Expression of γ-globin genes in β-thalassemia patients treated with sirolimus: results from a pilot clinical trial (Sirthalaclin).
Cristina ZuccatoLucia Carmela CosenzaMatteo ZurloJessica GasparelloChiara PapiElisabetta D'AversaGiulia BreveglieriIlaria LamprontiAlessia FinottiMonica BorgattiChiara ScapoliAlice StievanoMonica FortiniEric RamazzottiNicola MarchettiMarco ProsdocimiMaria Rita GamberiniRoberto GambariPublished in: Therapeutic advances in hematology (2022)
The data obtained indicate that low doses of sirolimus modify hematopoiesis and induce increased expression of γ-globin genes in a subset of patients with β-thalassemia. Further clinical trials are warranted, possibly including testing of the drug in patients with less severe forms of the disease and exploring combination therapies.
Keyphrases
- clinical trial
- poor prognosis
- genome wide
- study protocol
- phase ii
- sickle cell disease
- open label
- genome wide identification
- bioinformatics analysis
- phase iii
- randomized controlled trial
- electronic health record
- long non coding rna
- early onset
- dna methylation
- drug induced
- machine learning
- genome wide analysis
- adverse drug