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SARS-CoV-2 infection in children and an increased risk of thrombotic microangiopathy

SARS-CoV-2 infection in children and an increased risk of thrombotic microangiopathy

While vaccines are starting to become available for the prevention of SARS-Cov-2 infection, it is anticipated that the Coronavirus 2019 (COVID-19) pandemic will continue for at least the next year.

U.S. FDA approved immunecheckpoint inhibitors U.S. FDA approved immunotherapies for cancer U.S. FDA approved immunotherapies for cancer

US FDA approved immune-checkpoint inhibitors updated 08-Dec-2020

Know all the United States Food and Drug Administration (U.S. FDA) approved immune-checkpoint inhibitors. Members of MediPaper can download the free PPT slides summarising the U.S. FDA approved immune-checkpoint inhibitors and other U.S. FDA approved immunotherapies.

data-for-safe-and-effective-use-of-evolocumab-in-paediatric-familial-hypercholesterolaemia

Data for safe and effective use of evolocumab in paediatric familial hypercholesterolaemia

While SARS-CoV2 has been previously reported to exist in lung tissue in a 78-year-old patient who died of unexpected cardiac arrest when ready for discharge after COVID-19 hospitalisation, recently the first evidence of SARS-CoV2 infection in heart muscle cells has been reported in a case from Brazil and which is supported by evidence from other reports.

Keytruda® FDA approved for recurrent and metastatic Merkel cell carcinoma

On 19 December 2018, pembrolizumab (Keytruda®, Merck) received accelerated approval by the FDA for the treatment of recurrent locally advanced or metastatic (R/M) Merkel cell carcinoma (MCC) in adult and paediatric patients.

Calaspargase pegol-mknl (Asparlas®) FDA approved in paediatric and young adult acute lymphoblastic leukaemia (ALL) patients

On 20 December 2018, the FDA approved the asparagine specific enzyme calaspargase pegol-mknl (Asparlas®, Servier) as a component of multi-agent chemotherapeutic regimens for the treatment of paediatric and young adult patients aged 1 month to 21 years with acute lymphoblastic leukaemia (ALL).

TKR inhibitor larotrectinib (Vitrakvi®) FDA approved for cancers with NTRK gene fusion

On 26 November 2018, the FDA granted accelerated approval to the first-in-class tropomyosin receptor-kinase (TRK) inhibitor larotrectinib (Vitrakvi®, Loxo Oncology/Bayer) for the treatment of adult and paediatric patients with solid tumours that are metastatic or not amenable to surgery, harbour the neurotrophic receptor tyrosine kinase (NTRK) gene fusion in the absence of known acquired resistance mutations, and whose cancer progressed following treatment or for whom are no satisfactory other treatment options available.

Emapalumab FDA approved in adult and paediatric patients with primary hemophagocytic lymphohistiocytosis

On 20 November 2018, the FDA approved the interferon-γ neutralising monoclonal antibody emapalumab (Gamifant®, Novimmune) for the treatment of adult and paediatric (newborn and older) patients with refractory, recurrent, or progressive primary haemophagocytic lymphohistiocytosis (HLH) or in patients intolerant to conventional HLH therapy.

Iobenguane I-131 FDA approved for rare adrenal neuroendocrine tumours

On July 30, 2018, the FDA approved iobenguane I-131 (Azedra®, Progenics Pharmaceuticals), a radioactive therapeutic agent, for the treatment of adult and paediatric patients ≥12 years with iobenguane scan positive, locally advanced or metastatic pheochromocytoma or paraganglioma (PPGL) not amenable to surgery and indicated for systemic therapy.

FDA approved pembrolizumab for 3rd-line PMBCL in adult and pediatric patients

 

  • On June 13, 2018, the US FDA has granted accelerated approval to pembrolizumab (KeytrudaⓇ, Merck & Co. Inc.) for the treatment of adult and pediatric patients with primary mediastinal large B-cell lymphoma (PMBCL), relapsed after two or more prior lines of prior therapy.
US FDA approved CAR-t cell BiTE DAR-T cell CD19 Immunotherapies blinatumomab axicabtagene ciloleucel tisagenlecleucel

US FDA approved CAR-T cell and BiTE immunotherapy – Updated 2-May-2018

The two CAR-T cell immunotherapies axicabtagene ciloleucel (Yescarta®) and tisagenlecleucel (Kymriah®) and the BiTE blinatumomab (Blincyto®) have in common that they target CD19 on the cancer cells. Do you know all the US FDA approved CAR-T and BiTE immunotherapies?

Everolimus suspension approved in TSC-associated partial-onset seizures

On April 10, the FDA approved everolimus tablets for oral suspension (Afinitor Disperz®, Novartis) for the adjunctive treatment of adult and pediatric patients ≥2 years with tuberous sclerosis complex (TSC)-associated partial-onset seizures.

FDA granted accelerated approval to blinatumomab for the treatment of adult and paediatric patients with B-cell precursor ALL in first or second complete remission with MRD ≥0.1%

 

FDA approved nilotinib for paediatric patients with newly diagnosed Ph+ CML-CP or Ph+ CML-CP resistant or intolerant to prior TKI

 

  • FDA approved nilotinib (TASIGNA, Novartis Pharmaceuticals Corporation) for pediatric patients 1 year of age or older with newly diagnosed Philadelphia chromosome positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP) or Ph+ CML-CP resistant or intolerant to prior tyrosine-kinase inhibitor (TKI) therapy. More Information.  March 22, 2018

Hydroxyurea FDA approved for the reduction in frequency of painful crises and blood transfusions in paediatric sickle cell anemia patients

On 21 December the FDA granted regular approval to hydroxyurea (Siklos®, Addmedica) for the reduction in frequency of painful crises and the need for blood transfusions in paediatric sickle cell anaemia patients ≥2 years of age with recurrent moderate to severe painful crises.