Adjuvant psychological therapy in long‐term endocrine conditions

Consideration of psychological distress in long‐term endocrine conditions is of vital importance given the prevalence of anxiety and depression in such disorders. Poor mental health can lead to compromised self‐care, higher utilization of health services, lower rates of adherence, reduced quality of life and ultimately poorer outcomes. Adjuvant psychological therapy offers an effective resource to reduce distress in endocrine conditions. While the vast majority of work in this area has focused on psychological screening and intervention in diabetes, identification and recognition of psychological distress are equally important in other endocrinological conditions, with supportive evidence in polycystic ovary syndrome and Addison's disease. Referral pathways and recommendations set out by UK guidelines and the Department of Health mandate requires greater attention across a wider range of long‐term endocrine conditions to facilitate improved quality of life and health outcome.     

Stable long‐term risk of leukaemia in patients with severe congenital neutropenia maintained on G‐CSF therapy

In severe congenital neutropenia (SCN), long‐term therapy with granulocyte colony‐stimulating factor (G‐CSF) has reduced mortality from sepsis, revealing an underlying predisposition to myelodysplastic syndrome and acute myeloid leukaemia (MDS/AML). We have reported the early pattern of evolution to MDS/AML, but the long‐term risk remains uncertain. We updated a prospective study of 374 SCN patients on long‐term G‐CSF enrolled in the Severe Chronic Neutropenia International Registry. Long‐term, the annual risk of MDS/AML attained a plateau (2·3%/year after 10 years). This risk now appears similar to, rather than higher than, the risk of AML in Fanconi anaemia and dyskeratosis congenita.     

Alternate day prednisone therapy in congenital adrenal hyperplasia: adrenal androgen suppression and normal growth

Daily glucocorticoid therapy of children with congenital adrenal hyperplasia (CAH) frequently results in a suboptimal mature height. In contrast, pharmacological doses of prednisone given on alternate days generally allow normal growth in children with autoimmune, hematological, and renal disorders. Moreover, alternate day prednisone therapy suppresses adrenal androgen secretion on both the day on and the day off therapy in patients with systemic lupus erythematosus. We hypothesized that alternate day prednisone therapy might be efficacious in the treatment of CAH. To evaluate this hypothesis, we studied an 11-yr-old girl with salt-losing 21-hydroxylase deficiency and severe asthma treated with alternate day prednisone therapy (20 mg every other day) for over 3 yr. During this period her linear growth was along the 65th percentile, and her bone age paralleled her chronological age. Pubertal development was normal, and she had no signs of androgen or glucocorticoid excess. In keeping with her clinical picture, basal (24-h samples drawn every 60 min) and ovine CRH-stimulated plasma adrenal androgen (dehydroepiandrosterone sulfate and delta 4-androstenedione) concentrations and 24-h urinary 17-ketosteroid excretion were low on both the day on and the day off prednisone. However, her plasma ACTH and 17-hydroxyprogesterone levels were markedly elevated on both days. The adrenal androgen suppression, therefore, appeared independent of the level of ACTH, suggesting different regulation of the zona fasciculata and the zona reticularis. GH secretion, assessed by measurement of plasma GH every 20 min for 48 h, was normal on both the on and off days of prednisone therapy. Therefore, in this girl pharmacological doses of prednisone given on alternate days caused sustained adrenal androgen suppression and allowed normal growth and pubertal development, despite persistently elevated plasma ACTH and 17-hydroxyprogesterone levels.