Remdesivir in adults with severe COVID-19: a randomised, double-blind, placebo-controlled, multicentre trial

Yeming Wang et al published their clinical trial set in Hubei, China showed the use of remdesivir to not have a statistically significant benefit. This is in contrast to the more recent announcement by the National Institute of Allergy and Infectious Diseases (NIAID) stating their study found remdesivir to be effective in reducing the time to recovery in patients with severe COVID-19 symptoms and the FDA has approved the emergency use of this drug  for severe cases. This later study has not been published in a peer review journal but the earlier study by Wang and colleagues can be read in The Lancet.


COVID‐19 pneumonia as a cause of acute chest syndrome in an adult sickle cell patient

Frans Beerkens and colleagues published a case study involving a 21-year-old man with sickle cell disease who developed acute chest syndrome while being infected with COVID-19.  The authors noted that since sickle cell disease patients may be at higher risk of such complications if infected with COVID‐19 and should be monitored accordingly.

 To read the case report in the American Journal of Hematology, click here.

Autologous haematopoietic stem-cell transplantation versus bortezomib–melphalan–prednisone, with or without bortezomib–lenalidomide–dexamethasone consolidation therapy, and lenalidomide maintenance for newly diagnosed multiple myeloma (EMN02/HO95): a multicentre, randomised, open-label, phase 3 study

Michele Cavo  and colleagues published the results of a open-label phase III study showing autologous HSCT to significantly improve median progressive-free survival in patients with newly diagnosed multiple myeloma, compared to chemotherapy.

To read the full study in The Lancet Haematology, click here.

Treatment outcomes following continuous miglustat therapy in patients with Niemann-Pick disease Type C: a final report of the NPC Registry

Marc Patterson et al published their final report on the Neimann-Pick disease Type C Registry.  A total of 472 patients from 22 countries were enrolled in the Registry looking at the long term safety/efficacy of miglustat.

To read the full study in the Orphanet Journal of Rare Diseases, click here.


The natural history of infantile neuroaxonal dystrophy

Fadie Altuame et al analyzed 28 patients with infantile neuroxonal dystrophy (INAD), a  rapidly progressive neurodegenerative disorder of early onset causing premature death.  In their report, speech impairment and loss of gross motor milestones were the earliest signs of the disease.

To read the full study in the Orphanet Journal of Rare Diseases, click here.


Patient and Partner Perspectives on Health-Related Quality of Life in Adrenocortical Carcinoma

Rebecca Steenaard and colleagues report on their focus group interviews involving patients (and their partners) with adrenocortical carcinoma. Adrenocortical carcinoma is a rare cancer with high morbidity and mortality.

To read the full study in the Journal of the Endocrine Society, click here.


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