, Shcherbina A, Slatter MA, Sogkas G, Soler-Palacin P, Speckmann C, Stephan JL, Suarez F, Tommasini A, Trück J, Uhlmann A, van Aerde KJ, van Montfrans J, von Bernuth H, Warnatz K, Williams T, Worth AJJ, Ip W, Picard C, Catherinot E, Nademi Z, Grimbacher B, Forbes Satter LR, Kracker S, Chandra A, Condliffe AM, Ehl S; European Society for Immunodeficiencies Registry Working Party
J Allergy Clin Immunol 2023 Oct;152(4):984-996.e10. Epub 2023 Jun 28 doi: 10.1016/j.jaci.2023.06.015. PMID: 37390899
Nguyen T, Lau A, Bier J, Cooke KC, Lenthall H, Ruiz-Diaz S, Avery DT, Brigden H, Zahra D, Sewell WA, Droney L, Okada S, Asano T, Abolhassani H, Chavoshzadeh Z, Abraham RS, Rajapakse N, Klee EW, Church JA, Williams A, Wong M, Burkhart C, Uzel G, Croucher DR, James DE, Ma CS, Brink R, Tangye SG, Deenick EK
J Exp Med 2023 Jun 5;220(6) Epub 2023 Mar 21 doi: 10.1084/jem.20221020. PMID: 36943234Free PMC Article
Wang J, Xiu J, Farrell A, Baca Y, Arai H, Battaglin F, Kawanishi N, Soni S, Zhang W, Millstein J, Shields AF, Grothey A, Weinberg BA, Marshall JL, Lou E, Khushman M, Sohal DPS, Hall MJ, Liu T, Oberley M, Spetzler D, Korn WM, Shen L, Lenz HJ
Lancet Oncol 2023 Feb;24(2):151-161. Epub 2023 Jan 18 doi: 10.1016/S1470-2045(22)00783-5. PMID: 36681091Free PMC Article
Hu Y, Chen C, Tong X, Chen S, Hu X, Pan B, Sun X, Chen Z, Shi X, Hu Y, Shen X, Xue X, Lu M
Cell Death Dis 2021 Sep 9;12(9):842. doi: 10.1038/s41419-021-04127-3. PMID: 34504059Free PMC Article
Cancer Genome Atlas Research Network
Nature 2008 Oct 23;455(7216):1061-8. Epub 2008 Sep 4 doi: 10.1038/nature07385. PMID: 18772890Free PMC Article

Recent systematic reviews

Hanson J, Bonnen PE
Clin Exp Med 2024 Jan 27;24(1):17. doi: 10.1007/s10238-023-01259-y. PMID: 38280023Free PMC Article
López-Nevado M, González-Granado LI, Ruiz-García R, Pleguezuelo D, Cabrera-Marante O, Salmón N, Blanco-Lobo P, Domínguez-Pinilla N, Rodríguez-Pena R, Sebastián E, Cruz-Rojo J, Olbrich P, Ruiz-Contreras J, Paz-Artal E, Neth O, Allende LM
Front Immunol 2021;12:671755. Epub 2021 Aug 10 doi: 10.3389/fimmu.2021.671755. PMID: 34447369Free PMC Article

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