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.

Background: Acquired haemophilia A (AHA) is a rare and severe bleeding disorder generally associated with pregnancy or aging. Spontaneous remission and prompt inhibitor eradication are described more frequently in postpartum cases. We evaluated retrospectively 15 postpartum AHA cases between 2007 and 2023 in order to evaluate response in terms of inhibitor eradication. Results: The median age at diagnosis was 31 years (range 24–38). All patients reported bleeding at presentation after a median period of 40.6 days following delivery (range 2–180 days). The median FVIII level was 4.4% (range 0%–12.8%), with a median FVIII‐inhibitor titer of 35 BU (range 2–156). The most severe bleeding symptoms were metrorrhagia and genital bleeding in nine patients (60%), and one patient had an important muscular haematoma. Two patients underwent hysterectomy before diagnosis due to severe bleeding. All patients required anti‐haemorrhagic therapy with a median duration of 8 days (range 1–28 days): 60% (9/15) with eptacog alfa, two with an activated prothrombin complex concentrate, and in combination in four cases. The immunosuppressive treatment was corticosteroids alone in eight patients (53%), cyclophosphamide or azathioprine in combination with corticosteroids in four, while rituximab was used in two cases following traditional immunosuppressive therapy. After a median period of 28 days (range 10–210 days), the anti‐FVIII inhibitor was eradicated with normalisation of coagulation in all but one patient. However, immunosuppressive therapy, including tapering, had a median duration of 2.3 months (range 1–23 months). At the time of data censoring, all patients were alive and well at the last follow‐up with no significant adverse events. Summary/Conclusion: Notwithstanding that postpartum AHA has been reported to have a high rate of spontaneous remission, nearly half of this series experienced inhibitor eradication more than 1 month after disease onset and using immunosuppressive treatment for more than 2 months, with additional drugs being used in more than 40% of them, thus showing difficulties in disease remission in this postpartum AHA subpopulation.

Acute promyelocytic leukemia is a rare form of acute myeloid leukemia in which immature promyelocytes abnormally proliferate in the bone marrow. In most cases, the disease is characterised by the translocation t(15;17) (q24;q21), which causes the formation of PML::RARA, an oncogenic fusion protein responsible for blocking myeloid differentiation and survival advantage. Here, we present a case of acute promyelocytic leukemia with two unusual features: basophilic differentiation and a three-way translocation involving chromosomes 12, 15 and 17. In the few cases reported, basophilic differentiation was associated with a poor prognosis. In contrast, our patient responded promptly to the standard treatment with all-trans-retinoic acid (ATRA) and arsenic trioxide (ATO) and obtained complete remission. To our knowledge, this is the first report of basophilic acute promyelocytic leukemia with the three-way translocation t(12;17;15) (p13; q24;q21).

Background: Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a rare, life-threatening disorder caused by severe ADAMTS13 deficiency, leading to microvascular platelet-rich thrombi, hemolysis, and organ damage, mainly involving brain, heart, kidneys, and intestines. Standard treatment includes plasma exchange (PEX) and immunosuppressants. Caplacizumab (CAPLA), an anti-von Willebrand factor nanobody, was recently introduced into clinical practice. Methods: We retrospectively analyzed 81 patients diagnosed with iTTP at our center from 1990 to 2024, accounting for 165 acute episodes requiring treatment. We compared outcomes between episodes treated with standard of care (SOC) and those with CAPLA, evaluating number of PEX sessions to platelet normalization, hospitalization length, CAPLA use, and disease stage (new onset vs. relapse). CAPLA was administered from episode onset until 30 days after PEX suspension. Results: Median age at first episode was 39.9 years (range 13–75), with 63% female patients. Over a median follow-up of 6.4 years, 47 patients (58%) experienced at least one relapse (range 1–7), all treated with PEX and corticosteroids. Of the 165 episodes, 39 (25.5%) were treated with CAPLA, 126 (74.5%) with SOC. Baseline characteristics were similar. Median PEX sessions were significantly lower in the CAPLA group (6 vs. 9.5; p < 0.0001), as were hospitalization days (8 vs. 13.5; p < 0.0001). Exacerbation after PEX occurred in 11 patients, more frequently in the CAPLA group (13% vs. 5%), associated with higher inhibitor levels (66 U/mL). Interestingly, patients who relapsed later had lower inhibitor levels at baseline (34 vs. 63 U/mL; p = 0.045) but longer hospital stays (12 vs. 10 days; p = 0.03), possibly due to less frequent CAPLA use (18% vs. 30%). Conclusion: Caplacizumab significantly reduces PEX sessions and hospitalization duration, leading to faster clinical response, regardless of disease stage or inhibitor level. However, its use may be linked to a higher rate of exacerbation, potentially due to earlier PEX discontinuation.