Familial partial lipodystrophy type 2

Is metreleptin a treatment option for those with familial partial lypodystrophy type 2?

Metreleptin is not an appropriate treatment option for all individuals with familial partial lipodystrophy type 2. You should work with your healthcare provider(s) to determine if this medication would be an appropriate option for you. At this time, metreleptin is approved by the Food and Drug Administration (FDA) for treating metabolic complications in individuals with generalized lipodystropies. It is not currently approved for patients with familial partial lipodystrophy. Initial studies have shown, however, that metreleptin can be an effective treatment for these individuals, in particular those with severe metabolic abnormalities or low leptin levels. More specifically, the individuals with familial partial lipodystrophy most likely to respond to metreleptin are those with triglyceride levels greater than 500 mg/dL or HbA1c greater than 8% or those whose leptin levels were less than 4 ng/mL (which are comparable to leptin levels seen in generalized forms of lipodystrophy). Further studies are needed to better understand how metreleptin may be used in this population.

Last updated on 05-01-20

How can I gain access to treatment with metreleptin if it is not FDA approved for familial partial lipodystrophy type 2?

At this time, metreleptin is only available through clinical trials and through compassionate use programs. Global Genes provides a RareToolkit entitled "Access to Unapproved Medicine: Is This an Option for Me? (USA)" which may be of interest to you. An additional guide is available for those who reside in Canada.

Last updated on 05-01-20

How might familial partial lipodystrophy type 2 be treated?

Individuals with familial partial lipodystrophy type 2 may require the coordinated efforts of a team of specialists, including a primary care provider, cardiologist, endocrinologist, nutritionist, plastic surgeon, and psychiatrist/psychologist. Dietary modifications and daily physical activity can help improve the metabolic complications of lipodystrophy. Insulin sensitizers (such as metformin) and lipid-lowering drugs (statins, fibrates) may also be helpful. Due to the risk of cardiac complications, individuals with familial partial lipodystrophy type 2 should have regular cardiac check- ups. Metreleptin has shown some promise as a treatment for this condition, however, it is not widely approved for treatment at this time. The characteristic fat distribution seen in individuals with familial partial lipodystrophy type 2 cannot be reversed. For some individuals, cosmetic surgery may be beneficial in improving appearance and for management of metabolic complications. Liposuction may also be used to remove unwanted deposits of fat. Because familial partial lipodystrophy type 2 can cause anxiety and stress, psychological support is also an important part of treatment.

Last updated on 05-01-20

Selected Full-Text Journal Articles

Familial partial lipodystrophy type 2 - articles

Diker-Cohen T, Cochran E, Gorden P, Brown RJ. Partial and generalized lipodystrophy: comparison of baseline characteristics and response to metreleptin. J Clin Endocrinol Metab. 2015 May; 100(5):1802-10.

Last updated on 04-27-20

Name: Metabolic Support UK 5 Hilliards Court Sandpiper Way
Chester Business Park
Chester, CH4 9QP, United Kingdom
Phone: 0845 241 2173 Toll Free: 0800 652 3181 Email: https://www.metabolicsupportuk.org/contact-us Url: https://www.metabolicsupportuk.org
Name: American Dietetic Association 120 South Riverside Plaza, Suite 2000
Chicago, IL, 60606-6995, United States
Toll Free: 800-366-1655 Email: knowledge@eatright.org Url: http://www.eatright.org/Public/
Name: Lipodystrophy United 22 Frances Road
Los Lunas, NM, 87031,
Phone: (209) 845-7273 Email: info@lipodystrophyunited.org Url: http://www.lipodystrophyunited.org Facebook: https://www.facebook.com/LipodystrophyUnited/

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