Why the study?
Available drug treatments are inadequate for patients with homozygous familial hypercholesterolemia, and acceptable LDL-C levels are rarely achieved even with serial apheresis.
Does evinacumab improve LDL-C levels and reduce apheresis frequency in a patient with severe homozygous familial hypercholesterolemia?
Does evinacumab improve LDL-C levels and reduce apheresis frequency in a patient with severe homozygous familial hypercholesterolemia?
Evinacumab effectively lowered LDL-C and reduced the need for frequent apheresis in a patient with severe homozygous familial hypercholesterolemia refractory to standard therapies.
May support evinacumab in refractory HoFH; hypothesis-generating and requires prospective trials before adoption.
Patients with homozygous familial hypercholesterolemia (HoFH) have extremely elevated levels of low-density lipoprotein cholesterol (LDL-C), with premature atherosclerosis and aortic valve disease. Available drug treatments are inadequate, and even with serial apheresis, HoFH patients rarely achieve acceptable LDL-C levels. Evinacumab is a monoclonal antibody against angiopoietin-like protein 3 that lowers LDL-C via a novel receptor-independent mechanism. We describe an Ontario patient with HoFH who for 17 months has been treated with monthly infusions of evinacumab added to pre-existing statin, ezetimibe, and evolocumab therapy. Evinacumab in this HoFH patient was associated with markedly improved LDL-C levels and decreased frequency of apheresis.
No takes yet. Share an insight, caveat, or question.
Jeraj et al. (2021) studied this question.
Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context: