An Observational Study of Blinatumomab Safety and Treatment Practices
Research type
Research Study
Full title
An Observational Study of Blinatumomab Safety and Effectiveness, Utilisation, and Treatment Practices
IRAS ID
236933
Contact name
Richard Kelly
Contact email
Sponsor organisation
Amgen Ltd
Duration of Study in the UK
4 years, 1 months, 7 days
Research summary
An observational study of Blinatumomab(Blincyto) safety and effectiveness, utilisation, and treatment practices.
The primary objective of this study is to characterise the safety of Blinatumomab(Blincyto)in routine clinical practice. Blinatumomab(Blincyto)effectiveness, medication errors, and utilisation; and select healthcare resource use while using Blinatumomab(Blincyto)will also be described. Safety and effectiveness of Blinatumomab(Blincyto)in specified subgroups of patients will also be assessed.What does this summary cover?
This summary describes the main results from one research study. This study involved the collection of data from medical records. Other studies may find different results. Researchers and health authorities look at the results of many studies to decide which medicines may work best and are safe for participants.Amgen has committed to make research results available to the public. This summary has been provided as part of that commitment and should not be used for any other purpose. It should not be considered to make a claim for any product or to guide treatment decisions.
Some information in this summary may be different from the approved labelling for blinatumomab. Your healthcare professional should refer to the full prescribing information for proper use of blinatumomab.
2. Who Sponsored This Study?
Amgen Inc.
One Amgen Center Drive
Thousand Oaks, CA 91320-1799 USA
Phone (United States): +1 805-447-1000Amgen Inc. is the sponsor of the study who made blinatumomab, marketed as Blincyto®, the medicine studied in this study. Amgen would like to thank everyone who participated in this study and feels it is important to share the results of this study.
3. General Information About the Observational Research Study Where and when was the study done?
• This study took place in Austria, Czech Republic, Finland, France, Germany, Greece, Italy, Netherlands, Portugal, Poland, Sweden, Switzerland, and the United Kingdom.
• The study began in March 2017 and ended in March 2024.
• The study was completed as planned.Why and how was the study done?
This was an observational research study, which means researchers collected health information about study participants based on their doctor’s determination of medical care. In order to do this, the researchers reviewed the participants medical records, without any interaction with the participant. It was expected that participation in this study would not impact treatment practice and participants did not receive treatment above and beyond routine clinical practice.Researchers looked at people with acute lymphoblastic leukemia (ALL) who were prescribed blinatumomab to understand more about how blinatumomab was used in the “real world”, outside of the strict clinical trial setting. Blinatumomab is an immunotherapy medicine that is used to treat people with ALL, a type of cancer of the white blood cells. The researchers wanted to know what type of side effects people were having, looking specifically for events which affect the nervous system, an inflammatory response to treatment called “cytokine release syndrome” or “CRS”, and infections which occur in patients with weakened immune systems. They also wanted to see what type of medication errors and how often they happened in day-to-day practice.
4. Who Was Included in This Study?
This study enrolled 279 participants with ALL. Of the 279 participants,15 participants were excluded from the analysis either because there was no record of them receiving blinatumomab, or they didn’t meet the requirement to enter the study. This left 264 participants where a full analysis of their records could be made.125 participants (47%, or about 47 out of 100) were women and 139 participants (53%, or about 53 out of 100) were men. They ranged in age from 17 to 81 years.
• 1 participant (less than 1%, or less than 1 out of 100) was younger than 18 years.
• 87 participants (33%, or about 33 out of 100) were aged between 18 and less than 35 years.
• 82 participants (31% or about 31 out of 100) were aged between 35 and less than 55 years.
• 58 participants (22%, or about 22 out of 100) were aged between 55 and less than 65 years.
• 36 participants (14%, or about 14 out of 100) were older than 65 years old.This study took place at 74 study centres across Austria, Czech Republic, Finland, France, Germany, Greece, Italy, Netherlands, Portugal, Poland, Sweden, Switzerland, and the United Kingdom. The numbers of participants in each country are listed below:
• Austria – 6 participants
• Czech Republic – 15 participants
• Finland – 5 participants
• France – 60 participants
• Germany – 7 participants
• Greece – 18 participants
• Italy – 75 participants
• Netherlands – 19 participants
• Portugal – 12 participants
• Poland – 14 participants
• Sweden – 1 participant
• Switzerland – 28 participants
• United Kingdom– 19 participantsMedical records of participants starting blinatumomab were included in the review. If needed, informed consent was obtained from the participant as per local regulations and laws. The medical records were reviewed following participant consent. They were then reviewed every 4 months, for a minimum of 2 years, or until the participant died, or withdrew consent.
5. Which Medicine Was Studied?
Researchers studied blinatumomab (Blincyto®). This medicine helps the immune system find and destroy cancer cells and is given directly into the vein. The researchers looked at participants who were already taking this medicine, to better understand its safety, and to get an understanding of any medication errors which happened in a real-world setting.6. What Were the Side Effects?
What is a side effect?
A side effect is any unwanted health problem that was detected or observed in the study. Researchers keep track of side effects observed in this study, even if they are not related to the medicine. The table below shows how many participants had side effects during the study.What side effects were seen?
How many participants had serious side effects (out of a total of 264 participants)?
139 participants (53%)How many participants had non-serious side effects (out of a total of 264 participants)?
240 participants (91%)How many participants died from side effects (out of a total of 264 participants)?
20 participants (8%)How many participants stopped taking the study medicine because of side effects (out of a total of 264 participants)?
26 participants (10%)A side effect was serious if, for example, the participant had to stay in the hospital or died because of that side effect.
Of the 20 people who died, 12 participants died due to the underlying leukemia, or progression of the cancer and 6 participants died due to infections. None of these were thought to be related to blinatumomab. 1 patient died due to neurologic problems, and 1 due to lung failure (less than 1%) which was thought to be caused by blinatumomab by treating physician.
The serious side effects reported for 2% or more participants were:
• inflammatory response to treatment (cytokine release syndrome) (10%)
• low white blood cell count (8%)
• fever with low white blood cell count (5%)
• fever (4%)
• infection in the blood (4%)
• neurologic problems (3%)
• cancer of the white blood cells (3%)
• a dangerous drop of blood pressure caused by severe infection (2%).The side effects, regardless of seriousness, reported for 5% or more participants were
• fever (47%)
• headache (18%)
• inflammatory response to treatment (cytokine release syndrome) (15%)
• low white blood cell count (13%)
• fever with low white blood cell count (10%)
• small shakes in the hands (7%)
• a blood test that shows an increase in a liver enzyme called alanine aminotransferase (7%)
• low red blood cell count (6%)
• neurologic problems (6%).No single study can give a complete picture of the benefits and risks of a medicine. Information about other side effects may be available at the websites listed at the end of this summary.
7. What Were the Overall Results of the Study?
How many side effects which affected the nervous system were reported?
Events which affected the nervous system were reported in 110 (42%) of participants. 26 participants who experienced an event which was considered serious (10%) and 1 participant died (less than 1%). 8 participants stopped blinatumomab (3%) because of an adverse event which affected the nervous system. The most reported events affecting the nervous system were headache (18%), small shakes of the hands (7%), and neurologic problems (6%).How many side effects of inflammatory response to treatment (cytokine release syndrome) were reported?
Inflammatory response to treatment (cytokine release syndrome) events were reported in 39 participants (15%). These events were considered serious in 25 participants (10%). No inflammatory response to treatment (cytokine release syndrome) events were fatal or led to the participant stopping blinatumomab.How many infections which occur in people with weakened immune systems were reported?
Infections which occur in people with weakened immune systems were reported in 9 participants (3%). These events were considered serious in 7 participants (3%), and 1 participant died (less than 1%).How many medication errors were reported?
A total of 31 medication errors were reported in 26 participants (10%). Of these 26 participants, 22 participants had 1 medication error, 3 participants had 2 medication errors, and 1 participant had 3 medication errors.The most common type of medication error in the study was administration error (71%), which included operator (the person operating the pump) use error (39%) and pump malfunction (23%) as the most common types of administration error.
Of the 29 medication errors that reached the participant, 5 errors led to overdoses, 12 errors led to underdoses, and 12 errors led to neither overdose nor underdose.
More results may be available at the websites listed at the end of this summary.
8. How Has This Study Helped Participants and Researchers?
What else is important to know about these results?
These results are only for this study, which looked at a group of 264 people with ALL. Not all participants in the study had the same results. The results for any single participant could have been better or worse than the results for their group. Other studies may find different results. These results do not explain how a medicine may work in a single person. Many studies are needed to show the benefits and risks of a medicine that is still being monitored in the real-world setting. This research may help future participants and families by helping doctors understand more about the medicine being studied.9. Are There Plans for Further Studies?
If more observational or clinical research studies are done with regard to this medicine, they may be listed on public websites. You can search for the medicine name: blinatumomab, AMG 103, or Blincyto on the websites below.1. https://gbr01.safelinks.protection.outlook.com/?url=http%3A%2F%2Fwww.clinicaltrials.gov%2F&data=05%7C02%7Cderby.rec%40hra.nhs.uk%7C7c913b494843470816eb08dda9b05d26%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C638853297131708906%7CUnknown%7CTWFpbGZsb3d8eyJFbXB0eU1hcGkiOnRydWUsIlYiOiIwLjAuMDAwMCIsIlAiOiJXaW4zMiIsIkFOIjoiTWFpbCIsIldUIjoyfQ%3D%3D%7C0%7C%7C%7C&sdata=k9mQeGIeMOvJV98ykOjp4AKgNM1K5KXhzpeDICWRNMU%3D&reserved=0
2. https://gbr01.safelinks.protection.outlook.com/?url=http%3A%2F%2Fwww.amgentrials.com%2F&data=05%7C02%7Cderby.rec%40hra.nhs.uk%7C7c913b494843470816eb08dda9b05d26%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C638853297131731893%7CUnknown%7CTWFpbGZsb3d8eyJFbXB0eU1hcGkiOnRydWUsIlYiOiIwLjAuMDAwMCIsIlAiOiJXaW4zMiIsIkFOIjoiTWFpbCIsIldUIjoyfQ%3D%3D%7C0%7C%7C%7C&sdata=sg7iOeF%2F8S8jKme79t6aIWNtTnHbkRNJtnVFjjd1GAk%3D&reserved=0
3. EU Clinical Trial Information System (EU CTIS at euclinicaltrials.eu) 4. HMA-EMA Catalogues (https://gbr01.safelinks.protection.outlook.com/?url=https%3A%2F%2Fcatalogues.ema.europa.eu%2F&data=05%7C02%7Cderby.rec%40hra.nhs.uk%7C7c913b494843470816eb08dda9b05d26%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C638853297131749475%7CUnknown%7CTWFpbGZsb3d8eyJFbXB0eU1hcGkiOnRydWUsIlYiOiIwLjAuMDAwMCIsIlAiOiJXaW4zMiIsIkFOIjoiTWFpbCIsIldUIjoyfQ%3D%3D%7C0%7C%7C%7C&sdata=a0GLQmy%2F6H0Jik4PL2MoU76YG1ujaPizMszYTIGoL24%3D&reserved=0)10. Where Can I Find More Information About This Study?
To find out more about this study, check these websites:• https://gbr01.safelinks.protection.outlook.com/?url=http%3A%2F%2Fwww.clinicaltrials.gov%2F&data=05%7C02%7Cderby.rec%40hra.nhs.uk%7C7c913b494843470816eb08dda9b05d26%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C638853297131769280%7CUnknown%7CTWFpbGZsb3d8eyJFbXB0eU1hcGkiOnRydWUsIlYiOiIwLjAuMDAwMCIsIlAiOiJXaW4zMiIsIkFOIjoiTWFpbCIsIldUIjoyfQ%3D%3D%7C0%7C%7C%7C&sdata=N4va%2B1fzekyTGD52yTcNOCI769L3KHUiCAhOkDptf7g%3D&reserved=0. Use the study identifier NCT03117621
• https://gbr01.safelinks.protection.outlook.com/?url=https%3A%2F%2Ftrack.pstmrk.it%2F3ts%2Fcatalogues.ema.europa.eu%252F%2FNBTI%2FCPi9AQ%2FAQ%2F7cee3e08-f817-4eab-a070-6ba7179c5e3a%2F1%2FeAC4nrPT5S&data=05%7C02%7Cderby.rec%40hra.nhs.uk%7C7c913b494843470816eb08dda9b05d26%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C638853297131789070%7CUnknown%7CTWFpbGZsb3d8eyJFbXB0eU1hcGkiOnRydWUsIlYiOiIwLjAuMDAwMCIsIlAiOiJXaW4zMiIsIkFOIjoiTWFpbCIsIldUIjoyfQ%3D%3D%7C0%7C%7C%7C&sdata=5QpKZapJdnn6nfkHaWb7ym8PDppA57AQrl49mw%2BmdLM%3D&reserved=0 Use the study identifier EUPAS17848If you participated in the study and have questions about the study results, the doctor or staff at your study center may be able to answer them.
REC name
East Midlands - Derby Research Ethics Committee
REC reference
18/EM/0005
Date of REC Opinion
30 Jan 2018
REC opinion
Further Information Favourable Opinion