Oral Ezatiostat HCl (TLK199) and Myelodysplastic syndrome: A case report of sustained hematologic response following an abbreviated exposure

Treatment options for patients with lower risk non-del(5q) myelodysplastic syndromes (MDS) who fail erythroid stimulating agents are restricted to one of the hypomethylating drugs with an expected response rate of ~50%. Ezatiostat HCl, an agent with the potential for producing multi-lineage responses in this population is currently in clinical investigation phase. This case report describes a 77 year old male who received less than two cycles of therapy with ezatiostat HCl which had to be aborted due to intolerable side effects, but which produced a sustained normalization of all three blood counts. This trilineage response has now lasted for more than a year. Interestingly, the patient began with a del(5q) abnormality and responded briefly to lenalidomide. Upon relapse of the anemia, a bone marrow showed the disappearance of the del(5q) but the appearance of a new clonal abnormality t(2;3). Given that the patient had a complete cytogenetic response to a truncated exposure to lenalidomide followed by a trilineage response to an even briefer course of ezatiostat HCl suggests a potential role for ezatiostat HCl in del(5q) patients who relapse following lenalidomide

GERM CELL TUMOURS OF THE OVARY IN CHILDREN AND ADOLESCENTS: A CLINICAL STUDY OF 109 PATIENTS IN A SPECIALIZED CANCER CENTRE

Objective: Paediatric ovarian germ cell tumours (GCTs) are rare tumours withmalignant tumours extremely rare.Methods: All the paediatric patients who received treatment for histology proven ovarian GCT at Shaukat Khanum Memorial Cancer Hospital from January 2006 to December 2014 were retrospectively reviewed. Patients over the age of 18 years were excluded from the study. A total of 109 patients were included in the study. A set of parameters were identified to record initial clinical presentation and examination, imaging and laboratory investigations including tumour marker levels. Decisions of multidisciplinary team meetings, surgical treatment, neo adjuvant, adjuvant chemotherapy and radiation data retrieved. Data analysiscarried out using SPSS 20.Results: In total 109 girls presented to our hospital during the study period, most of them above the age of 5 years, with dysgerminoma being the most common followed by yolk sac tumour. Most of the patients received treatment outside our hospital and were referred here for chemoradiotherapy. Fertility preserving surgery was the most commonly performed surgical procedure with a mean follow-up of 50.4 months and >75% overall 5-year survival.Conclusion: Regardless of histologic types, the outcomes of GCT can be improved with a multidisciplinary approach.Key words: Dysgerminomas, germ cell tumours, ovarian tumour

Isolated hemolytic anemia: an unusual manifestation of occult malignancy

Hemolysis is an uncommon and usually late complication of malignancy, and very rarely the presenting feature. Cancer-associated hemolysis may be immune-mediated, or may result from thrombotic microangiopathy accompanied by thrombocytopenia. We describe an unusual case of isolated hemolysis in the setting of occult metastatic breast cancer. The patient initially presented with symptomatic anemia, with evidence of hemolysis but with negative direct antiglobulin testing and a normal platelet count. Subsequent investigation discovered metastatic adenocarcinoma of the breast involving bone marrow. Hemolysis worsened despite initial treatment with cytotoxic chemotherapy and a trial of corticosteroids, but later resolved with aromatase inhibitor therapy