The first study was a cross-section analysis examining key clinical characteristics of pediatric patients with confirmed familial partial lipodystrophy (FPL). Of the 13 pediatric patients 18 years or younger:
The analysis was conducted in patients who were referred to the
"FPL is a rare disease, and there is significant clinical variability among these patients, including differences in leptin levels, percent body fat and triglycerides, which makes accurate diagnosis of FPL challenging," said Brown. "Early diagnosis of pediatric FPL is important for management of these patients."
A second study examined the effect of metreleptin on liver volume among pediatric patients with generalized lipodystrophy (GL). Of the 13 patients assessed, all had an enlarged liver (hepatomegaly) at baseline.
For patients assessed within a year after initiating treatment with metreleptin, liver volume decreased by 25 percent. The mean duration of treatment was 9 months.
Among patients who had longer exposure to metreleptin (N=9) with a mean of 46 months, liver volume decreased by 34 percent.
The most commonly reported adverse events included gastrointestinal disorders, including abdominal pain and pancreatitis, and the two patients who experienced pancreatitis had reported episodes in their prior medical history.
This post-hoc analysis reviewed patients who participated in a prospective, open-label study conducted by the
"Fatty liver is a common complication among pediatric patients with GL," said
Lipodystrophy syndromes (LD) are ultra-rare disorders characterized by the irreversible loss of adipose tissue. In patients with lipodystrophy syndromes, levels of leptin are often very low. Leptin is a naturally occurring hormone produced in adipose tissue and is an important regulator of energy homoeostasis, fat and glucose metabolism, reproductive capacity, and other diverse physiological functions.
With generalized lipodystrophy, the loss of fat affects the whole body. With partial lipodystrophy, the loss of fat typically occurs in the arms, legs, head, and trunk regions, while accumulation of fat may occur in other areas of the body, including the neck, face, and intra-abdominal
regions. Metreleptin is approved in the
About MYALEPT® (metreleptin) for injection
MYALEPT® (metreleptin) for injection is only approved in
Highlights of Safety Information from
WARNING: RISK OF ANTI-METRELEPTIN ANTIBODIES WITH NEUTRALIZING ACTIVITY AND RISK OF LYMPHOMA
See full prescribing information for complete boxed warning.
Anti-metreleptin antibodies with neutralizing activity have been identified in patients treated with MYALEPT. The consequences are not well characterized but could include inhibition of endogenous leptin action and/or loss of MYALEPT efficacy. Worsening metabolic control and/or severe infection have been reported. Test for anti-metreleptin antibodies with neutralizing activity in patients with severe infections or loss of efficacy during MYALEPT treatment.
T-cell lymphoma has been reported in patients with acquired generalized lipodystrophy, both treated and not treated with MYALEPT. Carefully consider the benefits and risks of MYALEPT treatment in patients with significant hematologic abnormalities and/or acquired generalized lipodystrophy.
MYALEPT is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the MYALEPT REMS PROGRAM.
MYALEPT is contraindicated in general obesity not associated with congenital leptin deficiency and in patients with hypersensitivity to metreleptin.
WARNINGS AND PRECAUTIONS
Anti-metreleptin antibodies with neutralizing activity: Could inhibit endogenous leptin action and/or result in loss of MYALEPT efficacy. Test for neutralizing antibodies in patients with severe infections or loss of efficacy during MYALEPT treatment.
T-cell lymphoma: Carefully consider benefits and risks of treatment with MYALEPT in patients with significant hematologic abnormalities and/or acquired generalized lipodystrophy.
Hypoglycemia: A dose adjustment, including possible large reductions, of insulin or insulin secretagogue may be necessary. Closely monitor blood glucose in patients on concomitant insulin, or insulin secretagogue.
Autoimmunity: Autoimmune disorder progression has been observed in patients treated with MYALEPT. Carefully consider benefits and risks of MYLEPT treatment in patients with autoimmune disease.
Hypersensitivity reactions (e.g., anaphylaxis, urticaria or generalized rash) have been reported. Patients should promptly seek medical advice about discontinuation of MYALEPT if a hypersensitivity reaction occurs.
Benzyl Alcohol Toxicity: Preservative-free Water for Injection is recommended for use in neonates and infants.
Most common adverse reactions (≥10%) in clinical trials were headache, hypoglycemia, decreased weight, and abdominal pain.
USE IN SPECIAL POPULATIONS
MYALEPT should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus. No adequate and well-controlled studies have been conducted with metreleptin in pregnant women. Nursing Mothers should discontinue drug or nursing.
For additional information, please see the U.S. Prescribing Information including Box Warning.
The research described here is conducted in part by the Intramural Research Program of the
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