Is there a role for low-dose eltrombopag as maintenance therapy in the treatment of immune thrombocytopenia?

BACKGROUND/AIM: Thrombopoietin receptor agonists (romiplostim and eltrombopag) have recently been licensed for the treatment of thrombocytopenia in patients with chronic immune thrombocytopenia (ITP) with an insufficient response to corticosteroids, immunoglobulins or splenectomy. In the present case series, we present 4 nonresponding patients with chronic ITP who achieved maintenance of complete response (CR) for a period of at least 6 months on eltrombopag treatment administered in a modified regimen of 25 mg for 2, 3 or 5 days a week. METHODS: The present study is a retrospective, nonconsecutive case series of 4 eltrombopag-treated patients with chronic ITP. Secondary ITP had been excluded in each patient, first-line therapy had failed and splenectomy had been refused. Furthermore, each patient was treated with eltrombopag, which resulted in a CR for a mean of 2 months. Consequently, decreased eltrombopag dosages have been able to maintain long-term CR. RESULTS/CONCLUSION: Despite the low quality of evidence, our study results support the use of reduced-dose eltrombopag as a maintenance therapy after achieving CR. It seems a very promising strategy for the effective maintenance of response, improving health-related quality of life, lowering costs and possibly improving the safety in the treatment of ITP.

Initially lymphocytic Sweet's syndrome in male patients with myelodysplasia: a distinguished clinicopathological entity? Case report and systematic review of the literature.

BACKGROUND: Sweet's syndrome (SS) is an acute febrile neutrophilic dermatosis. It can occur as an idiopathic, drug-induced or malignancy-associated entity. SS is also seen in patients with myelodysplastic syndrome (MDS) where it may present atypically, both clinically and histologically. In a few rare cases of MDS, lymphocytic infiltrates are the presenting feature of SS. METHODS: MEDLINE and Scopus were the data sources for our review. RESULTS: A clinicopathological subsetemerged of 12 male SS patients with MDS and a mean age of 67.3 years in which the initial SS lesions were lymphocytic infiltrates. However, from 0.5 to 8 years later, sequential biopsies revealed neutrophilic dermal infiltration typical of SS. CONCLUSION: Initially lymphocytic infiltrates in this subset could be attributed either to an early timing of the biopsy concerning the age of the lesion or to the dysgranulopoiesis syndrome. A possible relationship between the dysfunction of the receptor of the granulocyte-macrophage colony stimulating factor, the gene of which is located on the pseudoautosomal X-Y region, may exist in MDS patients with initially lymphocytic SS. This could explain the male gender of this subset and might establish initially lymphocytic SS as a distinguished clinicopathological entity for predicting the occurrence and even the prognosis of MDS.

Evaluation of Effects of Diabetes Mellitus, Hypercholesterolemia and Hypertension on Bell's Palsy.

The aim of this study is to evaluate the effects of diabetes mellitus, hypertension and hypercholesterolemia on the clinical presentation and outcome of Bell's palsy. The study (comorbidity) group consisted of 50 patients with Bell's palsy associated with diabetes, hypertension, or hypercholesterolemia; the control group included 46 patients with Bell's palsy, but without comorbid diseases. The House-Brackmann grading system (I to VI) was used in order to assess the initial and final facial functions. Both groups of patients were treated with steroids and the antiviral agent acyclovir. The mean severity of initial facial paralysis was more significant in diabetes, hypercholesterolemia, and hypertension, in comparison to the control group. Patients suffering from Bell's palsy and concomitant comorbidities have a poorer prognosis (HB III-VI) compared to patients without comorbidities. Increased glycosylated hemoglobin A1c levels (>6.7%) were significantly correlated with unsatisfactory facial recovery. The pathogenetic mechanisms by which diabetes, hypercholesterolemia, and hypertension affect the vasa nervosum of facial nerve have been described.

Neurophysiologic and intellectual evaluation of beta-thalassemia patients.

In order to detect involvement of the central and peripheral nervous system in beta-thalassemic patients, 32 children and young adults (mean age 14.5 +/- 6.4 years) participated in a systematic neurophysiologic and intellectual prospective study. All patients were in a regular transfusion program, receiving subcutaneous desferrioxamine chelation and maintaining a mean serum ferritin level of 2,101.56 +/- 986.32 ng/ml. Study patients underwent neurophysiologic evaluation consisting of brainstem auditory, visual and somatosensory evoked potential examination (BAEP, VEP, SEP) as well as motor and sensory nerve conduction velocity studies (MCV, SCV). Additionally, the verbal, performance and total IQ were assessed in patients under 16 years of age using the Weschler Intelligence Scale for Children (WISC-III). The incidence of abnormal BAEP, VEP, SEP and NCVs was 0, 3.12, 3.12 and 18.75%, respectively, findings comparative to or better than previously reported. On the contrary, the prevalence of abnormal total IQ score was considerably high (36.4%), not correlating, however, to any of the parameters assessed (age, sex, ferritin level, BAEP, VEP, SEP, NCV). Factors associated with chronic illness, rather than the disease per se, could play a potential role in the development of cognitive dysfunction in beta-thalassemia patients.

Treatment of recurrent gastrointestinal haemorrhage in a patient with von Willebrand's disease with administration of octreotide LAR and propranolol.

We report a patient with von Willebrand' s disease who had had recurrent and life-threatening bleeding from the gastrointestinal tract. Despite extensive investigation, no apparent cause of haemorrhage was identified. He was successfully treated with combined administration of octreotide LAR (long-acting release) and propranolol. This is the first report on the successful use of octreotide LAR in a patient with von Willebrand' s disease.

Concurrent presentation of nodal myeloid sarcoma and bone marrow chronic lymphocytic leukemia/small lymphocytic lymphoma: a unique association.

Myeloid sarcoma (MS), previously known as granulocytic sarcoma, is a rare, localized, tumor mass composed of myeloid precursor cells, with or without maturation, and occurring at an anatomical site other than the bone marrow (BM). Chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL), in contrast, is a B-cell hematological malignancy. We describe the first reported case of concurrent presentation of nodal MS and of BM CLL/SLL in the same patient. Fatal leukemic central nervous system infiltration was the final outcome. We provide possible explanations and investigate the pathophysiology of this unique, previously unreported co-morbidity.

Copper levels in patients with hematological malignancies.

BACKGROUND: Copper levels are elevated in cancer patients compared to normal subjects. However, few studies have investigated the relationship between copper and hematological malignancies. METHODS: 84 patients with hematological diseases were studied, along with 50 healthy individuals. Copper was measured by flame atomic absorption spectrometry. The patients were classified to 2 homogeneous groups, acute and chronic hematological neoplasms, respectively. For the patients with acute hematological malignancies, relapse and remission were investigated in relation to serum copper levels. For chronic hematological neoplasms, serum copper was connected either with stable or progressive disease. Zeta-chain-associated protein kinase 70 (ZAP70) and CD38 expression, along with the unmutated VH immunoglobulin genes (IgVH) status were also determined for the 22 chronic lymphocytic leukemia (CLL) patients. RESULTS: 54 patients with relapse or progressive disease had elevated copper levels (mean value 1.8 mg/l), whereas 30 patients either in remission or in stable disease had normal copper levels (mean value 1.01 mg/l) (normal range 0.8-1.3mg/l). CONCLUSION: Hence, our study indicates that serum elevated copper levels are associated with hematological malignancies either in relapse or in disease progression, whereas normal copper levels are linked with hematological neoplasms in remission or in stable disease. Furthermore, we report for the first time an association between high serum copper levels and several adverse prognostic markers in CLL, such as increased expression of ZAP70 and CD38, along with elevated percentage of unmutated IgVH.

Deferiprone-related arthropathy of the knee in a thalassemic patient: report of a case and review of the literature.

Deferiprone (DFP), the first oral iron chelator, has been used in patients with beta-thalassemia major to reduce serum ferritin levels and total iron burden, leading to decreased cardiac iron levels. Major side effects include embryotoxicity, agranulocytosis, zinc deficiency and gastrointestinal disorders, while arthropathy is rarely reported. Herein, we present a 29-year-old male patient with beta-thalassemia major, who developed severe arthritis of both knees while under deferiprone therapy. Arthritis was managed successfully with non-steroid antiinflammatory drugs after DFP withdrawal.

Acute renal failure due to tumor lysis syndrome in a patient with non-Hodgkin's lymphoma.

Tumor lysis syndrome is characterized by multiple metabolic derangements resulting from the release of intracellular components into the bloodstream due to abrupt malignant cell death, spontaneously or following antineoplastic therapy. The syndrome is characterized by hyperkalemia, hyperuricemia, hyperphosphatemia, and hypocalcemia, while deposition of uric acid and calcium phosphate crystals may result in acute renal failure, which is often exacerbated by concomitant intravascular volume depletion. A case of tumor lysis syndrome complicated by acute renal failure in a patient with non-Hodgkin's lymphoma is reported and the pathophysiology, the clinical features, and the treatment options are discussed.