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Chapter 88 Immunoglobulin Light Chain Amyloidosis (Primary Amyloidosis) 1443

TABLE Active Nonchemotherapy Trials for Amyloidosis
88.3
Drug Mechanism Amyloidosis Type Trial Registration Number Comments
Tafamidis Misfolding interference Mutant (Val122Ile) and NCT00935012 Double-blind, placebo-
wild-type TTR controlled trial
ALN-TTRSC Suppress transthyretin TTR wild type including NCT01994889, None
expression cardiac involvement NCT01981837
ISIS-TTRRx Suppress transthyretin TTR neuropathy in ambulatory NCT01737398 Placebo-controlled
expression patients (use only one cane)
Doxycycline and tauroursodeoxycholic acid Misfolding interference TTR NCT01171859 None
Doxycycline Fibril disruption AL and TTR NCT01677286 None
NEOD001 Antibody-mediated AL NCT01707264 None
fibril dissolution
AL, Light chain amyloid; TTR, transthyretin.

a response to their primary therapy, immunomodulatory drug–con- Graziani MS, Merlini G: Serum free light chain analysis in the diagnosis and
taining therapies such as melphalan-dexamethasone-lenalidomide, management of multiple myeloma and related conditions. Expert Rev Mol
lenalidomide-dexamethasone, and cyclophosphamide-lenalidomide- Diagn 14:55, 2014.
dexamethasone are all appropriate combinations. Jazbeh S, Said A, Haddad RY, et al: Renal amyloidosis. Dis Mon 60:489,
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Mahmood S, Palladini G, Sanchorawala V, et al: Update on treatment of light
FUTURE DIRECTIONS AND SUMMARY chain amyloidosis. Haematologica 99:209, 2014.
Merlini G, Comenzo RL, Seldin DC, et al: Immunoglobulin light chain
Some of the nonchemotherapy-based imitatives for amyloidosis treat- amyloidosis. Expert Rev Hematol 7:143, 2014.
ment are listed in Table 88.3. Antibodies, small interfering RNA, and Mollee P, Renaut P, Gottlieb D, et al: How to diagnose amyloidosis. Intern
antisense oligonucleotides are all being explored. Combinations of Med J 44:7, 2014.
anti–light-chain synthesis and therapies to prevent misfolding are Sachchithanantham S, Wechalekar AD: Imaging in systemic amyloidosis. Br
likely the next generation of therapies. Med Bull 107:41, 2013.
Sanchorawala V: High dose melphalan and autologous peripheral blood
stem cell transplantation in AL amyloidosis. Hematol Oncol Clin North
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Chaulagain CP, Comenzo RL: New insights and modern treatment of AL combination regimens for myeloma or systemic light-chain amyloidosis:
amyloidosis. Curr Hematol Malig Rep 8:291, 2013. a retrospective chart review of response rates and toxicity in newly diag-
Ericson S, Shah N, Liberman J, et al: Fatal bleeding due to acquired factor nosed patients. Clin Ther 35:1614, 2013.
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