On 3 July 2006, a new orphan medicinal product was registered into the community register of orphan medicinal products under the EU orphan designation number 383. The active ingredient is siplizumab for treatment of T-cell and NK-cell neoplasms.
Publications
PubMed, the Internet portal of biomedical and life sciences literature, indexed an interesting article, entitled “Crigler-Najjar syndrome: Diagnosis and treatment.” (An Pediatr (Barc). 2006 Jul;65(1):73-8). Authors are Lodoso Torrecilla B, Palomo Atance E, Camarena Grande C, et al., from the Servicio de Hepatologia. Hospital Infantil Universitario La Paz. Madrid, Espana. Crigler-Najjar syndrome (CNS) is a very rare disease characterized by severe indirect hyperbilirubinemia from birth with normal liver function. It may cause kernicterus at any age. This disease is due to a total or partial deficiency of the UDP-glucuronosyltransferase enzyme caused by a mutation of the five exons of the ULT1A1 gene. The authors reviewed the clinical outcomes of 7 children diagnosed with CNS between 1987 and 2004. The conclusions are that patients with CNS are at greater risk of developing kernicterus, mostly associated with indirect bilirubin levels of around 25 mg/dl. Phototherapy is very useful in these patients but the only definitive treatment is liver transplantation. To access the full abstract of the article, click here.
PubMed, the Internet portal of biomedical and life sciences literature, indexed an interesting article, entitled “Churg-Strauss syndrome: update on clinical, laboratory and therapeutic aspects.” (Sarcoidosis Vasc Diffuse Lung Dis. 2006 Mar;23(1):3-12). Authors are Keogh KA and Specks U, from the Division of Pulmonary and Critical Care Medicine, Department of Medicine, Mayo Clinic College of Medicine, Rochester, MN, USA. Originally described over fifty years ago as a disorder of asthma, eosinophilic inflammation and small vessel vasculitis, Churg-Strauss syndrome is now defined as one of the ANCA (anti-neutrophil cytoplasmic antibodies) – associated vasculitides. The allergic background in which the vasculitis presents, typically characterized by asthma and prominent peripheral blood and tissue eosinophilia, render it unique among the primary systemic vasculitis syndromes. Despite recent interest in a potential link between leukotriene receptor antagonist use for asthma and the onset of Churg-Strauss syndrome, it remains a rare disease with poorly understood pathogenesis. To access the full abstract of the article, click here.
On 3 July 2006, a new orphan medicinal product was registered into the community register of orphan medicinal products under the EU orphan designation number 382. The active ingredient is pazopanib hydrochloride for treatment of renal cell carcinoma.
PubMed, the Internet portal of biomedical and life sciences literature, indexed an interesting article, entitled “Treatment of skeletal Erdheim-Chester disease with zoledronic acid: case report and proposed mechanisms of action” (Rheumatol Int. 2006 Aug 25). Authors are Srikulmontree T, Massey HD and Roberts WN, from the Rheumatology Section, Hunter Holmes McGuire Medical Center, Richmond, VA, USA. Erdheim-Chester disease (ECD) is a rare non-Langerhans cell histiocytosis characterized by tissue infiltration of lipid-laden macrophages, multinucleated giant cells, and inflammatory infiltrate of lymphocytes and histiocytes. The disease typically involves long bone, but may also affect the central nervous system, the orbit, retroperitoneal organs, and the lungs. There is no proven effective treatment for ECD to date. However, recent data suggested the potential use of bisphosphonates for the treatment of this rare disease. The authors report a case of biopsy-proven skeletal ECD, who received treatment with zoledronic acid, an aminobisphosphonate, with remarkable clinical improvement. To access the full abstract of the article, click here.
PubMed, the Internet portal of biomedical and life sciences literature, indexed an interesting article, entitled “Sildenafil for pulmonary arterial hypertension: when blue turns into white” (Expert Opin Pharmacother. 2006 Sep;7(13):1801-10). Author is Antoniu SA, from the Clinic of Pulmonary Disease, Gr.T.Popa Iasi, Iasi, Romania. Pulmonary arterial hypertension is a life-threatening, rare disease characterised by vasoconstriction and vascular remodeling of pulmonary artery vessels. Until several years ago, therapeutic approaches were represented mainly by ‘conventional therapy’ (anticoagulants, calcium channel blockers, diuretics and digoxin, and oxygen therapy). But recently ‘specific therapies’ (i.e., therapies targeting specific pathogenic pathways) have become available; these are therapies represented by prostacyclin and its derivatives, endothelin receptor antagonists or phosphodiesterase-5 inhibitors. To access the full abstract of the article, click here.
PubMed, the Internet portal of biomedical and life sciences literature, indexed an interesting article, entitled “Clinical and immunologic characteristics in peripartum cardiomyopathy” (Int J Cardiol. 2006 Aug 9). Authors are Lamparter S, Pankuweit S and Maisch B from the Diakonie Krankenhaus Wehrda, Internal Medicine, Marburg-Wehrda, Germany. Peripartum cardiomyopathy (PPCM) is a rare disorder of dilated cardiomyopathy and left ventricular dysfunction occurring in the last month of pregnancy or within 5 months postpartum. Outcome of PPCM is highly variable, comprising clinical improvement and rapid deterioration unresponsive to medical treatment requiring heart transplantation or even death. In this retrospective observational study the authors report the clinicopathologic findings of 10 patients with PPCM. The findings support the hypothesis of an underlying autoimmune pathomechanism in this rare disease. To access the full abstract of the article, click here.
On 3 July 2006, a new orphan medicinal product was registered into the community register of orphan medicinal products under the EU orphan designation number 381. The active ingredient is human monoclonal antibody against Pseudomonas aeruginosa serotype O11 for treatment of pneumonia caused by serotype O11 Pseudomonas aeruginosa.
PubMed, the Internet portal of biomedical and life sciences literature, indexed an interesting article, entitled “Primary systemic amyloidosis. Early diagnosis and therapy can improve survival rates and quality of life” (Postgrad Med. 2006 Jun-Jul;119(1):93-9 ). Authors are Roy A and Roy V from the Division of Hospital Medicine, Mayo Clinic Jacksonville, Florida, USA. Primary systemic amyloidosis is a rare disease with protean manifestations. Presence of nephrotic syndrome in the absence of diabetes and hypertension, cardiomyopathy in the absence of ischemia, restrictive cardiac defect, demyelinating polyneuropathy, or unexplained hepatomegaly should alert the physician to the possibility of amyloidosis. Initial steps in the diagnostic evaluation of patients with suspected amyloidosis include serum and urine immunoelectrophoresis and immunofixation studies. Demonstration of amyloid material on tissue biopsy (e.g., subcutaneous fat) is required for diagnosis. Availability of effective treatments has improved the outlook of patients with primary systemic amyloidosis. Early diagnosis is critical to optimizing the chances of effective therapy. To access the abstract of the article, click here.
PubMed, the Internet portal of biomedical and life sciences literature, indexed an interesting article, entitled “Long-term remission in BCR/ABL-positive AML-M6 patient treated with Imatinib Mesylate.” (Leuk Res. 2006 Aug 14). Authors are Pompetti F, Spadano A, Sau A et al., from the Molecular Biology Laboratory, Department of Transfusional Medicine, Ospedale Civile ‘Spirito Santo’, Pescara, Italy. BCR/ABL-positive acute myeloid leukemia (AML) is a rare disease, characterized by a poor prognosis, with resistance to induction chemotherapy and frequent relapses in responsive patients. The authors report a case of BCR/ABL-positive AML-M6 who, after relapse, was treated with Imatinib Mesylate (600mg/die) and within 4 months achieved a cytogenetic and molecular complete response. To access the abstract of the article, click here.