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

A multicenter phase II study was initiated to investigate the efficacy, toxicity and tolerability of an oral regimen of 9-cis retinoic acid (9CRA) as a differentiation-inducing agent stimulating both retinoic acid receptor (RAR) and retinoic X receptor (RXR). Thirty patients with myelodysplastic syndromes (MDS) were enrolled into the study. The MDS subtypes were distributed as follows: 14 refractory anaemia (RA), four refractory anaemia with ringed sideroblasts (RARS), and 12 refractory anaemia with excess blasts (RAEB). The age ranged from 40 to 81 years (median 70). None of these had previously received treatment for MDS other than supportive therapy. 9CRA (Alitretinoin capsules, kindly provided by Allergan-Ligand Retinoid Therapeutics) was given daily at 60 mg/m2 p.o. for 1 week, followed by an intra-patient escalation to 100 mg/m2 during the second week, up to a maximum of 140 mg/m2. The planned treatment duration was 48 weeks. Twenty-five were available for assessment. One patient (4%) with RA achieved complete hematological remission. Four (16%), two with RA, two with RAEB, had minor responses resulting in decreased transfusion requirements or increased neutrophils. Thus, the overall response rate was 20% in evaluable patients with MDS and 17% in the study group on an intention-to-treat basis. The most frequent side-effects included headache (77%), dry skin (57%), arthralgias (30%), and rash (23%). In conclusion, although modest responses were noted in this study, the treatment tolerability was suboptimal. It is conceivable that a lower dosage schedule may be efficacious and better tolerated so enabling prolonged exposure which may be required to induce a differentiation effect.
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PMID:Oral 9-cis retinoic acid (Alitretinoin) in the treatment of myelodysplastic syndromes: results from a pilot study. 1099 4

Botulinic toxin (BT) is a new method of lowering intraurethral pressure in symptoms of obstructive voiding in patients with neurogenic dysfunction of the lower urinary tracts (LUT). Transperineal introduction of 100 units of BT type A (botox, Allergan) was used under electromyographic control into the external urethral sphincter of 9 patients (6 males and 3 females) with LUT neurogenic dysfunction aged 17 to 68 years (mean age 37.2 years). Two patients had subnormal detrusor contractility due to myelodysplasia and diabetic polyneuropathy, two other patients--non-incontinent striated urethral sphincter after hemorrhagic stroke and spinal contusion, five patients suffered from detrusor-sphincteral dyssynergia (DSD) resultant from Schmorl's hernia, multiple sclerosis, Charcot-Marie disease and ischemic stroke of the spinal cord. Three patients had cystostomic drainage. The rest of the patients complained of dysuria, three patients performed self-catheterization, mean volume of the residual urine was 170 ml (180-240 ml). In 10 days residual urine was not found in 2 patients with subnormal detrusor contractility and in 4 patients with DSD. Abdominal pressure fell from 75 to 39 cm, on the average. In DSD patients maximal detrusor pressure fell from 59 to 29 cm, on the average. Mean maximal urinary flow rate rose from 4.3 to 9.6 ml/s. In 20 days, on the average, suprapubic fistula healed in all the patients. In a month, therapeutic effect persisted in all the patients. Complications, side effects were not registered. BT treatment to induce adequate urine evacuation in neurological patients is a promising approach in neurourology. Further studies should find answers to questions about regimen of BT introduction, loss of sensitivity, new indications in urology.
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PMID:[Botulinic toxin in patients with neurogenic dysfunction of the lower urinary tracts]. 1545 54