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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

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
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
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
Palladini G, Milani P, Merlini G
Blood 2020 Dec 3;136(23):2620-2627. doi: 10.1182/blood.2020006913. PMID: 33270858

Diagnosis

Mohan M, Monge J, Shah N, Luan D, Forsberg M, Bhatlapenumarthi V, Balev M, Patwari A, Cheruvalath H, Bhutani D, Thanendrarajan S, Dhakal B, Zangari M, Al-Hadidi S, Cooper D, Lentzsch S, van Rhee F, D'Souza A, Szabo A, Schinke C, Chakraborty R
Blood Cancer J 2024 Mar 5;14(1):35. doi: 10.1038/s41408-024-01003-z. PMID: 38443345Free 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
Palladini G, Milani P
Drugs 2023 Feb;83(3):203-216. Epub 2023 Jan 18 doi: 10.1007/s40265-022-01830-z. PMID: 36652193
Al Hamed R, Bazarbachi AH, Bazarbachi A, Malard F, Harousseau JL, Mohty M
Blood Cancer J 2021 May 18;11(5):97. doi: 10.1038/s41408-021-00486-4. PMID: 34006856Free PMC Article
Palladini G, Milani P, Merlini G
Blood 2020 Dec 3;136(23):2620-2627. doi: 10.1182/blood.2020006913. PMID: 33270858

Therapy

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
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
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
Palladini G, Milani P, Merlini G
Blood 2020 Dec 3;136(23):2620-2627. doi: 10.1182/blood.2020006913. PMID: 33270858

Prognosis

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
Oliver-Caldes A, Jiménez R, Español-Rego M, Cibeira MT, Ortiz-Maldonado V, Quintana LF, Castillo P, Guijarro F, Tovar N, Montoro M, Benitez-Ribas D, Bataller A, González-Navarro EA, Cid J, Lozano M, Perez-Amill L, Martin-Antonio B, Mena MP, Moreno DF, Rodríguez-Lobato LG, Campistol JM, Calvo G, Bladé J, Rosiñol L, Juan M, Pascal M, Urbano-Ispizua A, Fernández de Larrea C
J Immunother Cancer 2021 Dec;9(12) doi: 10.1136/jitc-2021-003783. PMID: 34876408Free PMC Article
Gavriatopoulou M, Musto P, Caers J, Merlini G, Kastritis E, van de Donk N, Gay F, Hegenbart U, Hajek R, Zweegman S, Bruno B, Straka C, Dimopoulos MA, Einsele H, Boccadoro M, Sonneveld P, Engelhardt M, Terpos E
Leukemia 2018 Sep;32(9):1883-1898. Epub 2018 Jul 23 doi: 10.1038/s41375-018-0209-7. PMID: 30038381
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
Berk JL, Keane J, Seldin DC, Sanchorawala V, Koyama J, Dember LM, Falk RH
Chest 2003 Sep;124(3):969-77. doi: 10.1378/chest.124.3.969. PMID: 12970025

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
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
Sanchorawala V, Wechalekar AD, Kim K, Schönland SO, Landau HJ, Kwok F, Suzuki K, Dispenzieri A, Merlini G, Comenzo RL, Cherepanov D, Hayden VC, Kumar A, Labotka R, Faller DV, Kastritis E
Am J Hematol 2023 May;98(5):720-729. Epub 2023 Feb 14 doi: 10.1002/ajh.26866. PMID: 36708469
Kfir-Erenfeld S, Asherie N, Grisariu S, Avni B, Zimran E, Assayag M, Sharon TD, Pick M, Lebel E, Shaulov A, Cohen YC, Avivi I, Cohen CJ, Stepensky P, Gatt ME
Clin Cancer Res 2022 Dec 1;28(23):5156-5166. doi: 10.1158/1078-0432.CCR-22-0637. PMID: 36107221

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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