A case report of a very late response to 5-azacytidine in a patient with lower risk myelodysplastic syndrome: Time to update treatment guidelines for lower risk patients.

RATIONALE: The hypomethylating agent 5-azacytidine has been approved in Europe for patients with intermediate 2 and high (i.e., higher) risk myelodysplastic syndrome according to the International Prognostic Scoring System (IPSS). A total of 91% of all first responses in higher risk patients occur within 6 cycles of treatment; however, data regarding the time to first response in clinical trials with lower risk patients are not available. PATIENT CONCERNS: Our case describes the late response of a lower risk (intermediate 1 according to the IPSS and intermediate according to the IPSS-R) patient to 5-azacytidine treatment.Diagnosis and interventions: Once diagnosed, the patient started supportive treatment due to persistent pancytopenia and recurrent infections. The use of a hypomethylating agent was decided because the patient was transfusion dependent, and suffering from recurrent severe febrile infections due to neutropenia. Other possible causes of fever except infections in the context of his neutropenia were excluded. OUTCOMES: After the 12th cycle of 5-azacytidine the patient showed a hematologic response, with transfusion independency and with no recurrent febrile episodes. LESSONS: This case report probably indicates that a subset of patients who belong to the lower risk category according to the previous prognostic systems and to the intermediate one according to the IPSS-R, may benefit from prolonged treatment with the drug. The indication of 5-azacytidine in Europe for patients with higher risk myelodysplastic syndrome (MDS) (according to the IPSS) could possibly include a wider range of patients if updated according to the IPSS-R.

Isolated skeletal muscle recurrence of an originally nodal diffuse large B cell lymphoma: A case report and review of the literature.

RATIONALE: Diffuse large B cell lymphoma (DLBCL) is a malignancy of the B cells with extranodal primary involvement being estimated at 30% to 40% of cases. Primary skeletal muscle presentation of DLBCL is extremely rare, with an estimated incidence of about 0.5% of extranodal lymphomas, presenting mostly in the lower extremities. The possible mechanisms of muscle involvement of DLBCL include primary extranodal disease, extension from adjacent organs (such as lymph nodes) or disseminated disease. PATIENT CONCERNS: We report a case of a 70-year-old woman with an advanced initially nodal DLBCL, treated with R-CHOP, that presented with an enlargement of her left thigh and restricted mobility 3 months after completion of chemotherapy. Imaging studies were performed, which showed possible infiltration of the muscles of the left thigh, without any nodal disease present. DIAGNOSES: Muscle biopsy documented the recurrence of the lymphoma at the left thigh. INTERVENTIONS: The patient started second-line treatment with gemcitabine and vinorelbine. OUTCOMES: A partial response was achieved after the first cycle. LESSONS: The remarkable element lies in the reappearance of the lymphoma at the left thigh muscles, with no radiographic or clinical evidence of involvement of lymph nodes, despite the extensive lymph node disease at initial presentation. The further management of such recurrences remains to be clarified, as the odd biological behavior of the malignant cells dictates a special handling of the disease.

5-Azacytidine in the Treatment of Intermediate-2 and High-risk Myelodysplastic Syndromes and Acute Myeloid Leukemia. A Five-year Experience with 44 Consecutive Patients.

BACKGROUND/AIM: The hypomethylating agent 5-azacytidine has been the standard-of-care for patients with higher-risk myelodysplastic syndrome (MDS) during the past few years. Its efficacy has been proven in large clinical trials, and its safety has been shown to be superior to that of conventional treatments. PATIENTS AND METHODS: We conducted a retrospective study on the efficacy and safety of 5-azacytidine in 44 consecutive patients with MDS and acute myeloid leukemia treated with 5-azacytidine during a 63-month period. We recorded the clinical and laboratory characteristics of the patients and we analyzed the response to treatment, overall survival and adverse events during treatment. RESULTS: The median overall survival was 13 months, while serious adverse events consisted mostly of neutropenic infections. CONCLUSION: We reached two possibly valuable conclusions: Younger patients (<73 years), as well as patients receiving treatment at longer than 28-day intervals had a significantly higher overall survival.