Good Clinical Practice of the Italian Society of Thalassemia and Haemoglobinopathies (SITE) for the Management of Endocrine Complications in Patients with Haemoglobinopathies

Abstract

Background: The treatment of endocrinopathies in haemoglobinopathies is a continually expanding research area; therefore, recommendations supporting the appropriateness of treatments are a pressing need for the medical community. Methods: The Management Committee of SITE selected and gathered a multidisciplinary and multi-professional team, including experts in haemoglobinopathies and experts in endocrinopathies, who have been flanked by experts with methodological and organizational expertise, in order to formulate recommendations based on the available scientific evidence integrated by personal clinical experience. The project followed the systematic approach for the production of clinical practice guidelines according to the methodology suggested by the National Center for Clinical Excellence, Quality and Safety of Care (CNEC). Results: Out of 14 topics, 100 clinical questions were addressed, and 206 recommendations were elaborated on. The strength of recommendations, panel agreement, a short general description of the topic, and the interpretation of evidence were reported. Conclusions: Good Practice Recommendations are the final outcome of translational research and allow one to transfer to the daily clinical practice of endocrine complications in haemoglobinopathies.

Beta thalassemia: Looking to the future, addressing unmet needs and challenges

“…It is in this respect that staff education and decision support must be founded on evidence-based standards and guidelines, which summarize updated recommendations aimed to achieve the best outcomes. 21,22 Quality of care also depends on caregivers garnering optimal experience by taking care of a sufficient number of patients in order to encounter a variety of clinical situations that involve different medical disciplines. Patients may, however, be scattered geographically and many cannot benefit from such experiences.…”

Iron overload disorders: Growth and gonadal dysfunction in childhood and adolescence

“…As discussed later, both iron excess and premature chelation with deferoxamine appear to impact on final height. Any nutritional deficits should be corrected, and endocrine disorders such as hypothyroidism and diabetes should be treated 79 …”

“…Recent Italian guidelines provide a framework for therapeutic management in growth deficiency and DP in the iron overload context. 79 Herein is presented an overview, also taking into account the latest guidelines in general endocrinology 63,65,[80][81][82][83][84] (Table 3).…”

“…Any nutritional deficits should be corrected, and endocrine disorders such as hypothyroidism and diabetes should be treated. 79 For growth disorders if GHD is diagnosed, specific treatment with recombinant human growth hormone (rhGH) should be initiated, with gradually increased dosage based on clinical evaluation every 6 months (Table 3). 85,86 Some small trials suggest that a 1-year GH treatment in BTM children may positively impact their growth rate.…”

Special Issue: “Emerging Therapies and Strategies in Thalassemia: Toward a New Era in Management”

“…The last article of this Special Issue is a formal document, entitled “Good Clinical Practice of the Italian Society of Thalassemia and Haemoglobinopathies (SITE) for the Management of Endocrine Complications in Patients with Haemoglobinopathies” [ 6 ]. Endocrine complications are the most frequent and most resource-draining complications in haemoglobinopathies.…”