Results of the Phase II BEYOND study announced: Rotexip can improve the quality of life of adults with non-transfusion dependent (NTD) β-thalassemia

Recently, Bristol-Myers Squibb (NYSE: BMY) and Acceleron Pharmaceuticals (NASDAQ: XLRN) jointly announced the first data of the Phase II clinical study BEYOND

For anemia, 77.
1% of patients treated with rotexcept achieved increased hemoglobin (≥1.
0 g/dl), compared to 0% in the placebo group
.
Changes in patient-reported outcomes are also associated with elevated hemoglobin
.
Transfusion-independent β-thalassemia refers to patients with thalassemia who do not need to receive red blood cell transfusions regularly throughout their lives.
Such patients usually only need occasional or frequent blood transfusions within a certain period of time
.
pixabay pixabay β- thalassemia is caused by a defective hemoglobin gene inherited blood disease is the most common autosomal recessive genetic disease, one of the genetic currently limited β- thalassemia-related anemia treatment, main treatment includes Frequent infusion of red blood cells may cause iron overload, which may lead to serious complications such as organ damage
.
Transfusion-independent thalassemia refers to thalassemia that does not require a life-long regular transfusion of red blood cells to survive.
Such patients usually only need occasional or frequent blood transfusions within a certain period of time
.
In the case of tumors


without red blood cell transfusion, 74 of the 96 patients (77.
1%) who received rotexip treatment reached the primary endpoint of the study during the 12-week interval from the 13th to the 24th week.
The average Hb increased by ≥1.
0 g/dL from the baseline.
There were 0 (0%) of 49 patients in the placebo group (P<0.
0001)
.


The most common adverse events that occurred during treatment at any level in ≥5% of patients were bone pain (36.
5% in the rotexip group vs 6.
1% in the placebo group), headache (30.
2% vs 20.
4%) and arthralgia ( 29.
2% vs 14.
3%)
.
Roth Sipp in patients receiving treatment, no malignant tumors or blood clots reported incidents
.
Tumor thrombus

Rotexipu is the world's first and currently the only red blood cell maturation agent used to treat β-thalassemia and low-risk myelodysplastic syndromes and other related anemias
.
It is reported that it has been approved in the European Union, the United States and Canada
.

The chronic anemia and iron overload experienced by patients with transfusion-independent β-thalassemia may lead to a series of clinical complications
.
Therefore, they urgently need treatment options
.






Note: Rotexip has not yet been approved in Mainland China.

References:
REBLOZYL Summary of Product Characteristics.
Accessed May 2021.

REBLOZYL US Prescribing Information.
Accessed May 2021.

REBLOZYL Canada Product Monograph.
Accessed May 2021.

ClinicalTrials.
gov.
A Study to Determine the Efficacy and Safety of Luspatercept in Adults With Non Transfusion Dependent Beta (β)-Thalassemia (BEYOND).
Available at: https:// =2.
Accessed May 2021.

Galanello R, Origa R.
Beta thalassemia.
Orphanet Journal of Rare Diseases.
2010;5(11).
Available at: https://ojrd.
biomedcentral.
com/articles/10.
1186/1750-1172-5-11.
Accessed May 2021 .

Musallam, KM, Rivella, S.
, Vichinsky, E.
, & Rachmilewitz, EA (2013).
Non-transfusion-dependent thalassemias.
Haematologica, 98(6), 833–844.
https://doi.
org/10.
3324 /haematol.
2012.
066845.
Accessed May 2021.
 







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