The risks of stopping multiple myeloma maintenance therapy include quicker disease progression and a shorter survival time. People may stop due to medication side effects, such as tiredness.

Many people with multiple myeloma undergo a stem cell transplant to treat the disease. After the transplant, they begin maintenance therapy to treat the cancer. However, some people may stop maintenance therapy.

This article discusses the risks and reasons for stopping treatment for multiple myeloma. It also examines current and new therapies for people whose treatment has stopped working.

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Stopping treatment can lead to the following:

Shorter overall survival

Researchers in a 2022 study compared the 5-year overall survival of people with multiple myeloma who stopped maintenance therapy after stem cell transplant at a later time with people who stopped it early.

There were 238 participants in the late group. They stopped treatment after 3 years. There were 102 participants in the early group. They stopped treatment before 3 years.

Researchers found the 5-year overall survival rate of the group who stopped maintenance therapy after 3 years was 96%.

The overall survival rate for the group who stopped maintenance therapy before 3 years was 79%.

Shorter progression-free survival

The same study also compared the 5-year progression-free survival rate between people who stopped maintenance therapy after stem cell transplant early and late.

Researchers found the 5-year progression-free survival rate was 80% in the late group and 50% in the early group.

Increased risk of complications

The risk of complications increases with no treatment to prevent or slow progression. Common complications include:

  • hypercalcemia, which is higher blood calcium levels
  • skeletal disease, which can manifest in bone pain and fractures
  • kidney insufficiency, a condition that involves some degree of kidney failure
  • anemia, a deficiency of red blood cells or hemoglobin, which is a protein in red blood cells that carries oxygen
  • infection
  • neuropathy, which is dysfunction in one or more nerves

Reasons people may stop maintenance therapy after a stem cell transplant include:

Side effects

In the 2022 study discussed above, 50% of people who stopped maintenance treatment after stem cell transplant early did so because of side effects from medication. This was by far the most common reason.

Most side effects involved atypical blood counts, particularly neutropenia and febrile neutropenia.

“Neutropenia” denotes a low count of neutrophils, a type of white blood cell that fights infections. “Febrile” refers to the presence of a fever.

The most common side effects unrelated to blood counts were diarrhea and tiredness.

Desire to try alternative treatments

Some people may prefer to try alternative medicine treatments.

Alternative medicine encompasses interventions such as:

While some of these treatments may help reduce symptoms, research has not proven the effectiveness of many of them.

Additionally, some alternative medicine treatments may be unsafe.

The Food and Drug Administration (FDA) also does not regulate dietary supplements for safety and effectiveness.

Treatment stops working

Some people may stop a specific treatment course for multiple myeloma if they feel it no longer benefits them or for other reasons. In this case, a doctor can best advise on the next steps.

When treatment for multiple myeloma stops working, doctors recommend changes in a person’s treatment regimen to prolong survival.

Research from 2021 states that current strategies when treatment stops working include:

  • a change in treatment if it stops working before 6 months
  • use of previous treatment if treatment stops working after 6 months
  • autologous, or self-derived, stem cell transplantation if treatment stops working after 18 months in people not taking maintenance therapy
  • autologous stem cell transplantation if treatment stops working after 36 months in people taking maintenance therapy or enrolled in a clinical trial

The current and newer treatments available for RRMM include:

Current treatment

A 2022 review notes that some cases of multiple myeloma will be refractory to most available treatments. “Refractory” means the treatments either do not reduce signs of the cancer or the cancer relapses (comes back).

For these cases of multiple myeloma, treatment options include:

  • Immunomodulatory agents (IMiDs): This class of drugs modifies the immune response, reduces the blood supply to cancer, and has direct anticancer activity. An example is lenalidomide (Revlimid).
  • Proteasome inhibitors: These drugs help promote the death of multiple myeloma cells. An example is carfilzomib (Kyprolis).
  • Monoclonal antibodies: These drugs attack specific targets on cancer cells and boost the body’s immune response to cancer. An example is elotuzumab (Empliciti).
  • Alkylating agents: These drugs act on DNA in a way that helps prevent cancer cells from multiplying. An example is cyclophosphamide (Cytoxan).

Newer treatment

Enrolling in a clinical trial is a way to access the latest treatments.

Clinical trials are carefully controlled studies that investigate the safety and effectiveness of new drugs or treatments.

However, clinical trials are not right for everyone. A person can discuss whether they would be a good candidate with their doctor.

A 2023 review of therapies notes that cellular and immunologic treatments are at the forefront of new, innovative treatments for multiple myeloma. The three types below show promise:

  • Chimeric antigen receptor T-cell (CAR T-cell) therapy: These drugs target a substance called anti-B cell maturation antigen, which is elevated in people with multiple myeloma. The FDA has approved two products in this category, one of which is ciltacabtagene autoleucel (cilta-cel).
  • Bispecific T-cell engagers (BITEs): These drugs lead to myeloma cell decomposition. The FDA has approved some options for people who previously received at least four therapy lines. One option is teclistamab (Tecvayli).
  • Cereblon E3 ligase modulators (CELMoDs): These drugs help degrade a substance involved in multiple myeloma. Clinical trials are currently studying iberdomide and mezigdomide.

Learn more about treatments for multiple myeloma.

The risks of stopping maintenance therapy after stem cell transplant for multiple myeloma include shorter overall and progression-free survival.

Another risk is the increased likelihood of developing complications, such as anemia and infection.

The most frequent reason for stopping maintenance treatment early is the side effects of medication. When treatment stops working, doctors have various options, such as changing the medication or using a previous medication.

One of the current treatment options for refractory multiple myeloma is a class of drugs called monoclonal antibodies. Newer options include various cellular and immunologic treatments.