Winst gevend schreef op 24 mei 2024 17:22:
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Eerste resultaten (ELSEVIER Medical Documents 2024) / Compassionate use using leniolisib.
Switch from sirolimus naar pediatrische patiënten met leniolisib veel belovend.Here we describe 2 pediatric patients with APDS2 who switched from the mTOR inhibitor,
sirolimus, to the specific PI3 K delta inhibitor, leniolisib.
Clinical history including initial immunological parameters have been described elsewhere.
Following incomplete clinical success with different immunomodulatory treatments,
patients switched from sirolimus to leniolisib, while maintaining monthly subcutaneous immunoglobulin therapy.
Methods: Observational 6-month study of real-life experience from the leniolisib early access program.
Two clinically and immunological stable pediatric APDS2 patients < 45 kg switched from sirolimus to leniolisib
(30 mg BID), while continuing immunoglobulin replacement therapy.
Clinical and immunological follow-up was performed during the first 6 months of treatment.
All previously available therapeutic data from these patients (antibiotics, IRT, MTOR, and steroids)
were compared with new clinical and immunological outcomes after starting leniolisib.
Results: Clinical and immunological response to leniolisib treatment are summarized.
During the 6-month treatment with leniolisib, both our patients showed excellent adherence, no infections,
and no other severe adverse events, apart from transitional hair loss in patient 2.
The children and parents reported subjective improved well-being (less tired, more active),
suggesting a potentially favorable risk-benefit profile with no clinical, infectious,
or immunological worsening after switching to leniolisib during this period.
Observation: Leniolisib has shown encouraging results in 2 pediatric
patients within a compassionate use program.
Further well-defined prospective studies are required to determine the long-term benefits
of this potentially promising targeted therapy in APDS, particularly in young children.
Vroege felicitatie is op zijn plaats.
Gaat over de 25% aan jonge patiënten wereldwijd, die nu nog niet onder het 12+ label vallen.Helaas Engelstalig.