Gene/Protein Disease Symptom Drug Enzyme Compound
Pivot Concepts:   Target Concepts:
Query: UMLS:C0026986 (myelodysplastic syndrome)
 14,926 document(s) hit in 31,850,051 MEDLINE articles (0.00 seconds)

The Chernobyl nuclear power plant accident on April 26, 1986 was the largest in the history of the peaceful use of nuclear energy. Of the 237 individuals initially suspected to have been significantly exposed to radiation during or in the immediate aftermath of the accident, the diagnosis of acute radiation sickness (ARS) could be confirmed in 134 cases on the basis of clinical symptoms. Of these, 54 patients suffered from cutaneous radiation syndrome (CRS) to varying degrees. Among the 28 patients who died from the immediate consequences of accidental radiation exposure, acute hemopoietic syndrome due to bone marrow failure was the primary cause of death only in a minority. In 16 of these 28 deaths, the primary cause was attributed to CRS. This report describes the characteristic cutaneous sequelae as well as associated clinical symptoms and diseases of 15 survivors of the Chernobyl accident with severe localized exposure who were systematically followed up by our groups between 1991 and 2000. All patients presented with CRS of varying severity, showing xerosis, cutaneous telangiectasias and subungual splinter hemorrhages, hemangiomas and lymphangiomas, epidermal atrophy, disseminated keratoses, extensive dermal and subcutaneous fibrosis with partial ulcerations, and pigmentary changes including radiation lentigo. Surprisingly, no cutaneous malignancies have been detected so far in those areas that received large radiation exposures and that developed keratoses; however, two patients first presented in 1999 with basal cell carcinomas on the nape of the neck and the right lower eyelid, areas that received lower exposures. During the follow-up period, two patients were lost due to death from myelodysplastic syndrome in 1995 and acute myelogenous leukemia in 1998, respectively. Other radiation-induced diseases such as dry eye syndrome (3/15), radiation cataract (5/15), xerostomia (4/15) and increased FSH levels (7/15) indicating impaired fertility were also documented. This study, which analyzes 14 years in the clinical course of a cohort of patients with a unique exposure pattern, corroborates the requirement for long-term, if not life-long, follow-up not only in atomic bomb survivors, but also after predominantly local radiation exposure.
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PMID:The outcome of local radiation injuries: 14 years of follow-up after the Chernobyl accident. 1118 91

We have analyzed the long-term outcome and toxicities in 98 patients with high-risk acute myelogenous leukemia (AML) who were treated with autologous bone marrow transplantation (ABMT) and monitored for a median observation period of 11.67 years. Between 1983 and 1994, 98 patients in our institution in first or second and higher complete remission (CR) underwent total body irradiation and high-dose cyclophosphamide prior to ABMT purged with mafosfamide. Twenty-seven out of the 90 evaluable patients (30%) were alive and in continuous CR for a median of 11.67 years (range, 6.39-15.53) after ABMT and could be considered as 'cured'. Among the 90 patients, 39 were transplanted at first CR and had a significantly higher survival rate than those transplanted at > or = 2 CR. Younger patients (<40 years) had a better prognosis and patients with FAB M1-4 had a more favorable outcome than those with M5. Long-term complications included four patients with cardiac complications, two with renal insufficiency. Five developed HCV infections, four myelodysplastic syndrome. The incidence of cataract among the long-term survivors was 44.4%. Therefore, a significant number of adult patients with AML in first CR derived long-term benefit from ABMT, despite the risks of a few long-term complications and of MDS (4.4%).
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PMID:Long-term outcome in acute myelogenous leukemia autografted with mafosfamide-purged marrow in a single institution: adverse events and incidence of secondary myelodysplasia. 1210 72

A 52-year-old woman with diabetes mellitus (DM) complained of weakness of the arms and legs. She was referred to our hospital in November 2002 because of anemia, thyroid tumor and meningioma including DM. She was short in stature, juvenile bilateral cataract, intractable skin ulcers, clavus on the sole of her foot, a bird-like face and high-pitched voice. Typical physical features led to the final diagnosis of Werner's syndrome. Although the myelogram revealed no abnormal findings except erythroid hypoplasia, cytogenetic analysis of bone marrow cells showed deletion of chromosome 20 in 10% of the analyzed cells, which suggested the possibility of that myelodysplastic syndrome (MDS) or acute myeloblastic leukemia (AML) could occur. She had a thyroidectomy because both lobes of the thyroid gland were enlarged and caused hoarseness, In addition, it is common knowledge that the goiter could become malignant. We need to follow her carefully because she might be vulnerable to malignant disease, including leukemia and malignant meningioma.
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PMID:[A case of Werner syndrome with chromosomal abnormality]. 1707 96

When corticosteroid therapy, immunoglobulin and splenectomy fail to control chronic idiopathic thrombocytopenic purpura and the risk of bleeding remains high, romiplostim is an acceptable option but close monitoring is needed to evaluate long-term risks. Eltrombopag (Revolade, GlaxoSmithKline) is a synthetic non-peptide agonist of endogenous receptors for thrombopoietin, a platelet growth factor. Clinical evaluation of eltrombopag in this setting is mainly based on a double-blind placebo-controlled trial in a heterogeneous group of 114 patients. The platelet count rose to at least 50,000/mm3 for five weeks in about one-quarter of patients receiving oral eltrombopag 50 mg/day. An indirect comparison providing weak evidence suggests that romiplostim is more effective. Clinical trials did not provide evidence that either drug reduced the frequency of bleeding. The haematological risks associated with eltrombopag are poorly evaluated, and mainly include: thrombosis, bone marrow disorders, and aggravation of thrombocytopenia after drug withdrawal. Aggravation of myelodysplastic syndrome cannot be ruled out in the long term. Hepatic disorders such as photosensitisation are frequent. The risk of cataract formation and renal impairment requires further study. There appears to be a high risk of pharmacokinetic interactions through a variety of mechanisms, including enzyme competition and cation binding, but this risk is not well documented. Eltrombopag is administered orally, making it more convenient than romiplostim, which necessitates weekly subcutaneous injections. In practice, when standard treatments fail in patients with chronic idiopathic thrombocytopenic purpura and a high risk of bleeding, it is better to use romiplostim, which appears to be somewhat more effective than eltrombopag. Eltrombopag also seems to carry a higher risk of non-haematological adverse effects and drug interactions.
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PMID:Eltrombopag. Idiopathic thrombocytopenic purpura after treatment failure: romiplostim is a better option. 2045 33