Treatment Advances for People With Blood Cancer

Newer therapies are emerging at a fairly rapid rate for patients with blood cancers, or hematologic malignancies, such as leukemia, lymphoma, and multiple myeloma.

Woman in lab handling samples
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The below treatment advances might be viewed as small steps, rather than giant leaps forward; however, these therapies may offer survival advantages that can be extremely meaningful to those affected. 

In some instances, emerging therapies might even keep the flame of hope burning—that curative treatment such as bone marrow transplant might eventually be pursued—whereas before, this may not have been an option.

Gains in survival have to be considered along with side effects and toxicity; in these situations, patients typically want to live both as well as they can (quality of life), and for as long as they can (survival).

Recently Approved Therapies


Disease Studied

Comparative Advantage

Inotuzumab ozogamicin (Besponsa)

Relapsed or refractory B-cell ALL

35.8 percent achieved a complete response (vs. only 17.4 percent with standard therapy)

8.0 month median survival time (vs. 4.9 months with standard therapy)

Lenalidomide (Revlimid)

Newly diagnosed multiple myeloma

Maintenance therapy with lenalidomide after transplant reduced mortality rates by 25 percent compared with placebo or observation.

Improved survival without disease progression: 52.8 months with lenalidomide vs. 23.5 months

Daunorubicin and cytarabine liposome for injection (Vyxeos)

Newly diagnosed therapy-related AML (t-AML)


AML with myelodysplasia-related changes (AML-MRC)

Improved survival compared with patients who received separate treatments of daunorubicin and cytarabine (median overall survival 9.56 months vs. 5.95 months).

1. Inotuzumab Ozogamicin (Besponsa) for Acute Lymphocytic Leukemia

About 5,970 new cases of acute lymphocytic leukemia (ALL) were anticipated in the United States in 2017, with about 1,440 deaths in the same year, according to American Cancer Society estimates. Despite improvements in recent decades in the treatment of many different blood cancers, the prognosis for these patients with ALL remains poor.

Allogeneic stem-cell transplantation (bone marrow transplant from a donor) offers the promise, potentially, of a cure for adults with ALL. However, there is a hurdle to be overcome: the low rates of complete remission with current chemotherapy regimens. Stem cell transplantation typically requires that a person has achieved a complete remission of there disease, and unfortunately, that means that relatively few adults with relapsed or refractory B-cell ALL (a disease that has come back, despite treatment) can get to transplantation.

Thus, drug developers have been looking for new tools to target these cancerous cells. Attacking cells that have a marker called CD22 may be one such tool, in the right circumstances. CD22 is a molecule that is made by certain cells in the body and placed by these cells, almost like tags, on the outside of the cell, within the cell membrane. In patients with B-cell ALL, the cancerous cells have this CD22 molecule in about 90 percent of cases—and those are pretty good odds in the business of cancer treatment.

Inotuzumab ozogamicin (Besponsa) is a humanized anti-CD22 monoclonal antibody that is attached to calicheamicin, an agent that can kill targeted cells.

Inotuzumab ozogamicin is called a conjugate because it is an antibody that is attached to, or conjugated with, an agent that can kill cells. The antibody part seeks cells that have the CD22 marker, and the conjugate part destroys the targeted cell.

The FDA approved inotuzumab ozogamicin based on evidence from a clinical trial in which researchers examined the drug’s safety and efficacy compared with an alternative chemotherapy regimen. This trial included 326 patients who had relapsed or refractory B-cell ALL and who had received one or two prior treatments.

According to the FDA, of the 218 evaluated patients, 35.8 percent who received inotuzumab ozogamicin experienced a complete response, for a median 8.0 months; of the patients who received alternative chemotherapy, only 17.4 percent experienced a complete response, for a median 4.9 months. Thus, inotuzumab ozogamicin is an important new treatment option for relapsed or refractory B-cell ALL.

Common side effects of inotuzumab ozogamicin include low levels of platelets (thrombocytopenia), low levels of certain white blood cells (neutropenia, leukopenia), infection, low levels of red blood cells (anemia), fatigue, severe bleeding (hemorrhage), fever (pyrexia), nausea, headache, low levels of white blood cells with fever (febrile neutropenia), liver damage (transaminases and/or gamma-glutamyltransferase increased), abdominal pain, and high levels of bilirubin in the blood (hyperbilirubinemia). For additional safety information, see the complete prescribing information.

2. Lenalidomide (Revlimid) After Transplant in Multiple Myeloma

Maintenance therapy with lenalidomide following autologous hematopoietic stem cell transplantation (bone marrow transplant via self donation) reduced mortality rates by 25 percent compared with placebo or observation among patients with newly diagnosed multiple myeloma, according to results of a recent meta-analysis study.

McCarthy and colleagues analyzed patients’ data from three randomized clinical trials from the United States, France and Italy. The studies included patients with newly diagnosed multiple myeloma who received self-donated (autologous) bone marrow transplant and then 1,208 of them were treated with lenalidomide afterward, while 603 patients received either a placebo or were simply observed, or monitored.

Patients treated with lenalidomide had improved survival, without progression of their disease, compared with those who received placebo or observation (52.8 months vs. 23.5 months). A total of 490 patients died. A significant survival benefit was seen in the lenalidomide group.

A greater proportion of patients in the lenalidomide group experienced a hematologic second primary malignancy and solid tumor second primary malignancy; however, the rates of progression, mortality due to all causes, or mortality as a result of myeloma all were greater in the placebo/observation group.

3. Fixed-Combination Chemotherapy for Acute Myeloid Leukemia

AML is a rapidly progressing cancer that begins in the bone marrow and quickly causes an increased number of white blood cells in the bloodstream. Approximately 21,380 people will be diagnosed with AML this year, and approximately 10,590 patients with AML will die of the disease.

Vyxeos is a fixed-combination of the chemotherapy drugs daunorubicin and cytarabine that may help some patients live longer than if they were to receive the two therapies separately. The FDA approved Vyxeos for the treatment of adults with two types of acute myeloid leukemia (AML):

  • Newly diagnosed therapy-related AML (t-AML), and
  • AML with myelodysplasia-related changes (AML-MRC).

T-AML occurs as a complication of chemotherapy or radiation in about 8 to 10 percent of all patients treated for cancer. On average, it occurs within five years after treatment. AML-MRC is a type of AML that is associated with having a history of certain blood disorders and other key mutations within the leukemia cells. Both patients with t-AML and those with AML-MRC have very low life expectancies.

In a clinical trial, 309 patients with newly diagnosed t-AML or AML-MRC who were randomized to receive Vyxeos or separately administered treatments of daunorubicin and cytarabine, patients who received Vyxeos lived longer than patients who received separate treatments of daunorubicin and cytarabine (median overall survival 9.56 months vs. 5.95 months).

Common side effects included bleeding events (hemorrhage), fever with low white blood cell count (febrile neutropenia), rash, swelling of the tissues (edema), nausea, inflammation of the mucous membranes (mucositis), and other adverse effects including gastrointestinal problems, serious infections and abnormal heart rhythm (arrhythmia).

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