Introduction

Pomalidomide (trade name EU: Imnovid, U.S.: Pomalyst) has been approved in Germany since May 2019 for the treatment of multiple myeloma in adults who have already had at least one treatment (with lenalidomide or another medication). It is used in combination with the drugs bortezomib and dexamethasone.

Multiple myeloma is a rare type of cancer in which mutated plasma cells multiply uncontrollably in the bone marrow. If the disease progresses, it destroys bone tissue and prevents normal blood formation. This can cause bone pain and bone fractures, as well as anemia. Multiple myeloma also weakens the immune system and is a life-threatening disease.

The drug aims to inhibit the growth of the cancer cells.

Application

Pomalidomide is available in capsules, in four different doses: 1, 2, 3 and 4 mg. The treatment follows a 21-day cycle: For the first 14 days, a 4 mg capsule of pomalidomide is taken once a day. Then there's a 7-day break where the medication isn't used. The dose can then be adjusted for the rest of the treatment. This treatment is stopped if severe side effects occur or if the disease gets worse despite treatment.

Other treatments

The following treatment options are available for adults with multiple myeloma who have already had at least one treatment (with lenalidomide or another drug):

  • Bortezomib in combination with doxorubicin or dexamethasone
  • Lenalidomide in combination with dexamethasone
  • Elotuzumab in combination with lenalidomide and dexamethasone
  • Carfilzomib in combination with lenalidomide and dexamethasone, or with dexamethasone only
  • Daratumumab in combination with lenalidomide and dexamethasone or with bortezomib and dexamethasone

Assessment

In 2019, the Institute for Quality and Efficiency in Health Care (IQWiG, Germany) looked into the advantages and disadvantages of pomalidomide in combination with bortezomib and dexamethasone compared to the standard treatments for multiple myeloma.

The manufacturer provided one study involving 559 people. In this study, one half of the people were given pomalidomide with bortezomib and dexamethasone, and the other half had treatment with only bortezomib and dexamethasone. Everyone in the study had already had one to three treatments (including lenalidomide). This is what was found:

What are the advantages of pomalidomide?

Overall health: For this aspect of health-related quality of life, the participants were asked how they would evaluate their health and quality of life as a whole. The study suggests that pomalidomide only has an advantage for people with an unfavorable prognosis.

There was no advantage for people with a more favorable prognosis, though.

What are the disadvantages of pomalidomide?

The study suggests that pomalidomide has a disadvantage in terms of severe side effects: Nearly 93 out of 100 people who had treatment with pomalidomide experienced severe side effects, compared to about 72 out of 100 people who had treatment with bortezomib and dexamethasone. The severe side effects also occurred later with this combination of drugs. They included venous thromboembolism and neutropenia. In neutropenia, the number of the white blood cells in the body is so low that it increases the risk of a serious infection.

Social functioning: In this aspect of health-related quality of life, the study suggests that there is a disadvantage for people who used pomalidomide and had only had one previous treatment. Compared to people who had treatment with only bortezomib and dexamethasone, they felt more restricted in their family life, activities with friends and other social activities.

There was no disadvantage for people who had already had more than one previous treatment, though.

No difference

Life expectancy: Based on initial estimates, 41 to 45 out of 100 people passed away in each group.

Health-related quality of life: There was no difference between the two treatments in many areas. These included physical activities of everyday life (physical functioning), restrictions affecting activities in their free time or at work (role functioning), as well as other aspects like concentration or memory (cognitive function) and irritability or feeling down (emotional functioning). There was also no difference in people's perspectives on the future or in their own body image.

No difference was found regarding the following aspects, either:

  • Exhaustion
  • Nausea, vomiting and diarrhea
  • Pain
  • Breathing problems
  • Sleep problems
  • Loss of appetite
  • Disease-related symptoms
  • Side effects
  • Treatments stopped due to side effects
  • Disorders of the peripheral nervous system

Constipation: There was no relevant difference here either.

More information

This information summarizes the main results of a review produced by the Institute for Quality and Efficiency in Health Care (IQWiG, Germany). The review was commissioned by the German Federal Joint Committee (G-BA) as part of the “early benefit assessment of medications.” On the basis of the review and the hearings received, the G-BA passed a resolution on the added benefit of pomalidomide (EU: Imnovid, U.S.: Pomalyst).