Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most mysterious diseases and conditions. There are thousands of rare diseases out there, but only a very small number of them have viable treatments and regularly make the news. This series is an opportunity to learn the basics about some of the diseases that almost no one hears much about or that we otherwise haven’t been able to report on very often.
Eyes front and ears open. Class is now in session.
The disease that we will be learning about today is:
Acquired Generalized Lipodystrophy
This disease is also known as Lawrence syndrome.
What is Acquired Generalized Lipodystrophy?
- Acquired lipodystrophy is a general term for types of lipodystrophy that are not inherited, but rather acquired at some point during life.
- Acquired lipodystrophies do not have a direct genetic cause, but rather many different factors may be involved.
How Do You Get It?
- AGL could be due to panniculitis (inflammation in the fat) or autoimmune disease
- Onset of AGL may also occur following infections such as varicella, measles, pertussis, diphtheria, pneuomonia, osteomyelitis, parotitis, infectious mononucleosis, and hepatitis
- Sometimes no cause can be identified
What Are The Symptoms?
- Patients have normal body fat at birth, distinguishing them from patients with congenital lipodystrophy.
- Loss of fat tissue, typically from under the skin
- Underlying reason is a deficiency in leptin, leading to an inability to store triglycerides in normal fat depots
- This often leads to severe insulin resistance, diabetes and elevated triglyceride levels
- Females are affected more than males
- Excess body hair
- Strong appetite
- ovarian cysts
- darkly pigmented skin in certain areas
- Enlarged genitalia
- Enlarged spleen and liver
How Is It Treated?
- Approved by US FDA in February 2014 as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency
- Myalept™ is a laboratory created form of human leptin
- First and only treatment approved by FDA for AGL
- Prescription medication used with a diet recommended to treat problems caused by not having enough leptin in the body in people with congenital or acquired generalized lipodystrophy
- Other treatments may include anti-diabetic medications
- Insulin, thiazolidinediones, metformin
- Statins or fibrates to treat hyperlipidemia
- Exercise and a diet low in fat are recommended lifestyle changes
- Some patients opt for cosmetic surgeries to replace lost adipose tissue
Where Can I Learn More???
- Check out our cornerstone on this disease here.