International workshop: what is needed to ensure outcome measures for Rett syndrome are fit-for-purpose for clinical trials? June 7, 2023, Nashville, USA.

Introduction: The clinical, research and advocacy communities for Rett syndrome are striving to achieve clinical trial readiness, including having fit-for-purpose clinical outcome assessments. This study aimed to (1) describe psychometric properties of clinical outcome assessment for Rett syndrome and (2) identify what is needed to ensure that fit-for-purpose clinical outcome assessments are available for clinical trials.

Methods: Clinical outcome assessments for the top 10 priority domains identified in the Voice of the Patient Report for Rett syndrome were compiled and available psychometric data were extracted.

Phase I clinical trial testing the dose escalation and expansion of Pressurized IntraThoracic Hyperthermic Aerosol Cisplatin administration (PITHAC) for the management of pleural carcinosis.

Background: Pleural carcinosis originates from various cancers. Its management consists in systemic therapies combined to dyspnea relief procedures. Prior studies have tested hyperthermic intrathoracic chemotherapy to treat pleural carcinosis with interesting patient survival results.

Papilledema in Children With Cystic Fibrosis Receiving Elexacaftor/Tezacaftor/Ivacaftor: A Multicenter Case Series.

Following the approval of elexacaftor/tezacaftor/ivacaftor (ETI), there have been post-marketing reports and published cases of papilledema and intracranial hypertension in children with cystic fibrosis (CF) taking ETI. In those reports, the patients often presented with marked symptoms and concomitant hypervitaminosis A. In this multicenter case series, we report eight cases of papilledema in children with CF taking ETI that were diagnosed via routine eye exam, the majority of whom presented with minimal to no symptoms and all had normal serum Vitamin A levels.

Bad habits that stick: adhesive tape use practices and beliefs.

Tape rolls are often used for multiple patients despite recommendations by manufacturers for single-patient use. We developed a survey to query Health Care Personnel about their tape use practices and beliefs and uncovered behaviors that put patients at risk for hospital-acquired infections due to tape use.