Effect of a single intra-articular administration of stanozolol in a naturally occurring canine osteoarthritis model: a randomised trial.

Osteoarthritis (OA) is a disease with a high negative impact on patient's quality of life and a high financial burden. It is a source of chronic pain and affects all mammals, including humans and dogs. As the dog is a common model for translation research of human OA, and exploring spontaneous dog OA can improve the health and well-being of both humans and dogs. To describe the effect of the intra-articular administration of stanozolol in a naturally occurring canine OA model, forty canine (N = 40) hip joints were randomly assigned to receive stanozolol or saline (control). On treatment day and at 8, 15, 30, 90, and 180 days post-treatment, several evaluations were conducted: weight distribution, joint range of motion, thigh girth, digital thermography, and radiographic signs. Also, synovial fluid C-reactive protein and interleukin-1 levels were evaluated. Results from four Clinical Metrology Instruments was also gathered. Results were compared with Repeated Measures ANOVA, with a Huynh-Feldt correction, paired-samples t-test, or Wilcoxon signed-rank test, with p < 0.05. OA was graded as mild (90%), moderate (5%), and severe (5%), including both sexes. They had a mean age of 6.5 ± 2.4 years and a bodyweight of 26.7 ± 5.2 kg. No differences were found between groups at treatment day in all considered evaluations. Weight distribution showed significant improvements with stanozolol from 15 days (p < 0.05) up to 180 days (p < 0.01). Lower values during thermographic evaluation in both views taken and improved joint extension at 90 (p = 0.02) and 180 days (p < 0.01) were observed. Pain and function scores improved up to 180 days. In the control group, radiographic signs progressed, in contrast with stanozolol. The use of stanozolol was safe and produced significant improvements in weight-bearing, pain score, and clinical evaluations in a naturally occurring canine OA model.

GnRHa/Stanozolol Combined Therapy Maintains Normal Bone Growth in Central Precocious Puberty.

Background: Gonadotropin-releasing hormone agonist (GnRHa) is the gold standard in the treatment of Central Precocious Puberty (CPP) with progressive puberty and accelerative growth. However, GnRHa treatment is reported to result in growth deceleration and prevents growth plate development which leads to a reduction in height velocity. Stanozolol (ST) has been used to stimulate growth in patients with delayed growth and puberty, nevertheless, the effects and mechanisms of ST on CPP with GnRHa treatment are currently unclear. Methods and Results: In the current study, we recorded the following vital observations that provided insights into ST induced chondrogenic differentiation and the maintenance of normal growth plate development: (1) ST efficiently prevented growth deceleration and maintained normal growth plate development in rats undergoing GnRHa treatment; (2) ST suppressed the inhibitory effect of GnRHa to promote chondrogenic differentiation; (3) ST induced chondrogenic differentiation through the activation of the JNK/c-Jun/Sox9 signaling pathway; (4) ST promoted chondrogenic differentiation and growth plate development through the JNK/Sox9 signaling pathway in vivo. Conclusions: ST mitigated the inhibitory effects of GnRHa and promoted growth plate development in rats. ST induced the differentiation of chondrocytes and maintained normal growth plate development through the activation of JNK/c-Jun/Sox9 signaling. These novel findings indicated that ST could be a potential agent for maintaining normal bone growth in cases of CPP undergoing GnRHa treatment.

Stanozolol for the treatment of anemic lower-risk myelodysplastic syndromes without del(5q) after failure of epoetin alfa: findings from a retrospective study.

Options for anemic lower-risk myelodysplastic syndromes (MDS) without del(5q) after failure of erythropoiesis-stimulating agents (ESAs) are very limited. The effectiveness of second-line treatments is uncertain. We retrospectively reviewed the clinical effectiveness and overall survival (OS) of lower-risk MDS without del(5q) patients exclusively treated with stanozolol (STZ) after failure of epoetin alfa. The response was defined according to the 2006 International Working Group (IWG) criteria. Fifty-six patients were included. The median follow-up time was 55 months (range: 5-156 months). Twenty-seven patients (48.2%) achieved hematologic improvement-erythroid response (HI-E). Higher response rates were observed in patients with lower IPSS-R scores (≤3.5, P = 0.008) and hypocellular bone marrow (P = 0.002). In univariate Cox analysis, HI-E was the strongest factor associated with better OS (P = 0.0003). In multivariate Cox, HI-E, age ≤ 50, and transfusion independence (TI) at the onset of STZ were factors associated with better OS. The estimated 5-year OS was 88.6% (68.7-96.2%) and 33.8% (14.9-54.0%) in responders and non-responders (P < 0.01), respectively. The most common side effects included masculinization and liver damage, but they were manageable with supportive measures and dose adjustments. STZ may be considered an alternative treatment in lower-risk MDS after failure of epoetin alfa.

Intraarticular triamcinolone hexacetonide, stanozolol, Hylan G-F 20 and platelet concentrate in a naturally occurring canine osteoarthritis model.

Osteoarthritis (OA) is a disease transversal to all mammals, a source of chronic pain and disability, a huge burden to societies, with a significant toll in healthcare cost, while reducing productivity and quality of life. The dog is considered a useful model for the translational study of the disease, closely matching human OA, with the advantage of a faster disease progression while maintaining the same life stages. In a prospective, longitudinal, double-blinded, negative controlled study, one hundred (N = 100) hip joints were selected and randomly assigned to five groups: control group (CG, n = 20, receiving a saline injection), triamcinolone hexacetonide group (THG, n = 20), platelet concentrate group (PCG, n = 20), stanozolol group (SG, n = 20) and hylan G-F 20 group (HG). Evaluations were conducted on days 0 (T0, treatment day), 8, 15, 30, 60, 90, 120, 150 and 180 days post-treatment, consisting of weight distribution analysis and data from four Clinical Metrology Instruments (CMI). Kaplan-Meier estimators were generated and compared with the Breslow test. Cox proportional hazard regression analysis was used to investigate the influence of variables of interest on treatment survival. All results were analyzed with IBM SPSS Statistics version 20 and a significance level of p < 0.05 was set. Sample included joints of 100 pelvic limbs (of patients with a mean age of 6.5 ± 2.4 years and body weight of 26.7 ± 5.2 kg. Joints were graded as mild (n = 70), moderate (n = 20) and severe (n = 10) OA. No differences were found between groups at T0. Kaplan-Meier analysis showed that all treatments produced longer periods with better results in the various evaluations compared to CG. Patients in HG and PCG took longer to return to baseline values and scores. A higher impact on pain interference was observed in THG, with a 95% improvement over CG. PCG and HG experienced 57-81% improvements in functional evaluation and impairments due to OA, and may be a better options for these cases. This study documented the efficacy of several approaches to relieve OA clinical signs. These approaches varied in intensity and duration. HG and PCG where the groups were more significant improvements were observed throughout the follow-up periods, with lower variation in results.

Multiple hepatocellular adenomas associated with long-term administration of androgenic steroids for aplastic anemia: A case report and literature review.

INTRODUCTION: Anabolic steroids are widely administered to patients with aplastic anemia (AA) and are associated with numerous medical complications. To assist with future diagnoses, we report about a young boy with multiple hepatocellular adenomas (HAs) induced by long-term use of anabolic androgenic steroids (AAS) for AA and present a related literature review. PATIENT CONCERN: A 15-year-old boy who was diagnosed with AA in 2011 had been treated with stanozolol (6 mg per day) and ciclosporin A (120-150 mg per day) for almost 4 years. He presented with epigastric pain and fever, and abdominal computed tomography showed a lesion of heterogenous density measuring 13.5 × 13.0 × 8.0 cm in the left hepatic lobe, which was initially misdiagnosed as a liver abscess. DIAGNOSIS: The patient went into hemorrhagic shock twice after invasive manipulation that aimed at diagnosis and was finally diagnosed with HA using fine needle aspiration. INTERVENTIONS: The patient discontinued AAS and only reserved ciclosporin A for AA treatment. OUTCOMES: Follow-up abdominal computed tomography performed 4 years after AAS discontinuation showed obvious regression of the hepatic lesions. CONCLUSION: It is of great importance for hematologists to completely understand that the long-term use of AAS may cause HA, which carries a great risk of hemorrhage and malignant transformation.

Design, synthesis and characterization of a PEGylated stanozolol for potential therapeutic applications.

Stanozolol (STZ) is a drug used to treat serious disorders like aplastic anemia and hereditary angioedema. It is also indicated as an adjunct therapy for the treatment of vascular disorders and growth failures. Encouraging results obtained using animal models demonstrated that STZ increases bone formation and mineralization, thus improving both density and biomechanical properties. Like natural androgens, such as TST and 5α-dihydrotestosterone (5α-DHT), STZ binds androgen receptor (AR) to activate AR-mediated signaling. Despite its therapeutic effects, this synthetic anabolic-androgenic steroid (AAS), or 5α-DHT derivative, due to its high lipophilicity, is poor soluble in water. Thus, to increase the water solubility and stability of STZ, as well as its bioavailability and efficacy, an innovative PEGylated STZ (STZ conjugated with (MeO-PEG-NH2)10kDa, (MeO-PEG-NH)10kDa-STZ) was synthesized. As confirmed by chromatography (RP-HPLC) and spectrometry (ATR-FTIR, 1H NMR, elemental CHNS(O) analysis, MALDI-TOF/TOF) analyses, a very pure, stable and soluble compound was obtained. Acetylcholinesterase (AChE) competitive ELISA demonstrated that the resulting PEGylated STZ competes against biological TST, especially at lower concentrations. Cytotoxicity of increasing concentrations (1, 10, 25 or 50 µM) of STZ and/or (MeO-PEG-NH)10kDa-STZ was also evaluated for up 80 h by performing the MTT assay on human osteosarcoma Saos-2 cells, which express AR and are responsive to STZ. PEGylation mitigated cytotoxicity of STZ, by increasing the cell viability values, especially at higher drug concentrations. Furthermore, these results suggest that (MeO-PEG-NH)10kDa-STZ is a promising and reliable drug to be used in clinical conditions in which TST is required.

Improvement of the hematologic toxicities of ruxolitinib in patients with MPN-associated myelofibrosis using a combination of thalidomide, stanozolol and prednisone.

Objective: Anemia and thrombocytopenia are the most frequently reported adverse events of ruxolitinib in patients with MPN-associated myelofibrosis (MPN-MF). Although thalidomide, androgens and prednisone have previously demonstrated improvements in myelofibrosis-associated anemia, it is unclear whether these drugs are effective in patients taking ruxolitinib. Method: We conducted a retrospective cohort study to evaluate the efficacy and tolerability of combination therapy with low dose thalidomide, stanozolol and prednisone (TSP) in patients with IPSS intermediate-2 or high-risk myelofibrosis (MF) who received ruxolitinib treatment. Results: Sixty-five patients with MPN-MF who took ruxolitinib were enrolled in this retrospective study, of which 46 patients also took TSP while 19 did not take TSP (TSP and non-TSP groups). Within the first 24 weeks, the proportion of patients with anemia response and platelet count increase ≥50 × 109/L were 45.7% and 67.4% in the TSP group as compared to 0% and 10.5% in the non-TSP group (p < 0.001). The mean hemoglobin level in the non-TSP group reached the nadir after approximately 12-16 weeks of therapy, but gradually increased in the TSP group. Conclusion: In summary, TSP regimen can improve anemia and thrombocytopenia during ruxolitinib treatment in patients with MPN-MF, and the associated adverse events were manageable.

Critical appraisal of androgen use in hereditary angioedema: a systematic review.

OBJECTIVE: To provide an objective basis for evaluating the risk-benefit ratio of long-term androgen use in patients with hereditary angioedema (HAE). DATA SOURCES: PubMed was searched with no time limitations using the keywords hereditary angioedema or angio-oedema combined with danazol, stanozolol, and androgen. STUDY SELECTION: Qualifying articles were English-language reports of androgen use in patients with HAE, with relevant safety and/or efficacy information. Reports were categorized according to level of evidence (LOE). RESULTS: The search process identified 153 citations, 63 of which contained relevant information; 2 additional publications were identified while other citations were being reviewed. Fifteen LOE 2 studies and multiple LOE 4 reports provided efficacy data, confirming a high level of prophylactic efficacy for androgen therapy in HAE, with occasional reports of poor prophylactic response. Common adverse events include weight gain, menstrual irregularities, virilization, headaches, myalgias or cramps, mood changes, and elevations in creatine phosphokinase level, liver function test results, and serum lipid level. The risk of adverse events is often correlated with dose and/or treatment duration. Rare cases of hepatic adenomas and hepatocellular carcinoma associated with long-term androgen use often had no preceding changes in liver function test results. CONCLUSION: Androgen therapy may be effective for most patients with HAE; however, potential risks and adverse effects must be carefully considered and discussed with patients when considering options for long-term HAE prophylaxis.

Liver enzymes and lipid levels in patients with lipodermatosclerosis and venous ulcers treated with a prototypic anabolic steroid (stanozolol): a prospective, randomized, double-blinded, placebo-controlled trial.

Anabolic steroids have been used to treat lower extremity ulcerations, including venous and cryofibrinogenemic ulcers and lipodermatosclerosis (LDS). Yet there have been no studies to determine the severity and reversibility of side effects of anabolic steroids on liver enzymes and lipid profiles in elderly patients. We therefore evaluated, in a prospective, randomized, double-blinded, placebo-controlled trial, the extent and reversibility of abnormal liver enzymes and lipid profiles in patients with LDS and venous leg ulcers treated with stanozolol at 2 mg twice daily for up to 6 months. Follow-up laboratory testing was done for 2 months after cessation of treatment. A total of 44 patients with LDS and venous ulcers were enrolled and treated with either leg compression alone (placebo) or leg compression plus oral stanozolol 2 mg twice daily (active). Baseline and follow-up laboratory testing of liver enzymes and lipid profiles were obtained. A total of 21 active and 23 placebo patients were treated and evaluated. We measured liver enzymes (aspartate aminotransferase [AST/SGOT], alanine aminotransferase [ALT/SGPT], γ-glutamyl transferase [GGT]) and lipid profile components (high-density lipoprotein [HDL], low-density lipoprotein [LDL], total cholesterol) before, during, and after the treatment period. We found that AST/SGOT and ALT/SGPT became significantly elevated in 29% (P = .0415 at 2 months) and 33% (P = .0182 at 1 month) of patients treated with stanozolol or placebo, respectively, with return to baseline in the posttreatment period. Unexpectedly, 91% of patients on stanozolol developed a significant (P < .0001) decrease in HDL levels, by as much as 37 U/L. All patients remained asymptomatic and levels returned to baseline after discontinuation of the drug. We conclude that low-dose stanozolol, 2 mg twice daily, produces asymptomatic and temporary elevation of liver transaminases and depression of the HDL level in a significant proportion of patients.

Clinical pattern and acute and long-term management of hereditary angioedema due to C1-esterase inhibitor deficiency

Background: Hereditary angioedema due to C1-esterase inhibitor deficiency (HAE-C1-INH) is a life-threatening disease. Objectives: To describe the clinical characteristics and management of patients with HAE-C1-INH during routine clinical practice. Methods: An observational, retrospective study was performed in patients with HAE-C1-INH. Demographic, clinical, and analytical data were collected from 2 periods: period A (October 2009-September 2010) and period B (October 2007-September 2009). Results: We studied 112 patients with HAE-C1-INH (57.1% females). Age at onset of symptoms was 14.4 years (lower in patients who had experienced attacks in the previous year). In period B (n=87), 62.1% of patients presented at least 1 edema attack (median, 3.5 attacks/patient/2 years), and 19.1% of attacks were treated. In period A (n=77), 58.4% of patients were on maintenance therapy. Stanozolol was the most widely used drug (48.9%), with a mean weekly dose of 6.7 mg. At least 1 attack was recorded in 72.7% of patients (median, 3.0 attacks/patient/year), and 31.5% of the attacks were treated. Treatment of acute attacks increased by 12.4%. Conclusion: Age at onset of symptoms is associated with clinical expression of disease. The higher age at onset of symptoms, the fewer number of attacks per patient and year, and the lower dose of attenuated androgens necessary to control the disease than in other series lead us to hypothesize that HAE-C1-INH could have a less severe expression in Spain. Acute attacks seem to be treated increasingly often (AU)

Antecedentes: El angioedema hereditario por déficit del inhibidor de la C1 esterasa (AEH-C1-INH) es potencialmente mortal. Objetivos: Describir las características clínicas y el manejo de pacientes con AEH-C1-INH durante la práctica clínica habitual. Métodos: Estudio retrospectivo observacional de pacientes con AEH-C1-INH. Se recogieron datos demográficos, clínicos y analíticos en los periodos A (Octubre 2009-Septiembre 2010) y B (Octubre 2007-Septiembre 2009). Resultados: Se estudiaron 112 pacientes con AEH-C1-INH (57,1% mujeres) con edad de inicio de los síntomas de 14,4 años (inferior en aquellos pacientes con ataques en el último año). En el periodo B (n=87) 62,1% tuvo al menos un ataque (mediana: 3,5 ataques/paciente /2 años) y el 19,9% de los ataques se trataron. En el periodo A (n=77) 58,4% recibieron tratamiento de mantenimiento, siendo el estanozolol el fármaco más utilizado (48,9%) (dosis media semanal 6,7mg). El 72,7% de los pacientes tuvo al menos un ataque (mediana: 3,0 ataques / paciente / año), el 31,5% se trataron. Hubo un incremento del 12,4% de tratamientos de ataques agudos. Conclusiones: La edad de inicio de los síntomas está relacionada con la expresión clínica de la enfermedad. La edad superior del inicio de los síntomas, el menor número de ataques por paciente/año, y una dosis inferior de andrógenos atenuados para controlar la enfermedad, comparado con otros países, permite hipotetizar que el AEH-C1-INH en España tendría una expresión clínica menos grave. Existe una tendencia al alza en la frecuencia de tratamiento de ataques agudos (AU)

Effects of intraarticular treatment with stanozolol on synovial membrane and cartilage in an ovine model of osteoarthritis.

Aim of the study was to verify the clinical and morphological effects of intra-articular stanozolol or placebo treatment, lasting 3 and 9 months, in sheep in which a femoro-tibial osteo-arthritis (OA) were surgically induced (medial bilateral meniscectomy). Twenty healthy sheep divided into four groups and two control animals group, after surgical medial bilateral meniscectomy, were weekly injected in femoral-tibial joint (FTJ) with stanozolol or placebo. Lameness evaluation was performed and synovial fluid was collected from all sheep at each treatment time. Necropsies were performed after 3 or 9 month as described in experimental design. Gross pathologies were described and specimen tissues collected from femoro-tibial articular joints were processed for routine histological examination. The gross anatomy of the FTJ was well-preserved in stanozolol-treated sheep; this also applied to the histological features of articular cartilage. Joint aseptic inflammation and fibrosis were observed in placebo-treated sheep, associated with a different degree of severity of condylar and tibial plate cartilage degeneration. Stanozolol intra-articular treatment reduces osteophytes formation and subchondral bone reaction and promotes articular cartilage regeneration.

The progression risk factors of children with transfusion-independent non-severe aplastic anemia.

Non-severe aplastic anemia (NSAA) in children is a rare, idiopathic condition of bone marrow insufficiency that can resolve spontaneously, persist for months or years, or progress to severe aplastic anemia (SAA). We reviewed clinical and laboratory data of patients diagnosed with transfusion-independent non-severe aplastic anemia (NSAA) from 1996 to 2009 at the Institute of Hematology and Blood Diseases Hospital, Peking Union Medical College, and analyzed the clinical course and outcomes in these patients. NSAA was defined as bone marrow cellularity <50 % and two or three cytopenias that persisted for 6 weeks or more: absolute neutrophil count (ANC) <1.5 × 10(9)/L, absolute reticulocyte count (ARC) <40 × 10(9)/L, platelet count <100 × 10(9)/L, without meeting criteria for SAA (bone marrow cellularity <30 % and two or three cytopenias: ANC <0.5 × 10(9)/L, ARC <20 × 10(9)/L, platelet count <20 × 10(9)/L). All patients were treated with reasonable supportive care, cyclosporine A, and stanozolol (0.1 mg/kg/day). Of a total of 284 patients, 117 (41.2 %) were female, and 167 (58.8 %) were male. With a median follow-up of 43 months (range 2-196 months), 38 patients (13.4 %) progressed to transfusion-dependent NSAA and among them 26 patients (9.2 %) progressed to SAA. One hundred and ninety-eight patients (69.7 %) had persistent NSAA. Forty-eight patients (16.9 %) showed the complete resolution of NSAA. The Kaplan-Meier estimates of all patients for progression-free survival were 86 ± 2.7 % and 66 ± 7.3 % at 60 and 120 months after diagnosis, respectively. Patients with ANC <1.0 × 10(9)/L or female had a higher probability of progression to transfusion-dependent NSAA (18.5 vs. 5.4 %, respectively; p = 0.002, and 17.1 vs. 10.8 %, respectively; p = 0.022). The patients with ARC <60 × 10(9)/L or with ANC <1.0 × 10(9)/L had a higher probability of progression to SAA (11.5 vs. 3.6 %, respectively; p = 0.035, and 12.7 vs. 3.6 %, respectively; p = 0.011). A categorical risk factor analysis showed that patients with ANC <1 × 10(9)/L had a higher probability of progression to SAA (p = 0.03) and had a higher probability of progression to transfusion-dependent AA (p = 0.007). NSAA patients may be benefited from early intervention with cyclosporine A and stanozolol.

[Effect of gonadotropin-releasing hormone analog combined with stanazolol on final height in girls with idiopathic central precocious puberty and apparent decrease of linear growth].

OBJECTIVE: To evaluate the effect of combined use of stanazolol (ST) on the final adult height (FAH) in girls with idiopathic central precocious puberty (ICPP) and apparently decreased linear growth during gonadotropin-releasing hormone analog (GnRHa) therapy. METHOD: Sixty-three girls with ICPP and decreased velocity of growth of height (HV<4 cm/yr) during GnRHa therapy were divided into 3 groups based on the following types of interventions:group 1 (n = 20), GnRHa+ST [25-30 µg/(kg·d) every 3-month followed by 3-month discontinuation], group 2 (n = 21), GnRHa+recombinant human growth hormone [rhGH, 1-1.1 U/(kg·w)], group 3 (n = 22), GnRHa alone.HV, the advancement of bone age (BA) for chronological age (CA) (ΔBA/ΔCA) and FAH were compared among groups. RESULT: (1)Total duration of ST combination therapy was (12.22 ± 3.62) months, while total duration of combination of rhGH was (13.22 ± 6.80) months. (2)HV increased significantly in both group 1 [ (2.79 ± 0.60) cm/yr vs. (6.27 ± 1.98) cm/yr, P < 0.01] and in group 2 [(2.80 ± 0.50) cm/yr vs. (6.25 ± 1.98) cm/yr, P < 0.01] during combined therapy, but maintained at low levels in group 3 [(3.95 ± 1.10) cm/yr vs. (3.34 ± 0.95) cm/yr, P > 0.05].No significant differences of ΔBA/ΔCA were found among the three groups [0.25(0.11∼0.28), 0.22(0.15∼0.31),0.19(0.10∼0.32), P > 0.05]. (3)FAH was significantly higher than predicted adult height (PAH) before combined therapy, as well as higher than target height (THt) in both group 1 [(156.25 ± 2.90) cm vs. (150.78 ± 3.70) cm, P < 0.01, (156.25 ± 2.90) cm vs. (153.94 ± 2.62) cm, P < 0.01], and in group2 [ (157.33 ± 4.69) cm vs. (152.61 ± 3.92) cm, P < 0.01, (157.33 ± 4.69) cm vs. (154.39 ± 4.72) cm, P = 0.01].In group 3, FAH was similar to PAH [(153.88 ± 2.6) cm vs. (152.54 ± 5.86) cm, P > 0.05], and was less than THt [(153.88 ± 2.6) cm vs. (155.60 ± 4.52) cm, P = 0.02]. (4)In girls treated with ST, no hirsutism, clitorism or hoarse voice was recorded.No polycystic ovary syndrome was found by B-mode ultrasound. CONCLUSION: Intermittent combined use of low dose ST therapy can increase HV and thus improve FAH in girls with ICPP and apparently decreased linear growth during GnRHa therapy.

Current management options for hereditary angioedema.

The aim of treatment of hereditary angioedema (HAE) due to C1 esterase inhibitor deficiency (HAE-C1-INH) is either treating acute attacks or preventing attacks by using prophylactic treatment. For treating acute attacks, plasma-derived C1 inhibitor (C1-INH) concentrates, a bradykinin B2 receptor antagonist, and a recombinant human C1-INH are available in Europe. In the United States, a plasma-derived C1-INH concentrate, a bradykinin B2 receptor antagonist, and a plasma kallikrein inhibitor have been approved. Fresh frozen plasma is also available for treating acute attacks. Short-term prophylactic treatment focuses on C1-INH and attenuated androgens. Long-term prophylactic treatments include attenuated androgens such as danazol, stanozolol, and oxandrolone, antifibrinolytics, and a plasma-derived C1-INH concentrate. Plasma-derived C1-INH and a bradykinin B2 receptor antagonist are permitted for self-administration and home therapy. The number of management options has increased considerably within the last few years, thus helping to diminish the burden of HAE.

[114 children with acquired non-severe aplastic anemia benefitted from androgen].

This study was purposed to assess the efficacy of stanozolol for treatment of childhood patients with acquired non-severe aplastic anemia (NSAA). The records of 114 children with acquired NSAA treated in hospital between January 1996 and January 2009 were analyzed retrospectively. All patients received stanozolol with the dose of 0.1 mg/(kg·d). Some patients were treated with supportive care. The incidence and the risk factors of progression severe aplastic anemia (SAA) including gender, age, absolute neutrophil count, absolute reticulocyte count, dependent or independent of transfusion, the ratio of granulocytes and erythrocytes were evaluated. The results indicated that at a median follow-up of 52 months (range 5 - 181), 6 patients (5.3%) progressed into SAA, 93 (81.6%) remained in NSAA, and 15 (13.2%) had complete remission. Patients with dependent of transfusion (platelet count < 10 × 10(9)/L and/or haemoglobin < 70 g/L) have higher risk to progress into SAA (19.2% vs 1.1%) (p = 0.016); patients with lower absolute neutrophil count (ANC) (< 0.8 × 10(9)/L) or with lower absolute reticulocyte count (ARC) (< 40 × 10(9)/L) at diagnosis have higher risk to progress into SAA (8.1% vs 0%) (p = 0.029); (9.1% vs 1.7%) (p = 0.034); Those patients with lower ANC (ANC < 0.8 × 10(9)/L) and lower ARC (ARC < 40 × 10(9)/L) have higher risk into progress to SAA (19.2% vs 1.1%) (p = 0.016). It is concluded that NSAA patients treated with Stanozolol progress into SAA with the rate of 5.3%. Those patients with dependent of transfusion or ANC < 0.8 × 10(9)/L or/and ARC < 40 × 10(9)/L have higher risk of progress into SAA.

Conservative management of canine tracheal collapse with stanozolol: a double blinded, placebo control clinical trial.

The objective of this study is to determine the efficacy of stanozolol in the treatment of tracheal collapse (TC) in dogs, which is the analogous disease to tracheomalacia (TM) in humans. Twenty-two dogs with endoscopically confirmed and graded TC were enrolled into five groups. Groups S1 (n=5), S2 (n=5) and S3 (n=4) with grade 1, 2 and 3 TC, respectively, received stanozolol orally for 75 days, while groups P1 (n=4) and P2 (n=4) with grade 1 and 2 TC, respectively, received placebo. The clinical score was evaluated every 15 days, whereas TC grade was reassessed at the end of the experiment. Clinical improvement was detected from the 30th day in S2 and S3 group dogs and from the 45th day in S1 group dogs and continued until the end of the experiment. Also, statistically significant differences were seen between S2 and P2 dogs from the 30th day, and between S1 and P1 dogs from the 60th day, and continued until the end of the study. Amelioration of the TC grade was seen in 13 of 14 (92.9%) dogs, which received stanozolol. Of the 14 dogs, 57.1% were cured and 35.8% demonstrated a less severe TC grade, while only one dog (7.1%) did not improve at all. Stanozolol seems to be an effective drug in the management of canine TC and it may have potential for use in humans with TM.

Stanozolol treatment for successful prevention of attacks of severe primary cryofibrinogenemia.

Severe essential cryofibrinogenemia is rare in childhood, and both the diagnosis and the management are challenging for pediatricians. An 11-year-old male, who had already lost two digits following cold exposure, was referred after multiple visits to various hospitals and subsequently diagnosed as primary cryofibrinogenemia. His history revealed unresponsiveness to calcium channel blockers, acetyl salicylic acid, pentoxifylline, dextran, and steroids. Stanozolol (2 mg/day, orally) prophylaxis was initiated and no new skin lesions developed following starting this treatment. Some of the newly formed lesions at the onset of stanozolol healed.

Acute lipodermatosclerosis: an open clinical trial of stanozolol in patients unable to sustain compression therapy.

Acute lipodermatosclerosis (ALDS) is a painful counterpart of chronic lipodermatosclerosis (LDS) resulting from venous insufficiency. Severe pain is the leading symptom and many patients with ALDS are unable to tolerate compression therapy. We evaluated clinical efficacy of stanozolol (2 mg bid for 8 weeks) for the reduction of pain and dermal-thickness in 17 patients with ALDS. All patients had superficial venous insufficiency documented by duplex scans, and in all of them incompetent perforators were found under the area of ALDS. Mean pain scores prior to institution of stanozolol (7+/-2; range 4-10) lowered significantly at the end of the 8-week treatment (3+/-2; range 0-5, p<0.001). Dermal thickness was also significantly reduced (p<0.01) over the treatment period. Side effects were not noted. Stanozolol, given over 8 weeks, effectively and safely alleviates pain and reduces dermal thickness in patients with ALDS.

[Effect of treatment for myelodysplastic syndrome by Qinghuang Powder combined with Chinese herbs for reinforcing shen and strenghening pi].

OBJECTIVE: To observe the clinical efficacy of Qinghuang Powder combined with Chinese herbs for reinforcing Shen and strenghening Pi in treating myelodysplastic syndrome (MDS). METHODS: fifty-five patients with diagnosis fitting to MDS were treated with Qinghuang Powder and decoction for strengthening Pi and reinforcing Shen, in combination with Stanozololum. RESULTS: Eleven patients (20.0%) out of the 55 were completely remitted (CR), the total effective rate being 74.5% (41/55 cases). By FAB typing, 9 (26. 5%) in the 34 patients of type RA/RAS were CR, with the total effective rate of 82.4% (28/34 cases), and in the 21 patients of type RAEB, 9.5% (2/21 cases) were CR with the total effective rate of 61.9% (13/21 cases), showing insignificant statistical difference between the two types (P > 0.05). By international prognostic scoring system (IPSS), the treatment was evaluated as CR in 10 patients, effective in 25 and ineffective in 11 in 36 patients of moderate risk group I , the responding numbers were 1, 4, 2 in 7 patients of moderate risk group II and 0, 3, 3 in 6 patients of high risk group, respectively, also showing insignificant difference between groups (P > 0.05). Levels of Hb, WBC and platelet significantly increased after treatment (P < 0. 05). By cytogenetics, the effective rate was 68.8% (11/16 cases) in patients with abnormal chromosome and 72.7% (24/33 cases) in those with normal chromosome, with insignificant difference (P > 0.05). CONCLUSION: The comprehensive therapy with TCM treatment for reinforcing Shen and dissolving stasis in dominance has a definite clinical effect in treating MDS, it was not significantly associated with FAB typing, IPSS score, and chromosome abnormality of patients.