High throughput screening aids clinical decision-making in refractory acute myeloid leukaemia.

BACKGROUND: Despite advances in therapeutics for adverse-risk acute myeloid leukaemia (AML), overall survival remains poor, especially in refractory disease. Comprehensive tumour profiling and pre-clinical drug testing can identify effective personalised therapies. CASE: We describe a case of ETV6-MECOM fusion-positive refractory AML, where molecular analysis and in vitro high throughput drug screening identified a tolerable, novel targeted therapy and provided rationale for avoiding what could have been a toxic treatment regimen. Ruxolitinib combined with hydroxyurea led to disease control and enhanced quality-of-life in a patient unsuitable for intensified chemotherapy or allogeneic stem cell transplantation. CONCLUSION: This case report demonstrates the feasibility and role of combination pre-clinical high throughput screening to aid decision making in high-risk leukaemia. It also demonstrates the role a JAK1/2 inhibitor can have in the palliative setting in select patients with AML.

Major flares in women with SLE on combined oral contraception.

Two recent trials concluded that the use of oral contraceptives (OC) did not induce flares in lupus patients. We record our experience with OC in patients with stable lupus. Eight patients were enrolled in an open trial. Six received a combined contraceptive pill and two were allocated to the control arm. During a 12 month follow-up, 3 patients in the active arm experienced 4 major flares. One patient died as a result of uncontrolled disease complicated by sepsis. At this point, we abandoned the trial. The 2 patients in the control arm experienced no disease exacerbation during the 7 months of observation. We would urge that patients who are placed on OC be closely monitored.

Cutaneous leucocytoclastic vasculitis: a clinical and aetiological study.

Cutaneous vasculitis may be the presenting sign in many different clinical disorders. The aetiology and clinical and laboratory findings were recorded in 69 consecutive patients with cutaneous leucocytoclastic vasculitis. Underlying connective tissue disease or malignancy was found in 19 patients. Evidence of recent streptococcal infection was detected in 24 patients. Application of the classification criteria proposed by the American College of Rheumatology (ACR) identified 35 patients with Henoch-Schonlein purpura (HSP) and 42 with hypersensitivity vasculitis (HSV). Thirty-three subjects fulfilled criteria for both HSP and HSV.

Loss of trabecular bone mineral density in systemic lupus erythematosus.

OBJECTIVE: To evaluate trabecular bone mineral density (BMD) in young ambulatory female patients with systemic lupus erythematosus (SLE). METHODS: Bone mineral density (gm/cm2) at the lumbar vertebrae (L1-L4) and at the left femur (neck, trochanter, intertrochanter, and Ward's triangle) was measured by dual x-ray absorptiometry in 46 SLE patients (mean age 31 years, mean disease duration 76 months) and in 108 healthy female controls (mean age 32 years). Twenty-two of the SLE patients were receiving corticosteroids (CS) at the time of the study. RESULTS: Lumbar BMD in the SLE patients was less severely reduced than was BMD at the femoral sites, but the SLE group was closer to the lumbar fracture threshold of 0.812 gm/cm2 than was the control group (P = 0.0009). There were no significant differences between the SLE patients currently being treated with corticosteroids and those who were not (P > 0.3). BMD at Ward's triangle and at the femoral neck was not significantly reduced in the SLE patients. Total femoral BMD had a sensitivity of 76% and specificity of 62% in differentiating the SLE group from the controls. The positive predictive value was 61% and the negative predictive value was 89%. The prevalence of osteopenia in the SLE patients was 25%. CONCLUSION: SLE causes significant trabecular bone loss, which is not due to corticosteroid therapy.