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pediatric patient / parent

Alexion Announces FDA Approval of ULTOMIRIS® (ravulizumab-cwvz) for Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH)

 – With this approval, ULTOMIRIS is the first and only medicine approved in the U.S. to treat children and adolescents with PNH –

– Approval based on interim results from Phase 3 study showing ULTOMIRIS demonstrated complete terminal complement inhibition through 26 weeks –

Pack your Bags and Head to the Hospital - Hailey's Story

Person's Name: 
Hailey Texeira

“You will need to pack your bags and head to Children’s Hospital, we think it’s leukemia.”

Only a few days after a Complete Blood Count, Christy, Hailey's mom, heard these words from Hailey’s doctor. Hailey had just started kindergarten.

At summer dance camp, Hailey won the award for “Most Tired.” That puzzled her mom. As kindergarten progressed, Christy noticed the fatigue. She assumed that kindergarten must be exhausting for children. Christmas was approaching when she noticed Hailey’s bruises, in odd places on her body.

Bone Marrow Disease(s): 
Content source: 

Clinical and molecular predictors of response and survival following venetoclax therapy in relapsed/refractory AML

Key Points
In patients with RR-AML, venetoclax combination therapy resulted in responses in 31% of patients and a median OS of 6.1 months.

NPM1 mutations predicted higher response rates; adverse cytogenetics and mutations in TP53, KRAS/NRAS, and SF3B1 predicted worse OS.