Abstrakt: |
Of the PNH positive patients, 8 patients were known to have a PNH clone with aplastic anaemia; one patient had a hypoplastic bone marrow and a known PNH clone whilst only one patient with cytopenia had a new positivity for PNH. Seven patients with progressive clinical features had ASCT consolidation (median age 59 years [range 43-68]): three patients had additional parenchymal intracranial disease on MRI and two patients also had peripheral nerve involvement, including one patient with suspicion of high-grade transformation (HGT) on CSF. Out of the 206 patients that received 1st line treatment, 92 (44%) patients proceeded to 2nd line treatment, 31 (15%) patients proceeded to 3rd line treatment and 21 (10%) of patients required treatment beyond 3rd line Within the poor risk category, 173 patients were identified with 38 had TP53 deletion or mutation and 135 patients had other poor risk mutations such as NOTCH1, SF3B1, ATM. Patients were risk stratified into good, poor or not poor risk categories as per Rodríguez-Vicente et al. SP 2 sp B Statistical Analysis and Results: b For 349 patients analysed, 143 (41%) patients were under active surveillance, and 206 (59%) patients received 1st line treatment. B Methodology: b Using a mixed methods approach we evaluated the app via: usage data from Google's Firebase platform (https://firebase.google.com/) healthcare professional benchmarking, using the Mobile App Rating Scale (MARS) questionnaire patient questionnaires, using the mHealth App Usability Questionnaire (MAUQ) patient focus groups one-to-one patient interviews Patient recruitment was undertaken via CML online support groups. [Extracted from the article] |