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 (
=0.393) and years of experience in clinical practice (
=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.
Imatinib has become first line therapy in chronic myeloid leukemia patients. Little is known about the infective consequences during the treatment with this drug in large series of chronic phase ...patients.
From January 2001 to September 2006 we treated with imatinib 250 patients in first line (early CP) or after interferon failure (late CP), out of clinical trials and recorded all the bacterial and viral infections occurred.
We recorded a similar incidence of bacterial and viral infections both in first line and late CP patients (respectively, 16% and 13%) during 3.5 years of follow-up. Analysis of presenting features predisposing to infections revealed differences only in late CP patients, with elevated percentage of high Sokal risk patients and a more longer median time from diagnosis to start of imatinib.
Opportunistic infections and reactivation of Herpes Zoster are observed during imatinib therapy at very low incidence.
The differential diagnosis of marrow failure (MF) is crucial in the diagnostic work‐up, since genetic forms require specific care. We retrospectively studied all patients with single/multi‐lineage MF ...evaluated in a single‐center to identify the type and incidence of underlying molecular defects. The diepoxybutane test was used to screen Fanconi Anemia. Other congenital MFs have been searched using Sanger and/or Next Generation Sequencing analysis, depending on the available tools over the years. Between 2009–2019, 97 patients (aged 0–32 years‐median 5) with single‐lineage (29%) or multilineage (68%) MF were evaluated. Fifty‐three (54%) and 28 (29%) were diagnosed with acquired and congenital MF, respectively. The remaining 16 (17%), with trilinear (n=9) and monolinear (n=7) MF, were found to have an underlying primary immunodeficiency (PID) and showed clinical and biochemical signs of immune‐dysregulation in 10/16 (62%) and in 14/16 (87%) of cases, respectively. Clinical signs were also found in 22/53 (41%) and 8/28 (28%) patients with idiopathic and classical cMF, respectively. Eight out of 16 PIDs patients were successfully transplanted, four received immunosuppression, two did not require treatment, and the remaining two died. We show that patients with single/multi‐lineage MF may have underlying PIDs in a considerable number of cases and that MF may represent a relevant clinical sign in patients with PIDs, thus widening their clinical phenotype. An accurate immunological work‐up should be performed in all patients with MF, and PID‐related genes should be considered when screening MF in order to identify disorders that may receive targeted treatments and/or appropriate conditioning regimens before transplant.
Background Survival of high-risk Myelodysplastic Syndromes (MDS) treated with azacytidine during the dysplastic phase and then evolved in Acute Myeloid Leukemia (AML-MDS) is very poor. While the ...association of hypometilating agents (HMA) and venetoclax (VEN) is widely used in de novo AML not eligible for intensive chemotherapy, very few data are available in patients (pts) with AML-MDS.
Methods Data of 42 pts with AML-MDS treated frontline with HMA+VEN in 19 hematologic Centres in Italy outside clinical trials from 5/2018 to 3/2023 were retrospectively collected and analysed. Composite overall response rate ORR; complete remission (CR) + CR with incomplete hematologic recovery (iCR) + partial remission (PR) + hematologic improvement (HI), duration of response and overall survival (OS) were assessed.
Results: Baseline characteristics at evolution in AML are reported in the Table. Median interval from initial MDS diagnosis to evolution was 16.7 months interquartile range (IQR) 8.2-30.1. Pts were treated for a median of 3 courses (IQR 2-6): HMA were administered at standard dosage, VEN daily doses in the 1 st cycle are reported in the Table. On the whole, 34 pts (81.0%) had at least one hematologic toxicity of grade 3-4: in particular, severe neutropenia (PMN < 0.5 x 10 9/l) was reported in 32 pts (76.1%). Eighteen pts (42.8%) had at least one infective episode during the treatment: pulmonary infections were reported in 9 pts (21.4%). Response to treatment is shown in the Table: ORR was 52.4%, with a median response duration of 17.8 months (95%CI 3.3-32.2). After a median follow-up from AML evolution of 6.1 months (IQR 2.2-11.6), 31 pts (73.8%) died and 11 (26.2%) were alive. Median OS from AML evolution of the whole cohort was 6.9 months (95%CI 4.7-9.1), Pts with any response to HMA+VEN had a significantly longer OS compared to pts with progressive/stable disease 15.6 (95%CI 11.3-19.8) versus 3.3 (95%CI 0.7-5.8) months, respectively (p<0.001) (Figure).
Conclusions: Our real-life data suggested that HMA+ VEN combination could be useful also in AML-MDS already treated with azacytidine in the dysplastic phase, with an ORR of about 50% in pts unfit for intensive approaches: however, hematologic and infective toxicities were severe and the response duration was short, with a persistently poor median OS. As a consequence, addition of other targeted therapies driven by NGS should be explored in the next future.
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