Physicians’ Perceptions of Clinical Utility of a Digital Health Tool for Electronic Patient-Reported Outcome Monitoring in Real-Life Hematology Practice. Evidence From the GIMEMA-ALLIANCE Platform

Digital health tools are increasingly being used in cancer care and may include electronic patient-reported outcome (ePRO) monitoring systems. We examined physicians’ perceptions of usability and clinical utility of a digital health tool (GIMEMA-ALLIANCE platform) for ePRO monitoring in the real-life practice of patients with hematologic malignancies. This tool allows for the collection and assessment of ePROs with real-time graphical presentation of results to medical staff. Based on a predefined algorithm, automated alerts are sent to medical staff. Participating hematologists completed an online survey on their experience with the platform. Of the 201 patients invited to participate between December 2020 and June 2021 (cut-off date for current analysis), 180 (90%) agreed to enter the platform and had a median age of 57 years. Twenty-three hematologists with a median age of 42 years and an average of 17 years of experience in clinical practice were surveyed. All hematologists agreed or strongly agreed that the platform was easy to use, and 87%, agreed or strongly agreed that ePROs data were useful to enhance communication with their patients. The majority of physicians (78%) accessed the platform at least once per month to consult the symptom and health status profile of their patients. The frequency of access was independent of physician sex (p=0.393) and years of experience in clinical practice (p=0.404). In conclusion, our preliminary results support the clinical utility, from the perspective of the treating hematologist, of integrating ePROs into the routine cancer care of patients with hematologic malignancies

Usefulness of bronchoalveolar lavage in suspect COVID-19 repeatedly negative swab test and interstitial lung disease

The diagnosis of coronavirus disease 2019 (COVID-19) relies on nasopharyngeal swab, which shows a 20–30% risk of false negativity [1]. Bronchoalveolar lavage (BAL) is reported to be useful in patients with pulmonary interstitial infiltrates on high-resolution computed tomography (HRCT). We investigated the usefulness of BAL in symptomatic patients with positive HRCT and a repeatedly negative swab test (‘grey zone’)

Heel raises versus prefabricated orthoses in the treatment of posterior heel pain associated with calcaneal apophysitis (Sever's Disease): study protocol for a randomised controlled trial

<p>Abstract</p> <p>Background</p> <p>Posterior Heel pain can present in children of 8 to 14 years, associated with or clinically diagnosed as Sever's disease, or calcaneal apophysitis. Presently, there are no comparative randomised studies evaluating treatment options for posterior heel pain in children with the clinical diagnosis of calcaneal apophysitis or Sever's disease. This study seeks to compare the clinical efficacy of some currently employed treatment options for the relief of disability and pain associated with posterior heel pain in children.</p> <p>Method</p> <p>Design: Factorial 2 × 2 randomised controlled trial with monthly follow-up for 3 months.</p> <p>Participants: Children with clinically diagnosed posterior heel pain possibly associated with calcaneal apophysitis/Sever's disease (n = 124).</p> <p>Interventions: Treatment factor 1 will be two types of shoe orthoses: a heel raise or prefabricated orthoses. Both of these interventions are widely available, mutually exclusive treatment approaches that are relatively low in cost. Treatment factor 2 will be a footwear prescription/replacement intervention involving a shoe with a firm heel counter, dual density EVA midsole and rear foot control. The alternate condition in this factor is no footwear prescription/replacement, with the participant wearing their current footwear.</p> <p>Outcomes: Oxford Foot and Ankle Questionnaire and the Faces pain scale.</p> <p>Discussion</p> <p>This will be a randomised trial to compare the efficacy of various treatment options for posterior heel pain in children that may be associated with calcaneal apophysitis also known as Sever's disease.</p> <p>Trial Registration</p> <p>Trial Number: ACTRN12609000696291</p> <p>Ethics Approval Southern Health: HREC Ref: 09271B</p

Cytogenetic and molecular responses in chronic phase chronic myeloid leukaemia patients receiving low dose of imatinib for intolerance to standard dose.

Imatinib mesylate is the gold standard treatment of chronic myeloid leukaemia (CML) and 400 mg/day is considered the standard dose. Although it is generally well tolerated, some patients require temporary drug discontinuation and permanent dose reduction because of haematological or non-haematological toxicities. Whether or not reduced doses are effective as the standard dose in inducing and/or maintaining complete cytogenetic and molecular response is not clear. We report the outcome of 45 CML patients in early (17) and late (28) chronic phase (CP) in whom, within a series of 250 patients treated with imatinib, reduced the dose of the drug after experiencing adverse events. Median time interval between the start of therapy and dose reduction was 58 days, whereas median administered dose was 300 mg/day. At 6 months from reduction, major cytogenetic responses (MCRs) were observed in 67% of patients, with 58% being complete cytogenetic remission (CCR), and complete molecular response (CMR) were obtained in 18% of patients. At 12 months, all patients who had obtained MCR reached CCR: this was significantly higher in low risk patients (87%) versus non-low risk patients (66 and 46%), and in early phase (82%) versus late phase (53.5%). CMR and major molecular response (MMR) were detected in 20 and 22% of patients, respectively. Low dose imatinib appears effective in patients with intolerance to standard dose, even though long-term effects remain to be established

Isolated central nervous system relapse after nine years of complete molecular remission in a lymphoid blast crisis of chronic myeloid leukemia treated with imatinib.

Extramedullary blast crisis (BC) of chronic myeloid leukemia (CML) has been observed in 7–10% of patients, with or without other manifestation of disease progression [1]. Cerebrospinal involvement as a site of extramedullary BC is rare, but since the advent of imatinib as first-line treatment for CML, there have been several reports describing patients with isolated central nervous system (CNS) involvement [1], [2], [3], [4] and [5] E. Simpson, S.G. O’Brient and J.T. Reilly, Extramedullary blast crisis in CML patients in complete haematological remission treated with imatinib mesylate. Clin Lab Haematol, 28 (2006), pp. 215–216. | View Record in Scopus | | Full Text via CrossRef | Cited By in Scopus (3)[5], because the drug poorly penetrates the blood-brain barrier. Here we report a case of a lymphoid BC, developed under IFN therapy, which reached complete molecular remission with imatinib and presented an isolated relapse of the same disease in CNS after nine years of imatinib therap

TROMBOCITOPENIA IDIOPATICA (ITP) CRONICA: COSA PUO’ NASCONDERE?

Circa il 30% di bambini con trombocitopenia idiopatica (ITP) sviluppa una forma cronica, di entità variabile anche nella sintomatologia emorragica. Per diagnosticare una eventuale forma ereditaria, abbiamo approfondito le indagini in 11 pazienti con ITP (età mediana alla diagnosi: 7 anni, range 2-16) e con una durata mediana della piastrinopenia di 12 anni, resistenti a più linee di trattamento, 4 dei quali con sintomatologia emorragica. Di 5 pazienti abbiamo valutato anche i familiari. Complessivamente, abbiamo studiato 20 soggetti (11 pazienti e 9 familiari) con metodica NGS che hanno evidenziato una o più mutazioni in eterozigosi dei geni che regolano lo sviluppo e la funzione delle piastrine. Abbiamo completato lo studio in 3 delle 5 famiglie: in una famiglia è stata riscontrata la mutazione FLNA, in un’altra le mutazioni ABCG8, ETV6 e WAS, e nella terza famiglia, un genitore presenta, oltre la mutazione ANRKD26, presente nel paziente, anche le mutazioni GP1BA e NBAL2. Dei 6 pazienti, di cui è in corso lo studio familiare, 4 presentano mutazioni associate alle forme familiari di ITP (SLFN14, ABCG8, ETV6, NBEAL2), 2 non presentano mutazioni. Dei 3 pazienti con mutazioni del gene NBAEL2: uno ha un fenotipo compatibile con sindrome delle piastrine grigie; negli altri la valutazione è in corso. In sintesi, i geni e le mutazioni possibilmente coinvolte nelle ITP familiari è ampio ed il loro significato clinico è ancora da chiarire. È quindi necessario condurre un’indagine genetica nei bambini affetti da ITP cronica ed estenderla alla famiglia per una corretta gestione delle forme di trombocitopenia