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Historical supportive care does not address the underlying cause of LAL-D1,2
Lipid-lowering medications do not prevent liver or extrahepatic complications in patients with LAL-D1
Lipid-lowering medications (LLMs) are typically used to treat dyslipidemia—a common complication in patients with LAL-D.1 However, LLMs do not address the underlying cause of LAL-D—accumulation of cholesteryl esters and triglycerides within the lysosome.1,3 Additionally, they do not prevent liver disease progression, a common complication of LAL-D.1,2
In an observational study of 48 patients with LAL-D4
It is important to monitor disease progression in patients with LAL-D who are taking LLMs. In a case review of 27 patients with LAL-D on statins:1
Liver transplant does not treat the multiorgan nature of LAL-D pathology and is associated with serious complications1,2
Liver transplant does not address the underlying cause of the disease—the deficiency of the vital LAL enzyme1—and does not halt disease progression in extrahepatic organs, including the heart and kidneys.1Approximately 33% (4/12) of patients who received liver transplant experienced transplant rejection, congestive heart failure, renal failure, or death.1
Historical Supportive Care: Hematopoietic stem cell transplant (HSCT)
HSCT is not proven safe or effective for LAL-D by any well-controlled studies, as it is associated with multiorgan failure, sepsis, graft-vs-host disease, and graft failure.2,3 In infants, 100% (10/10) who received HSCT died before 4 years of age; of these, 70% (7/10) died before 9 months of age.4
References: 1. Bernstein DL, et al. J Hepatol. 2013;58:1230-43. doi:10.1016/j.jhep.2013.02.014. 2. Reiner Ž, et al. Atherosclerosis. 2014;235:21-30. doi:10.1016/j.atherosclerosis.2014.04.003. 3. Tolar J, et al. Bone Marrow Transplantation. 2009;43:21-7.doi:10.1038/bmt.2008.273. 4. Jones SA, et al. Genet Med. 2016;18:452-8. doi:10.1038/gim.2015.108.