Androgen Therapy of Aplastic Anaemia ‐A Prospective Study of 352 Cases

Najean, Y; Pecking, A; Danvic, M. Le

doi:10.1111/j.1600-0609.1979.tb00430.x

Cited by 31 publications

(5 citation statements)

References 21 publications

(3 reference statements)

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“…Fanconi’s anemia is rare autosomal recessive disorder, characterized by a high clinical and genetic heterogeneity [14,16–21]. Diagnosis of FA on the basis of clinical features is often difficult and unreliable, due to the possible overlap of the FA phenotype with that of a variety of genetic and non genetic BFM syndromes [2,22].…”

Section: Discussionmentioning

confidence: 99%

Diagnosis of Fanconi's Anemia by Diepoxybutane Analysis in Children from Serbia

Cirkovic¹,

Guć-Šćekić²,

Vujić³

et al. 2011

Balkan Journal of Medical Genetics

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The high sensitivity of Fanconi’s anemia (FA) cells to drug induced DNA interstrand crosslinks (ICL) such as diepoxybutane (DEB) was used as a part of FA screening in the children with clinical suspicion of FA. The study considered a total of 66 children with the hematological and/or congenital phenotypic symptoms reminiscent of FA. Blood samples from patients with clinical suspicion of FA and controls were collected for chromosome fragility evaluation by the DEB test. According to the results of DEB test, the patients were divided into two subgroups: FA displaying typical DEB sensitive cellular response and non FA.In this study, 10 out of 66 patients were found to have a FA cellular phenotype. The percentage of DEB-induced aberrant cells was increased more than 26 times in FA patients (range 22.00–82.00% with a mean of 48.32%) when compared to non FA patients (range 0.00–12.00% with a mean of 1.84%). The number of DEB-induced breaks/cells was more than 68 times higher in FA patients (range 0.26–4.39 with a mean of 1.37 breaks/cell) when compared to non FA patients (range 0.00–0.20 with a mean of 0.02 breaks/cell). The spontaneous chromosome fragility values in FA patients were overlapping those in non FA patients. Our results indicate that the DEB sensitivity test is the most reliable in vitro method for verification of the FA cellular phenotype.

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Section: Discussionmentioning

confidence: 99%

Diagnosis of Fanconi's Anemia by Diepoxybutane Analysis in Children from Serbia

Cirkovic¹,

Guć-Šćekić²,

Vujić³

et al. 2011

Balkan Journal of Medical Genetics

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“…Several case reports have documented the efficacy of danazol in the treatment of AA refractory to IST. Although anabolic steroids are effective in restoring hematopoietic function in a subset of AA patients [11,12], the role of danazol in the treatment of AA has not been studied in a designed clinical trial. Hence, we conducted a prospective study to clarify the efficacy of danazol in the treatment of AA refractory to IST and determined the characteristics of responders to danazol.…”

Section: Introductionmentioning

confidence: 99%

Danazol therapy for aplastic anemia refractory to immunosuppressive therapy

et al. 2007

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Although there are anecdotal reports of the efficacy of danazol in the treatment of aplastic anemia (AA), there has been no systematic study to clarify its efficacy and toxicity. Therefore, we assessed the efficacy of danazol for treatment of patients with AA refractory to immunosuppressive therapy (IST) and those who relapsed after IST, in a prospective clinical trial. Sixteen patients (12 males and four females; six severe cases and 10 moderate cases) were treated with 300 mg of danazol daily for 12 weeks. All patients completed the treatment period without occurrence of severe toxicity. Three female patients achieved partial remission, whereas only two of the 12 male patients did so. None of the responders had shown a response to previous IST or an increase in the percentage of paroxysmal nocturnal hemoglobinuria (PNH)-type cells which are known to be a marker for a good response to IST. These findings indicate that danazol is effective for a subset of AA patients, and particularly for female patients with AA refractory to IST. Am. J. Hematol. 83:387-389, 2008. V

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“…It is no longer used medicinally but is still available for veterinary use. Norethandrolone ( 20 , Figure 4 ) has similar properties and is also used to treat muscle wasting [ 52 ], severe burns [ 53 ], and aplastic anemia [ 54 ]. Its medicinal use has largely been discontinued, though it is still used in some countries [ 53 ].…”

Section: Available Testosterone Analoguesmentioning

confidence: 99%

Medicinal Use of Testosterone and Related Steroids Revisited

et al. 2021

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Testosterone derivatives and related compounds (such as anabolic-androgenic steroids—AAS) are frequently misused by athletes (both professional and amateur) wishing to promote muscle development and strength or to cover AAS misuse. Even though these agents are vastly regarded as abusive material, they have important pharmacological activities that cannot be easily replaced by other drugs and have therapeutic potential in a range of conditions (e.g., wasting syndromes, severe burns, muscle and bone injuries, anemia, hereditary angioedema). Testosterone and related steroids have been in some countries treated as controlled substances, which may affect the availability of these agents for patients who need them for therapeutic reasons in a given country. Although these agents are currently regarded as rather older generation drugs and their use may lead to serious side-effects, they still have medicinal value as androgenic, anabolic, and even anti-androgenic agents. This review summarizes and revisits the medicinal use of compounds based on the structure and biological activity of testosterone, with examples of specific compounds. Additionally, some of the newer androgenic-anabolic compounds are discussed such as selective androgen receptor modulators, the efficacy/adverse-effect profiles of which have not been sufficiently established and which may pose a greater risk than conventional androgenic-anabolic agents.

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Androgen Therapy of Aplastic Anaemia ‐A Prospective Study of 352 Cases

Cited by 31 publications

References 21 publications

Diagnosis of Fanconi's Anemia by Diepoxybutane Analysis in Children from Serbia

Diagnosis of Fanconi's Anemia by Diepoxybutane Analysis in Children from Serbia

Danazol therapy for aplastic anemia refractory to immunosuppressive therapy

Medicinal Use of Testosterone and Related Steroids Revisited

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