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Refractory Primary Amyloidosis

MedGen UID:
1611881
Concept ID:
C4524852
Neoplastic Process

Definition

Primary amyloidosis that does not respond to treatment. [from NCI]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVRefractory Primary Amyloidosis

Professional guidelines

PubMed

Palladini G, Milani P
Drugs 2023 Feb;83(3):203-216. Epub 2023 Jan 18 doi: 10.1007/s40265-022-01830-z. PMID: 36652193
Palladini G, Milani P, Merlini G
Blood 2020 Dec 3;136(23):2620-2627. doi: 10.1182/blood.2020006913. PMID: 33270858
Palladini G, Merlini G
Blood 2016 Jul 14;128(2):159-68. Epub 2016 Apr 6 doi: 10.1182/blood-2016-01-629790. PMID: 27053535

Recent clinical studies

Etiology

Forgeard N, Elessa D, Carpinteiro A, Belhadj K, Minnema M, Roussel M, Huart A, Javaugue V, Pascal L, Royer B, Talbot A, Gounot R, Hegenbart U, Schonland S, Karlin L, Harel S, Kastritis E, Bridoux F, Jaccard A, Arnulf B
Blood 2024 Feb 22;143(8):734-737. doi: 10.1182/blood.2023022937. PMID: 38096365
San-Miguel J, Dhakal B, Yong K, Spencer A, Anguille S, Mateos MV, Fernández de Larrea C, Martínez-López J, Moreau P, Touzeau C, Leleu X, Avivi I, Cavo M, Ishida T, Kim SJ, Roeloffzen W, van de Donk NWCJ, Dytfeld D, Sidana S, Costa LJ, Oriol A, Popat R, Khan AM, Cohen YC, Ho PJ, Griffin J, Lendvai N, Lonardi C, Slaughter A, Schecter JM, Jackson CC, Connors K, Li K, Zudaire E, Chen D, Gilbert J, Yeh TM, Nagle S, Florendo E, Pacaud L, Patel N, Harrison SJ, Einsele H
N Engl J Med 2023 Jul 27;389(4):335-347. Epub 2023 Jun 5 doi: 10.1056/NEJMoa2303379. PMID: 37272512
Moreau P, Dimopoulos MA, Mikhael J, Yong K, Capra M, Facon T, Hajek R, Špička I, Baker R, Kim K, Martinez G, Min CK, Pour L, Leleu X, Oriol A, Koh Y, Suzuki K, Risse ML, Asset G, Macé S, Martin T; IKEMA study group
Lancet 2021 Jun 19;397(10292):2361-2371. Epub 2021 Jun 4 doi: 10.1016/S0140-6736(21)00592-4. PMID: 34097854
Shah BD, Ghobadi A, Oluwole OO, Logan AC, Boissel N, Cassaday RD, Leguay T, Bishop MR, Topp MS, Tzachanis D, O'Dwyer KM, Arellano ML, Lin Y, Baer MR, Schiller GJ, Park JH, Subklewe M, Abedi M, Minnema MC, Wierda WG, DeAngelo DJ, Stiff P, Jeyakumar D, Feng C, Dong J, Shen T, Milletti F, Rossi JM, Vezan R, Masouleh BK, Houot R
Lancet 2021 Aug 7;398(10299):491-502. Epub 2021 Jun 4 doi: 10.1016/S0140-6736(21)01222-8. PMID: 34097852
Jelinek T, Kufova Z, Hajek R
Crit Rev Oncol Hematol 2016 Mar;99:249-60. Epub 2016 Jan 15 doi: 10.1016/j.critrevonc.2016.01.004. PMID: 26806146

Diagnosis

Kim K, Phelps MA
Clin Pharmacokinet 2023 Jun;62(6):789-806. Epub 2023 May 2 doi: 10.1007/s40262-023-01240-8. PMID: 37129750Free PMC Article
Palladini G, Milani P
Drugs 2023 Feb;83(3):203-216. Epub 2023 Jan 18 doi: 10.1007/s40265-022-01830-z. PMID: 36652193
Palladini G, Milani P, Merlini G
Blood 2020 Dec 3;136(23):2620-2627. doi: 10.1182/blood.2020006913. PMID: 33270858
Simon SP, Thomas J
Saudi J Kidney Dis Transpl 2020 Mar-Apr;31(2):545-548. doi: 10.4103/1319-2442.284035. PMID: 32394933
Jelinek T, Kufova Z, Hajek R
Crit Rev Oncol Hematol 2016 Mar;99:249-60. Epub 2016 Jan 15 doi: 10.1016/j.critrevonc.2016.01.004. PMID: 26806146

Therapy

Forgeard N, Elessa D, Carpinteiro A, Belhadj K, Minnema M, Roussel M, Huart A, Javaugue V, Pascal L, Royer B, Talbot A, Gounot R, Hegenbart U, Schonland S, Karlin L, Harel S, Kastritis E, Bridoux F, Jaccard A, Arnulf B
Blood 2024 Feb 22;143(8):734-737. doi: 10.1182/blood.2023022937. PMID: 38096365
San-Miguel J, Dhakal B, Yong K, Spencer A, Anguille S, Mateos MV, Fernández de Larrea C, Martínez-López J, Moreau P, Touzeau C, Leleu X, Avivi I, Cavo M, Ishida T, Kim SJ, Roeloffzen W, van de Donk NWCJ, Dytfeld D, Sidana S, Costa LJ, Oriol A, Popat R, Khan AM, Cohen YC, Ho PJ, Griffin J, Lendvai N, Lonardi C, Slaughter A, Schecter JM, Jackson CC, Connors K, Li K, Zudaire E, Chen D, Gilbert J, Yeh TM, Nagle S, Florendo E, Pacaud L, Patel N, Harrison SJ, Einsele H
N Engl J Med 2023 Jul 27;389(4):335-347. Epub 2023 Jun 5 doi: 10.1056/NEJMoa2303379. PMID: 37272512
Moreau P, Dimopoulos MA, Mikhael J, Yong K, Capra M, Facon T, Hajek R, Špička I, Baker R, Kim K, Martinez G, Min CK, Pour L, Leleu X, Oriol A, Koh Y, Suzuki K, Risse ML, Asset G, Macé S, Martin T; IKEMA study group
Lancet 2021 Jun 19;397(10292):2361-2371. Epub 2021 Jun 4 doi: 10.1016/S0140-6736(21)00592-4. PMID: 34097854
Shah BD, Ghobadi A, Oluwole OO, Logan AC, Boissel N, Cassaday RD, Leguay T, Bishop MR, Topp MS, Tzachanis D, O'Dwyer KM, Arellano ML, Lin Y, Baer MR, Schiller GJ, Park JH, Subklewe M, Abedi M, Minnema MC, Wierda WG, DeAngelo DJ, Stiff P, Jeyakumar D, Feng C, Dong J, Shen T, Milletti F, Rossi JM, Vezan R, Masouleh BK, Houot R
Lancet 2021 Aug 7;398(10299):491-502. Epub 2021 Jun 4 doi: 10.1016/S0140-6736(21)01222-8. PMID: 34097852
Godara A, Palladini G
Hematol Oncol Clin North Am 2020 Dec;34(6):1145-1159. Epub 2020 Oct 3 doi: 10.1016/j.hoc.2020.08.005. PMID: 33099430

Prognosis

Dima D, Hughes M, Orland M, Ullah F, Goel U, Anwer F, Raza S, Mazzoni S, Bhutani D, Williams L, Lentzsch S, Samaras C, Valent J, Chakraborty R, Khouri J
Amyloid 2024 Sep;31(3):195-201. Epub 2024 Jul 2 doi: 10.1080/13506129.2024.2366806. PMID: 38956891
Martin T, Dimopoulos MA, Mikhael J, Yong K, Capra M, Facon T, Hajek R, Špička I, Baker R, Kim K, Martinez G, Min CK, Pour L, Leleu X, Oriol A, Koh Y, Suzuki K, Casca F, Macé S, Risse ML, Moreau P
Blood Cancer J 2023 May 9;13(1):72. doi: 10.1038/s41408-023-00797-8. PMID: 37156782Free PMC Article
Milani P, Gertz MA, Merlini G, Dispenzieri A
Amyloid 2017 Dec;24(4):213-216. Epub 2017 Aug 31 doi: 10.1080/13506129.2017.1370421. PMID: 28857614
Avula SR, Handa R, Balakrishnan B, Girard S
BMJ Case Rep 2017 Aug 7;2017 doi: 10.1136/bcr-2017-220538. PMID: 28784887Free PMC Article
Perfetto F, Cappelli F, Bergesio F, Ciuti G, Porciani MC, Padeletti L, Moggi Pignone A
Intern Emerg Med 2013 Apr;8(3):191-203. Epub 2011 Jul 8 doi: 10.1007/s11739-011-0647-y. PMID: 21739229

Clinical prediction guides

Yong K, Martin T, Dimopoulos MA, Mikhael J, Capra M, Facon T, Hajek R, Špička I, Baker R, Kim K, Martinez G, Min CK, Pour L, Leleu X, Oriol A, Koh Y, Suzuki K, Casca F, Macé S, Risse ML, Moreau P
Lancet Haematol 2024 Oct;11(10):e741-e750. Epub 2024 Jul 24 doi: 10.1016/S2352-3026(24)00148-0. PMID: 39067465
Bomsztyk J, Ravichandran S, Khwaja J, Cohen O, Rauf MU, Foard D, Martinez-Naharro A, Venneri L, Whelan C, Fontana M, Hawkins PN, Gillmore J, Lachmann H, Mahmood S, Wechalekar AD
Br J Haematol 2024 Jul;205(1):138-145. Epub 2024 Jun 5 doi: 10.1111/bjh.19529. PMID: 38840512
van Ede ES, Hoeks MPA, Hofland J, Linssen VD, van Kuppevelt TH, Versteeg EM, Hafmans TG, Diepstra A, Kusters B, Vermorgen SMM
BMC Anesthesiol 2023 Jun 21;23(1):217. doi: 10.1186/s12871-023-02147-4. PMID: 37344791Free PMC Article
Martin T, Dimopoulos MA, Mikhael J, Yong K, Capra M, Facon T, Hajek R, Špička I, Baker R, Kim K, Martinez G, Min CK, Pour L, Leleu X, Oriol A, Koh Y, Suzuki K, Casca F, Macé S, Risse ML, Moreau P
Blood Cancer J 2023 May 9;13(1):72. doi: 10.1038/s41408-023-00797-8. PMID: 37156782Free PMC Article
Kim K, Phelps MA
Clin Pharmacokinet 2023 Jun;62(6):789-806. Epub 2023 May 2 doi: 10.1007/s40262-023-01240-8. PMID: 37129750Free PMC Article

Recent systematic reviews

Ehsan H, Rafae A, Masood A, Wahab A, Sana MK, Ansar I, Neupane K, Umar A, Ehsan A, Hashmi H
Clin Lymphoma Myeloma Leuk 2022 May;22(5):e285-e292. Epub 2021 Nov 1 doi: 10.1016/j.clml.2021.10.014. PMID: 34879994
Shragai T, Gatt M, Lavie N, Vaxman I, Tadmor T, Rouvio O, Zektser M, Horowitz N, Magen H, Ballan M, Suru C, Luttwak E, Levi S, Ziv-Baran T, Avivi I, Cohen YC
Eur J Haematol 2021 Feb;106(2):184-195. Epub 2020 Nov 9 doi: 10.1111/ejh.13535. PMID: 33090552

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