A rare case of appendicular skeleton localization in a patient with chronic lymphocytic leukemia successfully treated with salvage radiation therapy

Bone involvements in chronic lymphocytic leukemia (CLL) are considered rare events, and the English-language medical literature describes them only in sporadic case reports. Consequently, robust indications for a rational clinical management are lacking. We report the case of a middle-aged man in clinical follow-up for CLL who experienced pain at the right tibial level that was refractory to nonsteroidal anti-inflammatory drugs and an acute episode of anemia. Instrumental examinations and a bioptic sample surprisingly demonstrated a bone tibial localization by CLL

Prednisone vs high-dose dexamethasone in newly diagnosed adult primary immune thrombocytopenia: a randomized trial

A debate exists regarding which type of corticosteroids (standard-dose prednisone [PDN] or high-dose dexamethasone [HD-DXM]) is the best first-line treatment for adult patients with newly diagnosed untreated primary immune thrombocytopenia (pITP). An ad hoc study compared PDN with HD-DXM in newly diagnosed untreated patients with pITP (aged >= 18 but <= 80 years, platelet count of <= 20 or >20 but <50 x 10(9)/L, and bleeding score of >= 8). Patients were randomised to receive PDN 1 mg/kg per day from days 0 to 28 (Arm A) or HD-DXM 40 mg per day for 4 days, every 14 days, for 3 consecutive courses (Arm B). Fifty-nine of 113 patients (52.2%) were randomized to Arm A and 54 of 113 (47.8%) to Arm B. In evaluable patients, total initial responses (complete response [CR], partial response [PR], minimal response [MR]) were 44 of 56 (78.57%) in Arm A and 46 of 49 (93.88%) in Arm B at days 42 and 46, respectively (P = 0.0284). Total final responses (at day 180 from initial response) were 26 of 43 (60.47%) in Arm A and 23 of 39 (58.97%) in Arm B (P = 0.8907). Total persistent responses (at 12 months from initial response) were 25 of 31 (80.65%) in Arm A and 20 of 36 (55.56%) in Arm B (P = 0.0292). Seven relapses occurred. Median follow-up was 44.4 months. Overall survival was 100% at 48 months, overall disease-free survival was 81.11% at 48 months from day 180. PDN and pulsed HD-DXM were well tolerated; HD-DXM allows effective initial responses but less long lasting than PDN. This trial was registered at www.clinicaltrials.gov as #NCT00657410

Elotuzumab plus pomalidomide and dexamethasone in relapsed/refractory multiple myeloma: a multicenter, retrospective real-world experience with 200 cases outside of controlled clinical trials

In the ELOQUENT-3 trial, the combination of elotuzumab, pomalidomide and dexamethasone (EloPd) proved a superior clinical benefit over Pd with a manageable toxicity profile, leading to its approval in relapsed/refractory multiple myeloma (RRMM), who had received at least two prior therapies, including lenalidomide and a proteasome inhibitor (PI). We report here a real-world experience of 200 RRMMs treated with EloPd in 35 Italian centers outside of clinical trials. In our dataset, the median number of prior lines of therapy was 2, with 51% of cases undergoing autologous stem cell transplant (ASCT) and 73% exposed to daratumumab. After a median follow-up of 9 months, 126 patients stopped EloPd, most of them (88.9%) because of disease progression. The overall response rate (ORR) was 55.4%, in line with the pivotal trial results. Regarding adverse events, our cohort experienced a toxicity profile similar to the ELOQUENT-3 trial, with no significant differences between younger (<70 years) and older patients. The median progression-free survival (PFS) was 7 months, shorter than that observed in the ELOQUENT-3, probably due to the different clinical characteristics of the two cohorts. Interestingly, the ISS stage III (HR:2.55) was associated with worse PFS. Finally, our series's median overall survival (OS) was shorter than that observed in the ELOQUENT-3 trial (17.5 versus 29.8 months). In conclusion, our real-world study confirms EloPd as a safe and possible therapeutic choice for RRMM who received at least two prior therapies, including lenalidomide and a PI

Diabetes and Second Neoplasia Impact on Prognosis in Pre-Fibrotic Primary Myelofibrosis

The 2016 WHO classification recognized pre-fibrotic primary myelofibrosis (pre-PMF) as a distinct entity. Nevertheless, a prognostic model specific for pre-PMF is still lacking. Our aim was to identify the most relevant clinical, histological, and driver mutation information at diagnosis to evaluate outcomes in pre-PMF patients in the real-world setting. We firstly assessed the association between IPSS or DIPSS at diagnosis and response variables in 378 pre-PMF patients. A strict association was observed between IPSS and DIPSS and occurrence of death. Other analyzed endpoints were not associated with IPSS or DIPSS as thrombo-hemorrhagic events at diagnosis or during follow-up, or did not show a clinical plausibility, as transformation into acute leukemia or overt PMF. The only covariates which were significantly associated with death were diabetes and second neoplasia, and were therefore included in two different prognostic settings: the first based on IPSS at diagnosis [class 1 vs. 0, OR (95%CIs): 3.34 (1.85–6.04); class 2 vs. 0, OR (95%CIs): 12.55 (5.04–31.24)], diabetes [OR (95%CIs): 2.95 (1.41–6.18)], and second neoplasia [OR (95%CIs): 2.88 (1.63–5.07)]; the second with DIPSS at diagnosis [class 1 vs. 0, OR (95%CIs): 3.40 (1.89–6.10); class 2 vs. 0, OR (95%CIs): 25.65 (7.62–86.42)], diabetes [OR (95%CIs): 2.89 (1.37–6.09)], and second neoplasia [OR (95%CIs): 2.97 (1.69–5.24)]. In conclusion, our study underlines the importance of other additional risk factors, such as diabetes and second neoplasia, to be evaluated, together with IPSS and DIPSS, to better define prognosis in pre-PMF patients

Multiple Myeloma in 2023 Ways: From Trials to Real Life

Multiple myeloma is a chronic hematologic malignancy that obstinately tends to relapse. Basic research has made giant strides in better characterizing the molecular mechanisms of the disease. The results have led to the manufacturing of new, revolutionary drugs which have been widely tested in clinical trials. These drugs have been approved and are now part of the therapeutic armamentarium. As a consequence, it is essential to combine what we know from clinical trials with real-world data in order to improve therapeutic strategies. Starting with this premise, our review aims to describe the currently employed regimens in multiple myeloma and compare clinical trials with real-life experiences. We also intend to put a spotlight on promising therapies such as T-cell engagers and chimeric antigen receptor T-cells (CAR-T) which are proving to be effective in changing the course of advanced-stage disease