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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

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
Zhang Y, Comenzo RL
Curr Treat Options Oncol 2022 Jul;23(7):1059-1071. Epub 2022 May 30 doi: 10.1007/s11864-021-00922-4. PMID: 35635625
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
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
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

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
Basset M, Kimmich CR, Schreck N, Krzykalla J, Dittrich T, Veelken K, Goldschmidt H, Seckinger A, Hose D, Jauch A, Müller-Tidow C, Benner A, Hegenbart U, Schönland SO
Br J Haematol 2021 Oct;195(2):230-243. Epub 2021 Aug 3 doi: 10.1111/bjh.17685. PMID: 34341985
Basset M, Nuvolone M, Palladini G, Merlini G
Expert Rev Hematol 2020 Sep;13(9):1003-1015. Epub 2020 Aug 11 doi: 10.1080/17474086.2020.1803060. PMID: 32721177
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

Clinical prediction guides

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
Hari P, Lin HM, Asche CV, Ren J, Yong C, Luptakova K, Faller DV, Sanchorawala V
Amyloid 2018 Mar;25(1):1-7. Epub 2018 Jan 5 doi: 10.1080/13506129.2017.1411796. PMID: 29303358

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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