FDA Approves First-Ever Treatment for Children with GPA and MPA

The FDA announced last week that it has approved Rituxan (rituximab)to treat granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA) in children 2 years of age and older in combination with glucocorticoids (steroid hormones).

It is the first approved treatment for children with those rare diseases!

What is GPA?

Granulomatosis with polyangiitis is a rare blood vessel disease that is potentially serious when not promptly diagnosed.  GPA is characterized by the abnormal inflammation of the small and medium-sized blood vessels throughout the body, which does not allow blood to flow properly and prevents cells from getting the oxygen that they need.

To learn more GPA, click here.

What is MPA?

Microscopic polyangiitis is a rare condition that is characterized by the inflammation of blood vessels known as vasculitis. Areas most commonly affected by MPA are the small blood vessels of the kidneys, lungs, skin, joints, and nervous system.

This inflammation from MPA can lead to weakening and narrowing of the blood vessels, which then can lead to aneurysms, vessel rupture, and narrowing of the vessels.

To learn more about MPA, click here.

About Rituxan

Rituxan was approved to treat adult patients with GPA and MPA in 2011. It is also approved to treat four additional diseases, first gaining approval to treat Non-Hodgkin’s lymphoma in 1997.

“The Rituxan application for pediatric GPA and MPA was approved under a priority review, and with orphan designation, to fulfil an unmet medical need for these rare and serious diseases,” said Nikolay Nikolov, M.D., of the FDA. “Rituxan provides a treatment option that has not existed until now for children who suffer from these diseases.”

The most common side effects are infections, infusion-related reactions, abnormally low level of lymphocytes in the blood (lymphopenia) and anemia.

Health care professionals are advised to monitor patients for tumor lysis syndrome (a treatment complication where tumor cells are killed off at the same time and released into the bloodstream), cardiac adverse reactions, damage to kidneys (renal toxicity), and bowel obstruction and perforation (small hole formation).

This is a big step for children and families dealing with those rare diseases!

To read more about, click here.


Does a child or adolescent in your family have a rare disease? Share your stories, thoughts, and hopes with the Patient Worthy community!

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