Objective: Immune thrombocytopenia (ITP) is an acquired thrombocytopenia resulting from autoantibodies against platelet antigens. In this study, we aimed to analyze demographics, clinical characteristics, and treatment responses in our patients with chronic ITP on follow-up.Methods: This is a retrospective study. Data, including sociodemographic and laboratory data, treatments received, and response rates, were retrieved from patients’ electronic health records or paper charts.Results: A total of 92 patients were included in the study, and 62 (67.4%) were females. The median age of study patients was 40 years (range: 18-81years). During follow-up, evidence of bleeding was detected in 35 patients. Gastrointestinal bleeding was observed in four patients, and intracranial bleeding in three patients. No statistically significant difference was found in response rates between methylprednisolone and dexamethasone (p=0.795) in the first-line treatment and between eltrombopag and splenectomy (p=0.502) in the second-line treatment. Patients undergoing splenectomy were more likely to develop thrombosis.Conclusion: In this cohort of chronic ITP patients, the disease was more prevalent in females, with a median age of 40 years, and bleeding complications occurred in a substantial proportion of patients, including rare but serious events such as intracranial hemorrhage. No significant differences were observed in response rates between first-line treatments (methylprednisolone vs. dexamethasone) or second-line treatments (eltrombopag vs. splenectomy). However, splenectomy was associated with a higher risk of thrombosis. These findings highlight the need for individualized treatment decisions and emphasize that standardization of treatment strategies and follow-up duration is essential to better evaluate treatment responses and long-term outcomes in chronic ITP.
Introduction & Objective: Graft-versus-host disease (GVHD) is a major complication of allogeneic hematopoietic stem cell transplantation (allo-HSCT), with limited treatment options for steroid-resistant cases. Ruxolitinib, a JAK1/2 inhibitor, has shown promise in treating steroid-resistant acute (aGVHD), chronic (cGVHD), and overlap GVHD (oGVHD), but real-world data remain limited. This study evaluated the real-world efficacy and safety of ruxolitinib in allo-HSCT patients with steroid-resistant GVHD. Materials & Methods: This retrospective, multicenter study included adult patients treated with ruxolitinib for Grade II or higher aGVHD or moderate-to-severe cGVHD at nine centers in Turkey (2017-2024). Clinical characteristics, treatment responses, and adverse events were recorded. Primary outcomes were overall response rate (ORR) and overall survival (OS). Results: Among 80 patients (mean age: 39.3 ± 13.3 years; 60 males), 39 had aGVHD, 68 cGVHD, and 15 oGVHD. The ORR was 72 of 80 patients (90.0%) (complete response: 37 of 80 [46.3%], partial response: 35 of 80 [43.8%]). The 1-year and 2-year OS rates were 91.3% and 82.5%. Severe cGVHD (p < 0.001) and lack of response to ruxolitinib (p = 0.018) were associated with reduced OS. Adverse events included infections in 40 of 80 patients (50.0%), cytopenias in 23 of 80 (28.7%), and cytomegalovirus reactivation in 20 of 80 (25.0%). Conclusion: In this retrospective multicenter cohort, ruxolitinib was associated with high response rates in steroid-refractory GVHD, while disease severity remained a key determinant of survival, and findings should be interpreted as exploratory.
PEGylated interferon-α (Peg-IFN) is a highly effective therapy for polycythemia vera (PV) and essential thrombocythemia (ET), owing to its lower immunogenicity and reduced toxicity compared with conventional interferon-α. This retrospective study aimed to evaluate the efficacy on hematological response, the response generation process and safety of Peg-IFN alfa-2a treatment in patients with PV or ET. In this study, 68 patients with diagnosed ET or PV who had received Peg-IFN alfa-2a between May 2016 and May 2022 were enrolled. Peg-IFN therapy response assessment was performed according to the European Leukemia Net and the International Working Group-Myeloproliferative Neoplasms Research and Treatment consensus revised response criteria. Forty patients (58.8
The use of venetoclax in combination with hypomethylating agents (HMAs) has become a standard treatment approach for both newly diagnosed and relapsed/refractory (R/R) AML patients who are ineligible for intensive chemotherapy. We aimed to share the real-life data of this combination therapy. This retrospective study included 38 patients with newly diagnosed or R/R AML who received at least one cycle of venetoclax-HMA combination therapy between July 2018 and August 2025. Response status, survival rates, and the incidence of hematological adverse events were analyzed. Twelve patients (31.6
Background: Elranatamab, a bispecific antibody targeting BCMA and CD3, has demonstrated clinical activity in relapsed/refractory multiple myeloma. Real-world evidence regarding infectious complications and supportive care remains limited. We evaluated the early clinical activity, safety profile, infectious complications, and supportive care practices associated with relapsed/refractory multiple myeloma (RRMM). This study represents one of the first multicenter real-world evaluations of elranatamab in Türkiye. Methods: This multicenter retrospective study included 87 patients with relapsed/refractory multiple myeloma treated with elranatamab. Clinical characteristics, treatment responses, immune-mediated toxicities, infectious complications, and supportive care practices were assessed. Overall response rate (ORR), progression-free survival (PFS), and overall survival (OS) were evaluated. Multivariable analyses were performed to identify factors associated with treatment response and clinical outcomes. Results: At 3 months, ORR was 47.1% in the ITT population, 54.7% in the mITT population, and 83.7% among evaluable patients; corresponding 6-month ORRs were 31.0%, 42.2%, and 81.8%, respectively. The high proportion of patients without landmark response assessments primarily reflected insufficient follow-up, early death, or disease progression. Elevated LDH remained independently associated with lower response probability and inferior clinical outcomes. Cytokine release syndrome (CRS) occurred in 69% of patients, with grade ≥ 3 events in 5.7%, whereas immune effector cell-associated neurotoxicity syndrome (ICANS) was infrequent (4.6%) and no grade ≥ 3 events occurred. Grade ≥ 3 infections occurred in 39% of patients, including CMV events requiring antiviral treatment in 24.1%. No HBV reactivation occurred among patients receiving antiviral prophylaxis. Median PFS and OS were 8.1 and 10.6 months, respectively. Conclusions: Elranatamab demonstrated early clinical activity and a manageable safety profile in a heavily pretreated real-world RRMM population. Infectious complications, including CMV events, remained clinically relevant, emphasizing the importance of supportive care. Longer follow-up is needed to characterize long-term outcomes.
Abstract The lifespan of patients with chronic phase chronic myeloid leukemia undergoing tyrosine kinase inhibitor treatment has nearly reached that of the general population. However, treatment switching due to resistance or intolerance demonstrates the significance of effective communication between patients and physicians in decision-making. This study aimed to evaluate priorities, expectations, and perceptions of managing CML-CP from the perspective of both patients and physicians in Türkiye. This cross-sectional survey study was conducted from July to December 2024 by the Turkish Society of Hematology. A total of 129 hematologists completed a web-based survey, while 120 patients receiving TKI treatment completed a structured telephone survey. Eleven common questions were analyzed descriptively to compare perspectives. Patients (53% male; median age 54 years) and physicians agreed on essential information to be communicated at treatment start, although priorities differed. Patients prioritized information about disease progression, daily life impact, and treatment effectiveness, whereas physicians focused on treatment safety, patient monitoring, and follow-up assessments. Patients (40.8%) emphasized treatment effectiveness when switching therapies, while physicians (46.7%) focused on the manageability of adverse events. Approximately 25% of patients reported suboptimal adherence, mostly due to forgetfulness (52%). Follow-up visits occurred every 2 to 3 months, with a mean duration of approximately 10 min. Although patients and physicians shared broadly similar views, discrepancies existed in their prioritization of factors and overall opinions. Patients expressed greater optimism regarding the disease impact on their daily lives. Enhancing communication between patients and physicians, as well as promoting shared decision-making during treatment transitions, may improve adherence and disease management outcomes.
Background/Objectives: Real-world data on the therapeutic use of FLT3 inhibitors in Turkey remain limited. Therefore, we retrospectively evaluated outcomes from 13 academic centers nationwide, focusing on the multikinase inhibitor midostaurin in patients with newly diagnosed FLT3-mutated acute myeloid leukemia (AML). Methods: We collected comprehensive information regarding treatment efficacy, safety, and tolerability. Results: The overall response rate to intensive chemotherapy (3 + 7) plus midostaurin was 87.7%, with a complete remission rate of 84.2%, consistent with previously reported clinical trial results. Treatment discontinuation due to intolerance or toxicity was low (3.5%). One patient discontinued therapy because of septic shock during induction, and another due to a drug-drug interaction during consolidation. Median overall survival was 21.4 months. Allogeneic stem cell transplantation was performed in first remission in 52.6% of patients. Five patients (8.8%) were refractory to induction therapy, and relapse occurred in 21.1% (12 patients). Conclusions: These findings support the effectiveness and acceptable tolerability of midostaurin in routine clinical practice for FLT3-mutated AML.
Objective: This retrospective study aimed to evaluate the effects of ferritin level on outcomes of allogeneic hematopoietic stem cell transplantation (allo-HSCT) including the neutrophil/platelet engraftment, febrile neutropenia, transplant related mortality (TRM), graft versus host disease (GvHD), sinusoidal obstruction syndrome (SOS)/veno-occlusive disease (VOD) and overall survival (OS). Materials and Methods: Sixty-nine patients with ferritin values measured at the beginning of allo-HSCT between 2018 - 2021 were enrolled in this study. The ferritin cut-off value was determined as 1000ng/mL and the patients were divided into 2 groups (.05). The median OS in patients with ferritin level ≥1000 ng/ mL and ferritin level <1000 ng/mL 4 months (95% CI: 1.4-6.6) and 8 months (95% CI: 0-24.2), respectively. There was no statistically significant correlation between ferritin value and OS (p=0.206). There was no statistically significant difference between the ferritin groups on both acute and chronic GvHD (p=0.713 and p=0.999, respectively). Conclusion: Our study did not demonstrate any negative effects of serum ferritin levels on allo-HSCT outcomes; however, large-scale prospective studies are needed to clarify the effect of iron overload on the outcomes of allo-HSCT.
Objective: This study aimed to evaluate the prognostic significance of lymphocyte-associated inflammatory markers and the HALP score in patients with Hodgkin’s lymphoma. Methods: This was a retrospective study that included patients who were diagnosed with Hodgkin’s lymphoma and followed up between 2004 and 2024. The inflammatory markers (NLR, PLR, MLR, SII, SIRI, and PIV) and HALP score were calculated from the patients’ biochemical and hematological parameters, and the relationship between these parameters and stage, spleen and liver involvement, relapse, mortality, overall survival, and progression-free survival was analyzed. Results: A total of 117 patients were included, and multivariate analysis indicated that progression-free survival was statistically and significantly associated with treatment type (p = 0.0285), PLR (p = 0.0188), and PIV (p = 0.0297). In terms of overall survival, age (p = 0.0011), treatment type (p = 0.0108), and SIRI (p = 0.0108) remained as statistically significant predictors. Although the HALP score showed a significant association with PFS in the univariate analysis (p = 0.0104), this association did not persist in the multivariate model. In addition, no statistically significant relationship between the HALP score and OS was observed in either the univariate or multivariate analysis. Conclusions: The SIRI is a prognostic marker in Hodgkin’s lymphoma and may be useful for predicting overall survival.
Splanchnic vein thrombosis (SVT) is an uncommon but clinically significant complication of myeloproliferative neoplasms (MPNs), contributing to morbidity and management complexity. Evidence regarding prognostic factors and optimal anticoagulation strategies remains limited. We aimed to evaluate the clinical characteristics, risk factors, treatment strategies, and survival outcomes in patients with SVT associated with MPNs. In this multicenter retrospective cohort study, 289 adult patients with SVT associated with MPNs were analyzed. The median age at SVT diagnosis was 49 years, with 74
Background: T-cell lymphomas (TCLs) constitute a heterogeneous group of mature T-cell neoplasms characterized by aggressive clinical behavior and poor outcomes, particularly in the relapsed/refractory (R/R) setting. Belinostat is a pan-HDAC inhibitor approved for R/R peripheral T-cell lymphomas. In this retrospective study, we analyze the real-world outcomes of single-agent belinostat in Turkish patients with R/R TCLs. Methods: Patients with R/R TCLs across Turkey who received at least one course of belinostat were included in the study. The primary end point was the overall response rate (ORR). The secondary end points included progression-free survival (PFS), overall survival (OS) and duration of response (DOR). Results: A total of 59 patients with R/R TCL were enrolled in the study, with a median age of 62 years. The median number of previous lines of therapy before belinostat was 2.5. The ORR for belinostat was 30.5% in the overall study cohort. Response assessment was available in 53 patients, among whom the ORR was 34% (18/53). The median PFS was 3 months. The median OS was 12 months, and the median DOR was not reached in responders. At least one adverse event occurred in 39.0% of patients. Conclusions: Belinostat produced objective responses in a subset of patients. These findings support belinostat as a potential individualized treatment option rather than a broadly effective standard for all patients with R/R TCLs. This multicenter real-world study demonstrates that the observed response rates closely mirror those reported in prospective clinical trials, confirming the reproducibility of belinostat efficacy in routine clinical practice.
Objective Anemia is a common complication in patients with Chronic Kidney Disease (CKD), particularly in those not receiving dialysis. Roxadustat, a Hypoxia-Inducible Factor Prolyl Hydroxylase Inhibitor (HIF-PHI), has been investigated as a therapeutic option for anemia management in this population. This study aimed to evaluate the efficacy of Roxadustat compared to control interventions in Non-Dialysis-Dependent CKD (NDD-CKD) patients. Methodology A comprehensive literature search was conducted in Cochrane CENTRAL, Ovid Medline, PubMed, and Web of Science up to December 14, 2024. Randomized Controlled Trials (RCTs) directly comparing Roxadustat with a control group were included. Data were pooled using an inverse variance-weighted random-effects model. The primary efficacy outcome was the change in Hemoglobin (Hb) levels at weeks 24–28 and during follow-up. Subgroup analyses were performed based on the type of control intervention (Erythropoiesis-Stimulating Agents [ESAs] vs. placebo) and prior ESA use. Results A total of six RCTs, including 5,330 patients, from 520 unique records from the databases were included. Roxadustat significantly increased Hb levels during follow-up compared to the control group (Mean Difference [MD = 1.21 g/dL], 95% confidence interval [95% CI 0.45 to 1.97], I² = 99%, p = 0.0017). However, at weeks 24–28, the increase in Hb levels was not statistically significant (MD = 0.86 g/dL, 95% CI -0.11 to 1.83, I² = 99.4%, p = 0.0833). Iron-related parameters showed mixed results. Roxadustat was associated with a significant reduction in ferritin levels (MD = -38.54 ng/mL, 95% CI -68.21 to -8.87, I² = 84.1%, p = 0.0109). Conversely, Total Iron-Binding Capacity (TIBC) was significantly increased with Roxadustat treatment (MD = 20.33 μg/dL, 95% CI 1.15 to 39.51, I² = 98.5%, p = 0.0377). No significant difference was observed in serum iron (MD = 3.1 μg/dL, 95% CI -0.39 to 6.6, I² = 93.1%, p = 0.0820) and Transferrin Saturation (TSAT) levels (MD = -1.08%, 95% CI -2.42 to 0.26, I² = 40.1%, p = 0.1151) between the two groups. Subgroup analyses revealed that in placebo-controlled trials, Roxadustat significantly increased Hb levels at both weeks 24–28 and during follow-up. However, in trials comparing Roxadustat with ESAs, the changes in Hb levels were not significant at either time point. Conclusion Roxadustat reduced ferritin but increased TIBC without significantly affecting free iron and TSAT levels compared to the control group in patients with NDD-CKD.
Primary mediastinal B-cell lymphoma (PMBCL) is a rare and distinct subtype of non-Hodgkin lymphoma. No consensus exists on optimal frontline treatment, and the use of R-CHOP ± radiotherapy (RT) and DA-EPOCH-R ± RT remains common, yet comparative real-world data are limited. In our multicenter retrospective study, we analyzed PMBCL patients, stratified by the first-line therapy (R-CHOP-21 ± RT or DA-EPOCH-R ± RT). Primary outcomes were complete response (CR) rate, progression-free survival (PFS), and overall survival (OS), alongside assessment of treatment-related toxicities and prognostic factors for PFS and OS. We included 157 patients [R-CHOP ± RT group (n = 80) and DA-EPOCH-R ± RT group (n = 77)] with a median age of 31 years, of whom 68.2
Aim: Nodular lymphocyte-predominant Hodgkin lymphoma (NLPHL) is a rare subtype of lymphoma classified within Hodgkin lymphomas. Often the diagnosis is made at an early stage. Treatment varies according to the stage of the disease and the underlying prognostic factors. Because of cluster of differentiation 20 expression, rituximab-based agents can be used for treatment. In this study, we aimed to present the demographic, treatment, and survival data of patients with NLPHL in the context of the existing literature. Methods: In our study, demographic characteristics, laboratory findings, disease stage, and treatments administered to patients diagnosed with NLPHL between 2012 and 2024 were evaluated. Results: Of the 13 patients enrolled in the study, seven were male, and the median age was 44 years. Of the patients, eight were in the early stage and five were in the advanced stage. One patient had liver involvement, three had splenic involvement, seven had subdiaphragmatic involvement, and four had a bulky mass. Rituximab was added to the treatment regimens of six patients. Progression was observed in two patients. One patient died from Coronavirus disease 2019-related pneumonia. While the 2-year and 5-year overall survival of our patients were both 92%, progression-free survival was 100% at 2 years and 45.5% at 5 years. Conclusion: NLPHL is a rare condition that, despite its generally favorable prognosis, requires effective treatment because of the risk of recurrence and transformation into diffuse large B-cell lymphoma (DLBCL). Although data on the transformation of subdiaphragmatic involvement into DLBCL exist in the literature, information on disease progression is lacking.
BACKGROUND: Hodgkin’s lymphoma (HL) exhibits a cure rate of 90% in patients diagnosed at an early stage and a cure rate ranging from 70% to 90% in patients diagnosed at an advanced stage. In the case of patients with relapsed/refractory HL (r/rHL), it is recommended to provide salvage chemotherapy initially, followed by autologous stem cell transplantation (ASCT). The ideal conditioning regimen for the transplantation process is still being investigated. OBJECTIVES: For individuals with r/rHL, high-dose chemotherapy combined with ASCT (HD-ASCT) is thought to be the most effective method of treatment. The purpose of this research was to evaluate the effectiveness and safety of the busulfan, cyclophosphamide, and etoposide (BuCyE) preparation regimen in r/rHL patients. MATERIALS AND METHODS: Retrospective analysis was conducted on the data of 67 lymphoma patients older than 18 years who had HD-ASCT with the BuCyE conditioning regimen between September 2014 and November 2021 (86 months). The research consisted of 34 r/r HL patients among them. A parenteral regimen of 0.8 mg/kg of busulfan every 6 h from day −7 to day −5, 50 mg/kg of cyclophosphamide on days −3 and −2, and 400 mg/m2 of etoposide on days −5 and −4 comprised the patient preparation regimen before ASCT. All data were collected from inpatient files and the Inonu University Turgut Ozal Medical Center Hospital Information System. RESULTS: The median age of the patients was 43 years, and 67.6% were males. The most common type of HL was nodular sclerosis, which was followed by mixed cellularity. The median time for platelet and neutrophil engraftment was 14 and 11 days, respectively. 5.0 × 106/kg was the median transplanted dose of CD34+ cells (2.1–13.55). Liver toxicity was observed in 6 (17.6%) patients. Eight patients suffered from pulmonary side effects. The median number of previous chemotherapies was 2 (2–4). In all lymphoma patients, the complete response rate was 61.8% (n = 21), whereas the disease progression rate was 32.3% (n = 11). Transplantation-related mortality on the 100th day was 8.8% (n = 3). Three-year overall survival was 57.17%. CONCLUSION: When the literature was reviewed, the studies with the BuCyE preparation regimen in patients with r/rHL were limited. This conditioning regimen was found to have fewer side effects and a lower cost. It can be preferable when compared to carmustine (BCNU), etoposide, cytarabine (ARA-C), and melphalan (known as BEAM) in r/rHL.
This multicenter retrospective study evaluated the efficacy of allogeneic hematopoietic stem cell transplantation (allo-HSCT) on survival and safety in patients with relapsed/refractory (R/R) Hodgkin lymphoma (HL) and non-Hodgkin lymphoma (NHL). A total of 110 patients with R/R HL or NHL who underwent allo-HSCT between July 2007 and October 2022 at 7 adult stem cell transplant centers were evaluated. Progression-free survival (PFS), graft versus host disease-free survival (GRFS), and overall survival (OS) were the primary endpoints, and NRM was the secondary endpoint. Forty-one (37.3
Introduction: Although the prognosis of chronic myeloid leukemia (CML) changed dramatically with the introduction of imatinib (IM), some patients (pts) still need further BCR::ABL1 tyrosine kinase inhibitor (TKI) therapies due to resistance and/or intolerance to IM. 2nd-generation TKIs (2GTKIs) including bosutinib (BOS) can be utilized in those pts. In Turkey, IM is the only BCR::ABL1 TKI reimbursed in newly diagnosed pts, and BOS can be utilized in the 2nd and later lines of therapy. Real-life data of BOS in pts with CML failing previous lines of TKI therapy is still limited in the literature and the aim of this multicenter study is to evaluate the efficacy and safety of BOS therapy in pts with CML beyond first-line TKI therapy in the real-life setting. Methods: All information on demographics, previous treatments, TKI responses and toxicities, and follow-up data were gathered from the files of the pts retrospectively. Early molecular response (EMR) was defined as a BCR::ABL1IS transcript level <10% at 3 months. Major molecular response (MMR) and deep molecular response (DMR) were defined as BCR::ABL1IS transcript levels ≤0.1% and ≤0.01% (MR4.0) or deeper, respectively. between BCR::ABL1IS transcript levels between 0.1-1% were considered as complete cytogenetic response (CCyR). Results: Two hundred eighty-three pts from 40 centers were included. The median age was 58 years (range, 20-86 years), and 58% of the pts (n=167) were male. At diagnosis, 260 pts were in chronic phase (CML-CP) (91.3%), and 15 (5.3%) and 8 (2.8%) pts were in accelerated and blastic phases, respectively. Prior to BOS therapy, the median follow-up was 45.5 months (range, 0-288 months) and 5 pts (1.8%) received an allograft. The median number of prior TKIs was 2 (range, 0-4), and BOS was used as 1st-, 2nd-, 3rd-, and >3rd-lines of therapy in 2 (0.7%), 74 (26.1%), 98 (34.6%), and 109 (38.6%) pts, respectively. Of the CML-CP pts, 123 (47.3%) were switched to BOS due to resistance and 108 (40.4%) due to intolerance to prior TKI therapy and in 29 (11.2%), both due to intolerance and resistance. One hundred and eighty-three pts (64.6%) had at least one comorbidity, and the most common comorbidities were hypertension (39.9%), cardiovascular diseases (29%), and diabetes (23.3%). The initial daily dose was 500 mg in 194 pts (68.6%), 400 mg in 50 pts (17.7%), 300 mg in 28 pts (9.9%), 200 mg in 7 pts (2.5%), and 100 mg in 4 pts (1.4%). The mean BOS dose intensity was 449.47 mg/day (range, 100-500 mg/day). With a median duration of 17 months of BOS therapy (range, 3-178 months), 75.6% of 281 pts (n=214) experienced at least one AE, and the most common non-hematological adverse event (AE) was diarrhea, observed in 144 pts (43.8%). Of these, 130 (90.3%) and 14 (9.7%) pts experienced grade 1-2 and grade 3-4 diarrhea, respectively. The percentages of grade 3-4 anemia, neutropenia, and thrombocytopenia were 7.1% (20/282), 3.2% (9/282), and 3.2% (9/282), respectively. Dose reduction was required in 75 pts (26.5%) due to any AEs. In 58 pts (20.5%), BOS was interrupted and 92 pts (32.5%) discontinued BOS therapy permanently. Main reasons for permanent discontinuation were AEs (47.8%) and loss of response and/or progression (39.1%). The incidences of any AEs were comparable across treatment lines; 70.3% (52/74) in 2nd-line, 78.6% (77/98) in 3rd-line, and 77.1% (84/109) in >3rd-line (p=0.420). At time of BOS start, rates of any response less than CCyR, CCyR, MMR, and DMR were 53.7%, 11.7%, 15.9%, and 17.7%, respectively. Under BOS therapy, CCyR, MMR, and DMR rates were 4.6%, 23%, and 43.1%, respectively and 63 pts (22.3%) achieved a response level less than CCyR. The percentage of pts with optimal responses were significantly higher with BOS when compared to those achieved prior to BOS therapy (p<0.001). EMR rates were significantly higher in pts receiving 500 mg/day BOS than those with a daily dose <500 mg (34.2% vs. 64.8%, p=0.023). Conclusion: Our multicenter, nationwide study among pts with CML in the real world setting demonstrated that BOS is an effective 2GTKI in pts who failed at least one TKI therapy with a generally manageable toxicity profile.
Background and Objectives: Acute myeloid leukemia and myelodysplastic syndrome are both clonal hematologic malignancies that primarily affect older adults. Current treatments for AML/MDS are both limited in number and efficacy. This study aims to evaluate venetoclax-based therapies in AML/MDS, focusing on overall survival and recurrence-free survival rates, and to expand real-world data on its use. Materials and Methods: Clinical and laboratory data on patients with AML/MDS aged 18≥ treated with venetoclax between January 2019 and July 2022 were included. Survival analysis was calculated based on the period from 2019 to December 2023. Results: A total of 161 AML and 40 patients with MDS were included. The median age was 63.53 ± 15.30 years for AML and 70.12 ± 10.21 years for MDS. In both groups, over 55% are male. A total of 77.6% of patients with AML and 75% of patients with MDS received treatment prior to venetoclax. Venetoclax was administered in combination with azacitidine to 84.5% of AML and 67.5% of MDS. The relapse rate in AML is approximately 15%. Overall, the 2-year survival rate is 46% and 18.73 months. The overall CR/CRi rate for patients with AML is 49.1%, while for patients with MDS, it is 50%. The 2-year survival rate for patients with MDS is 52.7%. The 2-year RFS rate was 75.5% for AML and 90.9% for MDS. The relapse rate in AML is approximately 15%. The percentage of adverse events leading to treatment discontinuation among those with grade 3–4 toxicity is low; 26.7% for AML (n = 43) and 15% for MDS (n = 6). Conclusions: Our real-world data demonstrate that venetoclax has the potential to improve overall survival rates when used in combination with HMAs and supports its use in patients with AML/MDS.