Single agent vemurafenib or rituximab-vemurafenib combination for the treatment of relapsed/refractory hairy cell leukemia, a multicenter experience.

BACKGROUND: Hairy cell leukemia (HCL) is a rare mature B-cell malignancy that is primarily treated with purine analogues. However, relapse remains a significant challenge, prompting the search for alternative therapies. The BRAF V600E mutation prevalent in HCL patients provides a target for treatment with vemurafenib. PATIENTS AND METHODS: This multicenter retrospective study included nine patients with relapsed/refractory (R/R) HCL from six different centers. Patient data included demographics, prior treatments, clinical outcomes, and adverse events. RESULTS: Patients received different treatment regimens between centers, including vemurafenib alone or in combination with rituximab. Despite the differences in protocols, all patients achieved at least a partial response, with seven patients achieving a complete response. Adverse events were generally mild with manageable side effects. The absence of myelotoxic effects and manageable side effects make BRAF inhibitors attractive, especially for patients ineligible for purine analogues or those with severe neutropenia. CONCLUSION: Single agent vemurafenib or in combination with rituximab appears to be a promising therapeutic option for R/R HCL. Further research is needed to establish standardized treatment protocols and to investigate long-term outcomes.

Eltrombopag treatment in thrombocytopenia following hematopoietic stem cell transplantation: A multicenter real-world experience.

INTRODUCTION: Thrombocytopenia is among the most common complications following hematopoietic stem cell transplantation and is associated with increased mortality and morbidity with no standard treatment yet. In this multicenter and retrospective study, we aim to present our multi-center experience of Eltrombopag treatment in patients with isolated thrombocytopenia following HSCT. MATERIAL-METHOD: A total of 73 patients from 5 centers who underwent autologous or allogeneic stem cell transplantation, had no primary disease relapse, all of whom had neutrophil engraftment, complete chimerism, and who were diagnosed with Prolonged Isolated Thrombocytopenia (PIT) or Secondary Failure Of Platelet Recovery (SFPR) were included in the study. The patients were initiated on Eltrombopag at a dose of 50-150 mg. Complete response was defined as a platelet count >50×109/L for 7 consecutive days with no transfusion support. RESULTS: A total of 50.3% of the patients underwent Autologous and 49.7% Allogeneic Stem Cell Transplantation, 54.8% were diagnosed with PIT, and 45.2% were diagnosed with SFPR, and the treatment with 50-150 mg/day Eltrombopag was initiated on the median day +42. Complete response was achieved in 71.2% of these patients on the median day 23 of the treatment. No significant effects of the initial dose (50-150 mg/day) were detected in the Complete Response in the multivariate analysis on response. An insufficient number of Megakaryocytes in the bone marrow before Eltrombopag treatment was determined as an independent risk factor in determining the response (OR 3.57, 95% CI 1.21-10.55). The overall survival of the patients who did not respond to Eltrombopag was found to be significantly worse than that of patients who responded (p=0.022, HR:2.74, 95% CI 1.12-6.54). CONCLUSION: As a result of the present study, Eltrombopag treatment was found to be effective and safe in thrombocytopenia that develops following hematopoietic stem cell transplantation. It was concluded that its use may be more effective in patients with sufficient bone marrow megakaryocytes before the treatment and an initial dose of 50 mg/day may be appropriate in terms of cost, effectiveness, and toxicity. Large-scale randomized and controlled prospective studies are needed to determine the roles of Eltrombopag treatment in patients with post-transplant PIT and SFPR.

Relapsed refractory multiple myeloma with CNS involvement successfully treated with Elranatamab: first reported case.

Central nervous system (CNS) involvement in multiple myeloma (MM) is a rare and challenging complication associated with poor prognosis and limited treatment options. Emerging T-cell directing therapies, such as bispecific antibodies (bsAbs) and chimeric antigen receptor T cells (CAR-T), have shown remarkable success in treating MM, but their efficacy in CNS involvement remains unclear. Elranatamab, a humanized bispecific antibody targeting B-cell maturation antigen (BCMA) and CD3-expressing T cells, has demonstrated promising results in relapsed refractory MM. However, its efficacy in treating CNS-MM has not been reported. We present a case of a 37-year-old male MM patient with CNS involvement who has been successfully treated with Elranatamab.

Newly onset cytopenias not always indicate a relapsing AML after allogeneic HSCT, a case of non-destructive post transplant lymphoproliferative disorder.

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains an effective option for the treatment of intermediate and high-risk Acute myeloid leukemia (AML). Post-transplant lymphoproliferative disorder (PTLD) is related to the intensity of post-transplant immunosuppression. Although Epstein-Barr virus (EBV) seropositivity and reactivation can be a major risk factor for PTLD. A few PTLDs could be EBV negative. There are a very limited number of PTLD cases following HSCT in patients with AML. We present a differential diagnosis of cytopenias after allo-HSCT. This is the first report of an AML patient developing bone marrow EBV-negative PTLD relatively late in their post-transplant course.

Urinary kidney injury molecule-1 levels as a marker of early kidney injury in hypertensive patients.

BACKGROUND: Kidney injury molecule-1 (KIM-1) is a type 1 tubular cell transmembrane protein that is found in high levels in early stages of acute kidney injury and is stated to have predictive value in the early diagnosis of chronic kidney diseases. In this study, the hypothesis was that higher levels of KIM-1 would be detected in hypertensive patients for the early detection of nephropathy. With this goal, urinary KIM-1 levels of hypertensive cases were compared with those of healthy controls, and associations of KIM-1 levels with microalbuminuria and glomerular filtration rate (GFR) were investigated. METHODS: The study included a total of 80 patients aged ≥20 years (55 male, 25 female, mean age: 57.21±9.12 years). The patient group consisted of 40 patients (28 males, 12 females, mean age: 57.58±8.79 years) who had had hypertension for at least 5 years, and the control group consisted of 40 healthy subjects (27 female, 13 male, mean age: 56.85±9.53 years). Groups were compared based on demographic, anthropometric and biochemical data, and urinary KIM-1 levels. Correlation analysis was made to assess the association of KIM-1 levels with microalbuminuria and GFR. Levels of urinary KIM-1 enzyme were measured using linked immunosorbent assay (ELISA). RESULTS: KIM-1 levels were found to be 0.86±0.48 ng/mg creatinine in the patient, and 0.71±0.46 pg/mL in the control groups (P>0.05). A positive correlation was detected between KIM-1 levels and both systolic blood pressure and duration of disease (r=0.308, P=0.032 and r=0.339, P=0.032, respectively). CONCLUSIONS: While not supporting the hypothesis that KIM-1 levels may increase in hypertensive patients as an early indicator of hypertensive nephropathy, these findings suggested that this molecule might be associated with kidney injury in hypertensive nephropathy due to its positive correlation with the duration of hypertension.