Yescarta: A New Era for Non-Hodgkin Lymphoma Patients.

The use of conventional therapeutic approaches in patients with lymphoma demonstrates significant drug resistance leading to poor prognosis with reduced median survival period. T-cell immunotherapy has diverted huge attention of the researchers in recent times to engage in the stated research studies in the pool of chemotherapy-refractory lymphoma patients. B-cell antigen CD19-targeted chimeric antigen receptor (CAR) T-cell products are approved for the treatment of non-Hodgkin B-cell refracting or relapsing lymphoma. The aim of this article is to give an idea about the use of FDA-approved anti-cancer gene therapy, Axicabtagene ciloleucel, marketed under the name of Yescarta®. Axicabtagene ciloleucel is developed from the patients' mononuclear peripheral blood cells during which T cells are orchestrated to articulate a CAR that diverts them to identify CD19-expressing cells. It is used in patients with non-Hodgkin B-cell refracting or relapsing lymphoma who had no response to prior therapeutic regiment involving the use of chemotherapeutics. Here, we review the mode of action, safety, and efficacy of Yescarta.

Voxelotor: A Ray of Hope for Sickle Disease.

Sickle cell disease is one challenging blood disorder, affecting around 100,000 people in the United States alone. None of the currently approved drugs can modify the underlying pathology of the disease. Voxelotor, first of its kind, is an orally administered drug that can alter the underlying disease pathology (by increasing the affinity between Hb and oxygen) and inhibit sickling of red blood cells. Several clinical trials and case series have documented the benefits and safety of voxelotor therapy in sickle cell disease. Currently, the US FDA has approved the drug for treatment of sickle cell disease and also granted the status of orphan drug.

Mini review: leg ulcers - a secondary complication of sickle cell disease.

Sickle cell disease (SCD) is a group of inherited blood disorders recognized by WHO as a major public health problem. It affects morbidity and mortality of the affected population considerably. Leg ulcer in the lower limbs is a hallmark feature of SCD. Meticulous physical examination, thorough history, laboratory tests, and imaging will lead to proper diagnosis and lead to proper treatment and management of the cases. Although newer treatment strategies have improved the prognostic outcome of SCD, leg ulcers still are a disabling and difficult to treat a complication of the condition. This mini review summarizes this common complication of SCD.

Acromegaly: a challenging condition to diagnose.

Acromegaly is an uncommon, chronic disease, characterized by hypersecretion of a pituitary growth hormone by somatotroph adenomas, along with increased levels of insulin-like growth factor-1. Although acromegaly presents a wide array of clinical manifestations, the salient symptoms include acral and soft tissue enlargement, joint pain, heart and respiratory failure, diabetes mellitus, and hypertension, leading to increased morbidity and mortality. Hence, early diagnosis of the disease is critical to enhance life expectancy and quality of life. New approaches are being developed for diagnosis and surveillance (both screening and follow-up), including sensitive biochemical assays and the use of MRI to visualize extremely small tumors, and are helpful in the early diagnosis of acromegaly, subsequent treatment, and disease control. This mini-review summarizes the most common and effective tools used in the diagnosis of acromegaly.

Prevalence of Anemia in Type 2 Diabetic Patients.

BACKGROUND: The aim of the study was to determine the prevalence of anemia in patients with type 2 diabetes and to assess the risk of anemia according to gender, age and glycemic control. METHODS: The study group comprised of patients with type 2 diabetes attending Outpatient Diabetic Department of Amiri Hospital (Al-Asimah Capital area) from January 1, 2016 to December 31, 2017. Patients were divided into groups according to glycemic status and gender. Glycated hemoglobin (HbA1C) values and hemoglobin (Hb) levels were evaluated. The presence of anemia was defined by an Hb level < 13.0 g/dL for men and < 12.0 g/dL for women. RESULTS: The prevalence of anemia is significantly greater in diabetic females (38.5%) than in diabetic males (21.6%) and in poorly controlled diabetics (33.46%) than those with glycemic status under control (27.9%) (P < 0.05). The average age of patients with anemia was found to be 60.69 ± 0.198 years and the average age of patients without anemia was found to be 54.07 ± 0.121 years. This indicates that the risk of anemia increases with age. CONCLUSION: Screening for anemia at the time of diagnosis of diabetes, diabetic medication compliance, awareness of the risk of anemia and other complications in the diabetic patients helps in reducing the prevalence of anemia in diabetic population.

Ofatumumab - a valid treatment option for chronic lymphocytic leukemia patients.

Ofatumumab Arzerra® is a human monoclonal antibody, which induces killing of a panel of tumor B-cell lines and primary tumor cells by the activation of in vitro complement-dependent cytotoxicity and antibody-dependent, cell-mediated cytotoxicity. The humanized anti-CD20 monoclonal antibody has been approved by the US Food and Drug Administration for the treatment of chronic lymphocytic leukemia patients. This article summarizes this antibody's therapeutic effect on chronic lymphocytic leukemia.

Long Standing Eculizumab Treatment without Anticoagulant Therapy in High-Risk Thrombogenic Paroxysmal Nocturnal Hemoglobinuria.

Paroxysmal nocturnal hemoglobinuria (PNH) is an ultra-orphan disease affecting all hematopoietic cell types. The abnormality of red blood cells in this disease predisposes to intravascular complement-mediated hemolysis. Eculizumab is an orphan drug used to treat this rare disease. Thrombosis is the key cause of death in PNH patients in about 40% to 67% of cases. We report the case of a woman presenting with PNH complicated with serious Budd-Chiari syndrome thrombosis and with a stent inserted in the portal vein. She refused to take any anticoagulant treatment since she commenced eculizumab 4 years before. No thrombotic events happened since that time. This case could add an extra benefit for eculizumab, which could be used as an anti-thromboembolic prophylactic agent in PNH, especially in patients with thrombocytopenia, where the use of anticoagulant agents is extremely hazardous. More randomized studies might establish the use of eculizumab without anticoagulants to avoid serious bleeding that could happen in thrombocytopenic PNH patients.

Successful nilotinib treatment in a child with chronic myeloid leukemia.

A 16-year-old female was diagnosed incedentally with chronic myeloid leukemia (CML) in the chronic phase. She showed complete remission after 3 months of nilotinib treatment. CML is a rare malignant neoplasm in pediatric age. It is characterized by a Philadelphia chromosome, which comes from a genetic translocation between chromosomes 9 and 22. This translocation results in an abnormal fusion called BCR-ABL oncogene which encodes a chimeric BCR-ABL protein. This protein is the underlying cause of CML. Nilotinib is a newly licensed drug for CML in adults. Structurally, it is similar to imatinib (the older tyrosine kinase inhibitor), but it is much more potent in inhibiting BCR-ABL due to its much increased affinity for its binding site. Specific guidelines for CML treatment in children have yet to be determined. In our patient, nilotinib was used as an off-label drug because it is not licensed for children. According to the pharmacokinetic response to drugs, children cannot be considered small adults irrespective of their weight. Off-label drug use based on evidence that it is the best treatment available is an important tool in the hands of expert treating physicians.