Hook, Christopher C.

AbstractThe current study was approached with the assumption that response to induction chemotherapy, in acute myeloid leukemia (AML), overshadows pre‐treatment risk variables in predicting survival and therefore be used as an anchor for a simplified risk model. We considered 759 intensively‐treated patients with AML, not promyelocytic: median age 60 years; primary 66%, secondary 25%, and therapy‐related 9%; European LeukemiaNet cytogenetic risk category favorable 8%, intermediate 61%, and adverse 31%. Complete remission with (CR) or without (CRi) count recovery was achieved in 608 (80%) patients. After a median follow‐up of 22 months, 503 deaths, 272 relapses, and 257 allogeneic hematopoietic stem cell transplants (AHSCTs) were recorded. Multivariable analysis identified failure to achieve CR/CRi (HR 3.8, 95% CI 3.1–4.8), adverse karyotype (2.2, 1.8–2.8), and age >55 years (2.1, 1.6–2.7) as main risk factors for survival. HR‐weighted scoring resulted in four‐tiered risk stratification: low (0 points; N = 183), intermediate‐1 (1 point; N = 331), intermediate‐2 (2 points; N = 117), and high (≥3 points; N = 128), with respective median survival (5‐year rate) not reached (68%), 34 (37%), 13 (20%), and 5 (5%) months (p < .001). FLT3‐ITD mutation was associated with inferior survival in intermediate‐1 (p = .004) and TP53 in intermediate‐2 (p = .06) and high (p = .02) risk disease; the latter was fully accounted for by the close association between TP53 mutation and complex/monosomal karyotype while the observations regarding FLT3‐ITD were not affected by treatment with midostaurin. AHSCT had a favorable impact on survival, most apparent in intermediate‐1 (p < .001), intermediate‐2 (p = .03), and high (p = .01) risk disease. The proposed 3‐factor survival model offers a novel prototype that is amenable to further enhancement by molecular information and was validated in an external cohort of 1032 intensively‐treated AML patients.

SummaryInstitutional database search (1999–2020) for acute myeloid leukaemia (AML) identified 109 cases of myeloid sarcoma (MS), of which 19 were isolated and presented de novo. The latter displayed longer survival (median 78 months), compared to MS with synchronous intramedullary AML (n = 32; median 16 months) and de novo AML without MS (n = 729; median 22 months; P = 0·13). However, the difference in survival was no longer apparent after accounting for bone marrow cytogenetic risk status (P = 0·67). Treatment‐induced MS tumour resolution was not affected by the presence of intramedullary disease (P = 0·61). The current study clarifies the prognosis of de novo isolated MS, in the context of AML.

Abstract Background: Percutaneous nephrolithotomy (PCNL) for large stone burden can be problematic in patients with significant risk of bleeding complications, specifically thrombocytopenia. This report demonstrates effective correction of two patients' thrombocytopenia, subsequently leading to removal of large stone burden through PCNL. Case Presentation: We present two Middle Eastern patients who presented with medical histories significant for thrombocytopenia, secondary to splenomegaly and hepatic vein thrombosis, and large volume nephrolithiasis. Patient 1 is a 65-year-old female with a right 5 cm stone and a platelet count of 34,000. Patient 2 is a 45-year-old female with a 3 cm left staghorn stone and a platelet count of 44,000. After consultation with hematology, both underwent therapy with prednisone and intravenous immunoglobulin without improvement in their platelet count. They then received 3 μg/kg/dose of romiplostim weekly that improved their platelet counts to 133,000 and 195,000 in 2 weeks, respectively. Patient 1's PCNL was completed in a single-stage procedure with stone-free status shown on CT postoperative day 1. Patient 2 underwent PCNL and a secondary ureteroscopy for residual stone fragments on postoperative day 2. Both patients experienced no complication during the procedure, hospital stay, or postoperative course. Both continued romiplostim for 20 days postoperatively with platelet levels returning to their baseline range after 1 month. Conclusion: In the appropriately selected patient, romiplostim can correct thrombocytopenia enough to safely and effectively perform PCNL in patients with underlying hematologic disorders. Close coordination between urology and hematology is imperative to ensure an effective outcome in this challenging patient population.