FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W4377696088> ?p ?o ?g. }

• W4377696088 endingPage "109547" @default.
• W4377696088 startingPage "109547" @default.
• W4377696088 abstract "A three-year-old male presented to our multispecialty clinic with recurrent fever without periodicity, neutropenia, diffuse lymphadenopathy, and splenomegaly. Family history was notable for two maternal cousins with hypogammaglobulinemia and neutropenia. Pertinent labs include elevated CD3 positive, double negative cells (7.7%, 156 cells/mcl), elevated IL-10 (85.6 pg/mL); and elevated IL-18 (3145 pg/mL). Rheumatologic studies returned unremarkable (ANA was weakly positive, with extracted nuclear antigen antibody panel within normal limits). Abdominal ultrasound revealed splenomegaly (14 cm). Whole genome sequencing for the patient and parents was negative, including no diagnostic abnormalities in the FAS, FASLG or CASP10 genes. The patient was diagnosed with probable ALPS, per the 2009 revised ALPS criteria (Oliveira et al, Blood, 2010). He was started on colchicine for fevers but was transitioned to sirolimus several weeks later due to lack of clinical improvement. Mirroring a prior study that evaluated sirolimus for children with ALPS, sirolimus was initiated at a dose of 2.3 mg/m2, targeting goal trough levels 5–15 ng/mL (Bride et al. Blood, 2016). Sirolimus trough levels are depicted in Table 1 and Figure 1.Download : Download high-res image (57KB)Download : Download full-size imageFigure 1. Sirolimus trough level over time Figure 1. Sirolimus trough level over time At follow up 3 months later, the patient’s CD3 positive, double negative T cells normalized (0.2%, 3 cells/mcl), splenomegaly improved (10.7 cm), lymphadenopathy was no longer palpable, and cytokine levels normalized. Despite consecutive subtherapeutic sirolimus trough levels preceding the 3-month evaluation, probable ALPS markers improved. Sirolimus has caused toxicity. Baseline lipid panel prior to sirolimus therapy demonstrated elevated triglycerides and low HDL, likely related to ALPS (Sriram et al, Case Reports Immunol, 2016). While HDL improved, total cholesterol and LDL increased and triglycerides remain elevated. He has developed hypertension while on sirolimus. These findings demonstrate that sirolimus trough alone may not predict clinical response. Given the excellent correlation between trough levels and area under the concentration-time curve (AUC) for sirolimus (Stenton et al. Clin Pharmacokinetics 2005), it is unlikely that AUC sirolimus measurements would either be of clinical benefit or better explain the patient’s clinical response. This patient’s excellent clinical response despite multiple subtherapeutic sirolimus trough levels suggests that further research to determine the optimal sirolimus trough range may be indicated." @default.
• W4377696088 created "2023-05-24" @default.
• W4377696088 creator A5051464718 @default.
• W4377696088 creator A5067213148 @default.
• W4377696088 creator A5076514127 @default.
• W4377696088 creator A5091465356 @default.
• W4377696088 date "2023-05-01" @default.
• W4377696088 modified "2023-10-17" @default.
• W4377696088 title "ALPS like: Trekking Sirolimus and its Fallouts" @default.
• W4377696088 doi "https://doi.org/10.1016/j.clim.2023.109547" @default.
• W4377696088 hasPublicationYear "2023" @default.
• W4377696088 type Work @default.
• W4377696088 citedByCount "0" @default.
• W4377696088 crossrefType "journal-article" @default.
• W4377696088 hasAuthorship W4377696088A5051464718 @default.
• W4377696088 hasAuthorship W4377696088A5067213148 @default.
• W4377696088 hasAuthorship W4377696088A5076514127 @default.
• W4377696088 hasAuthorship W4377696088A5091465356 @default.
• W4377696088 hasConcept C126322002 @default.
• W4377696088 hasConcept C141071460 @default.
• W4377696088 hasConcept C203014093 @default.
• W4377696088 hasConcept C2776694085 @default.
• W4377696088 hasConcept C2777063308 @default.
• W4377696088 hasConcept C2777921159 @default.
• W4377696088 hasConcept C71924100 @default.
• W4377696088 hasConcept C90924648 @default.
• W4377696088 hasConceptScore W4377696088C126322002 @default.
• W4377696088 hasConceptScore W4377696088C141071460 @default.
• W4377696088 hasConceptScore W4377696088C203014093 @default.
• W4377696088 hasConceptScore W4377696088C2776694085 @default.
• W4377696088 hasConceptScore W4377696088C2777063308 @default.
• W4377696088 hasConceptScore W4377696088C2777921159 @default.
• W4377696088 hasConceptScore W4377696088C71924100 @default.
• W4377696088 hasConceptScore W4377696088C90924648 @default.
• W4377696088 hasLocation W43776960881 @default.
• W4377696088 hasOpenAccess W4377696088 @default.
• W4377696088 hasPrimaryLocation W43776960881 @default.
• W4377696088 hasRelatedWork W1983542179 @default.
• W4377696088 hasRelatedWork W2002120878 @default.
• W4377696088 hasRelatedWork W2003938723 @default.
• W4377696088 hasRelatedWork W2047967234 @default.
• W4377696088 hasRelatedWork W2118496982 @default.
• W4377696088 hasRelatedWork W2364998975 @default.
• W4377696088 hasRelatedWork W2439875401 @default.
• W4377696088 hasRelatedWork W4238867864 @default.
• W4377696088 hasRelatedWork W2519357708 @default.
• W4377696088 hasRelatedWork W2525756941 @default.
• W4377696088 hasVolume "250" @default.
• W4377696088 isParatext "false" @default.
• W4377696088 isRetracted "false" @default.
• W4377696088 workType "article" @default.