Body system/
|
REBLOZYL(n=223) |
Placebo(n=109) |
||
|---|---|---|---|---|
| All Grades n (%) |
Grade 3* n (%) |
All Grades n (%) |
Grade ≥3 n (%) |
|
| Musculoskeletal and connective tissue disorders Bone pain Arthralgia |
44 (20) 43 (19) |
3 (1) 0 (0) |
9 (8) 13 (12) |
0 (0) 0 (0) |
| Infections and infestations Influenza Viral upper respiratory infection |
19 (9) 14 (6) |
0 (0) 1 (0.4) |
6 (6) 2 (2) |
0 (0) 0 (0) |
| Nervous system disorders Headache Dizziness |
58 (26) 25 (11) |
1 (<1) 0 (0) |
26 (24) 5 (5) |
1 (1) 0 (0) |
| General disorders and administration-site conditions Fatigue |
30 (14) | 0 (0) | 14 (13) | 0 (0) |
| Gastrointestinal disorders Abdominal pain† Diarrhea Nausea |
31 (14) 27 (12) 20 (9) |
0 (0) 1 (<1) 0 (0) |
13 (12) 11 (10) 6 (6) |
0 (0) 0 (0) 0 (0) |
| Vascular disorders Hypertension‡ |
18 (8) | 4 (2) | 3 (3) | 0 (0) |
| Metabolism and nutrition disorders Hyperuricemia |
16 (7) | 6 (3) | 0 (0) | 0 (0) |
| Respiratory, thoracic, and mediastinal disorders Cough |
32 (14) | 0 (0) | 12 (11) | 0 (0) |
PATIENT OUTCOMES: SAFETY
REBLOZYL offers a demonstrated safety profile for beta-thalassemia patients1
ADVERSE REACTIONS (>5%) IN PATIENTS WITH BETA-THALASSEMIA RECEIVING REBLOZYL WITH A DIFFERENCE BETWEEN ARMS OF 1% IN THE BELIEVE TRIAL1
*Limited to Grade 3 reactions with the exception of 4 events of Grade 4 hyperuricemia.
†Grouped term includes: Abdominal pain and abdominal pain upper.
‡Grouped term includes: Essential hypertension, hypertension, and hypertensive crisis.
The majority of adverse reactions with REBLOZYL were Grade 1 or 2 (mild to moderate)
- The most common adverse reactions (at least 10% for REBLOZYL and 1% more than placebo) were headache (26%), bone pain (20%), arthralgia (19%), fatigue (14%), cough (14%), abdominal pain (14%), diarrhea (12%), and dizziness (11%)
- Serious adverse reactions occurred in 3.6% of patients on REBLOZYL
- Serious adverse reactions reported in 1% of patients were cerebrovascular accident and deep vein thrombosis
- A fatal adverse reaction occurred in 1 patient treated with REBLOZYL, who died due to an unconfirmed case of acute myeloid leukemia (AML)
Important considerations while treating patients with REBLOZYL
LIVER FUNCTION LABORATORY ABNORMALITIES IN THE BELIEVE TRIAL1
REBLOZYL
|
Placebo
|
|
|---|---|---|
| ALT ≥3 x ULN | 26 (12) | 13 (12) |
| AST ≥3 x ULN | 25 (11) | 5 (5) |
| ALP ≥2 x ULN | 17 (8) | 1 (<1) |
| Total bilirubin ≥2 x ULN | 143 (64) | 51 (47) |
| Direct bilirubin ≥2 x ULN | 13 (6) | 4 (4) |
- In adult patients with transfusion-dependent beta-thalassemia, EMH masses were observed in 3.2% of REBLOZYL-treated patients, with spinal cord compression symptoms due to EMH masses occurring in 1.9% of patients (BELIEVE and REBLOZYL long-term follow-up study)
- In a study of adult patients with non–transfusion-dependent beta-thalassemia, a higher incidence of EMH masses was observed in 6.3% of REBLOZYL-treated patients vs 2% of placebo-treated patients in the double-blind phase of the study, with spinal cord compression due to EMH masses occurring in 1 patient with a prior history of EMH. REBLOZYL is not indicated for use in patients with non–transfusion-dependent beta-thalassemia
- Possible risk factors for the development of EMH masses in patients with beta-thalassemia include history of EMH masses, splenectomy, splenomegaly, hepatomegaly, or low baseline hemoglobin (<8.5 g/dL). Signs and symptoms may vary depending on the anatomical location. Monitor patients with beta-thalassemia at initiation and during treatment for symptoms and signs or complications resulting from the EMH masses and treat according to clinical guidelines. Discontinue treatment with REBLOZYL in case of serious complications due to EMH masses. Avoid use of REBLOZYL in patients requiring treatment to control the growth of EMH masses
- REBLOZYL was detected in milk of lactating rats. When a drug is present in animal milk, it is likely that the drug will be present in human milk. There are no data on the presence of REBLOZYL in human milk, the effects on the breastfed child, or the effects on milk production. Because of the potential for serious adverse reactions in the breastfed child, advise patients that breastfeeding is not recommended during treatment with REBLOZYL, and for 3 months after the last dose
Permanent discontinuations due to an adverse reaction (Grade 1-4)
5.4%
(n=12/223)
Most frequent adverse reactions requiring permanent discontinuation in patients who received REBLOZYL included arthralgia (1%), back pain (1%), bone pain (<1%), and headache (<1%).
Dose reductions due to adverse reaction
2.7%
(n=6/223)
Most frequent adverse reactions requiring dosage reduction in >0.5% of patients who received REBLOZYL included hypertension and headache.
Dose interruptions due to an adverse reaction
15.2%
(n=34/223)
Most frequent adverse reactions requiring dosage interruption in >1% of patients who received REBLOZYL included upper respiratory tract infection, ALT increase, and cough.
ALP=alkaline phosphatase; ALT=alanine aminotransferase; AST=aspartate aminotransferase; EMH=extramedullary hematopoietic; ULN=upper limit of normal.
Review how to dose REBLOZYL to achieve response
References: 1. REBLOZYL [US Prescribing Information]. Summit, NJ: Celgene Corporation; 2026. 2. Viprakasit V, Taher AT, Hermine O, et al. Evaluating luspatercept responders in the phase 3, randomized, double-blind, placebo-controlled BELIEVE trial of luspatercept in adult β-thalassemia patients who require regular red blood cell transfusions. Poster presented at: The 61st Annual Meeting of the American Society of Hematology (ASH); December 7-10, 2019. Orlando, FL, USA. 3. Data on file. Celgene Corporation. Summit, New Jersey. 4. Cappellini MD, Viprakasit V, Taher AT, et al. A phase 3 trial of luspatercept in patients with transfusion-dependent β-thalassemia. N Engl J Med. 2020;382(13):1219-1231.
