Articles: disease.
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Mayo Clinic proceedings · Feb 2024
ReviewClinical Trials for Special Populations: Children, Older Adults, and Rare Diseases.
Research cannot maximize population health unless it improves health for all members of the public, including special populations such as children, older adults, and people living with rare diseases. Each of these categories require special considerations when planning and performing clinical trials, and common threads of ethical conduct of research in vulnerable populations appear throughout. In this review, definitions of each of the three categories of special population (children, older adults, and rare diseases) are discussed in terms of US research regulations, the unique challenges to conducting clinical trials for these special populations, critical ethical issues, and opportunities for innovative ways to design and operationalize clinical trials in special populations. Additional critical attention is focused on factors that influence the generalizability of study results to reduce health disparities, as well as the importance of community engagement and advocacy groups that can help to educate potential trial participants of the benefits of clinical trial participation.
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Dtsch. Med. Wochenschr. · Feb 2024
Review[Compression therapy for inflammatory dermatoses of the legs].
In addition to venous and lymphatic diseases, there is increasing scientific evidence that inflammatory dermatoses of the legs are also indications for compression therapy. Specifically, diseases such as pyoderma gangrenosum, livedoid vasculopathy, cutaneous vasculitides, necrobiosis lipoidica, psoriasis, or erysipelas are conditions for which adjunctive compression therapy may be used when manifestations occur on the lower extremities. ⋯ Especially because of the often problematic pain symptoms, compression therapy can be performed with low resting pressures around 20 mmHg, especially in inflammatory dermatoses. In this review article, the current scientific aspects of compression therapy in inflammatory dermatoses of the legs and the corresponding limitations are presented in a differentiated manner.
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Comparative Study
Comparison among populations with severe and intermediate alpha1-antitrypsin deficiency and chronic obstructive pulmonary disease.
Severe alpha1-antitrypsin (AAT) deficiency (AATD) is associated with a high risk of airflow obstruction and emphysema. The risk of lung disease in those with intermediate AAT deficiency is unclear. Our aims were to compare pulmonary function, time of onset of symptoms, and indicators of quality of life among patients with severe AATD (PI*ZZ), patients with intermediate AATD (PI*MZ) from the Italian Registry of AATD with a chronic obstructive pulmonary disease (COPD) cohort of patients without AATD (PI*MM). ⋯ The comparison of populations with PI*ZZ, MZ and MM genotypes allows to delineate the role of alpha1-antitrypsin deficiency on respiratory function and on the impact on quality of life, in relation to other risk factors. These results highlight the crucial role of primary and secondary prevention on smoking habits in PI*MZ subjects and the importance of an early diagnosis.
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There is concern that sodium-glucose cotransporter-2 inhibitors during hospitalization for acute heart failure (aHF) may precipitate diabetic ketoacidosis (DKA). A retrospective study of all hospitalization encounters for aHF defined by a primary HF International Classification of Diseases (ICD)-10 code in 15 Kaiser Permanente Southern California medical centers hospitalized between January 1, 2021 and August 31, 2023 was performed to describe rates of DKA with empagliflozin use. DKA was defined by the presence of either a DKA ICD-10 code or ketoacidosis lab criteria (bicarbonate <18 mmol/L and urine ketone 1+ or more or elevated serum beta-hydroxybutyrate within 12 h) during hospitalization. ⋯ There were 2 (0.1%) probable DKA cases in empagliflozin encounters and 15 (0.1%) in nonexposed encounters. These rates were similar when stratified by diabetes status and ejection fraction. Empagliflozin may be safe during aHF hospitalization.
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Randomized Controlled Trial
Trial of N-Acetyl-l-Leucine in Niemann-Pick Disease Type C.
Niemann-Pick disease type C is a rare lysosomal storage disorder. We evaluated the safety and efficacy of N-acetyl-l-leucine (NALL), an agent that potentially ameliorates lysosomal and metabolic dysfunction, for the treatment of Niemann-Pick disease type C. ⋯ Among patients with Niemann-Pick disease type C, treatment with NALL for 12 weeks led to better neurologic status than placebo. A longer period is needed to determine the long-term effects of this agent in patients with Niemann-Pick disease type C. (Funded by IntraBio; ClinicalTrials.gov number, NCT05163288; EudraCT number, 2021-005356-10.).