Advances in Transplant: Helping More Patients

Tens of thousands of patients have received bone marrow or cord blood transplants (also called BMT). As more patients receive transplants, research studies and experience lead to better care for transplant patients. Doctors also know more about which patients are likely to do well after transplants. Because of these advances, today more patients can be treated with transplants and more patients have better results from their transplants.

More cell sources for transplant

For more than 30 years, doctors have done transplants using the blood-forming cells found in bone marrow. Today, doctors are also doing transplants using two other sources of these blood-forming cells.

Peripheral blood

To move the blood-forming cells from the marrow into the bloodstream (peripheral blood), a person can be given a shot of a growth factor — a drug that helps the body make more white blood cells. Then the cells can be taken from the blood and used for transplant. (To learn more about how these cells are collected, see Peripheral Blood Stem Cell (PBSC) Donation.)

Doctors now almost always use PBSC for autologous transplants (which use the patient's own cells). They also use PBSC more often than marrow for adult patients who receive allogeneic transplants (which use cells from a family member, unrelated donor or cord blood unit). One reason doctors choose PBSC is that the cells usually take less time to engraft (begin making healthy new blood cells). In 2009, 75 percent of adult donors - more than 2,800 - provided a PBSC donation to help a patient in need of a transplant. The time to engraftment is important because patients are at a higher risk for infections until the cells engraft.

Umbilical cord blood

The NMDP facilitated more than 1,000 umbilical cord blood transplants in 2009. Cord blood transplants grew at a rate of 18 percent, compared to 8 percent for marrow, 10 percent for PBSC and an 11 percent overall increase in the number of transplants. Umbilical cord blood is rich in the blood-forming cells used in transplants. Cord blood is donated when a baby is born. Then the donated cord blood is tested, frozen and stored until someone needs it for a transplant. In particular, cord blood may offer hope to some patients who do not have a suitable adult donor or patients who need a transplant more quickly than an unrelated adult donor can be found. To learn more, see Cord Blood Transplants.

For a more detailed discussion of the sources of blood-forming cells, see the information written for physicians on cell sources for transplant.

Newer ways to prepare patients for transplant

Before transplant, a patient is usually given high dose chemotherapy and/or radiation. This treatment is called a preparative regimen or conditioning regimen. The standard preparative regimen is hard on a patient's body. In the past, patients who were older or less healthy could not go through a transplant. Today, doctors are developing transplants that use less harsh preparative regimens. Although they are not right for all patients, these reduced-intensity transplants may be an option for some patients who cannot tolerate a standard transplant.

Better methods for managing transplant complications

Doctors continue to find better ways to manage the problems many transplant patients face. Two of the most common complications are infection and graft-versus-host disease (GVHD). Both can be life-threatening for some patients. Transplant doctors now use medications to prevent infections and GVHD before they occur (infection and GVHD prophylaxis). Preventing these complications is the best way to help patients avoid serious effects. Even so, some patients still get infections or GVHD. Researchers continue to search for even better treatments.

Better matches between patients and donors

Donors and cord blood units are matched to patients using HLA tissue types. Today labs can test for tissue types at very detailed levels (high resolution). Research has found that when patients and donors are matched at detailed levels, the chances for a successful transplant are better. The likelihood of the patient getting serious GVHD is also lower. Researchers continue to learn more about which details of tissue type are important to match.

In addition, for patients who need an unrelated donor or cord blood unit, the options are expanding. Because HLA tissue types are complex, a large and diverse registry of adult donors and cord blood units is needed to provide more patients with a match. Every search conducted through the Be The Match Registry^®, operated by the National Marrow Donor Program^® (NMDP), provides patients with access to more than 13 million potential donors on the global donor listing. This includes more than 8 million potential donors and more than 100,000 cord blood units on the Be The Match Registry, along with potential donors available through agreements with international cooperative registries. Many patients have a good chance of finding a suitable match. However, even with a registry this large, some patients cannot find a match because of the rarity of their tissue types. The NMDP continues its work to grow and diversify the registry to increase the chances for all patients to find a match.

Ongoing research for better transplant outcomes

Researchers keep working to learn more about the best treatments to improve outcomes for transplant patients. The NMDP conducts and supports research to help more patients get a transplant and to improve transplant results. The NMDP conducts research through its affiliate organizations:

• CIBMTR: The Center for International Blood and Marrow Transplant Research^®. To learn about CIBMTR, visit www.cibmtr.org.
• BMT CTN: The Blood and Marrow Transplant Clinical Trials Network.

Patients helping patients

The medical advances that help transplant patients today were gained with the help of transplant patients of the past. New drugs and treatment approaches are developed through patients' participation in clinical trials (prospective research) and studies of treatment results over time (retrospective research).

As a patient, you will likely have chances to be part of both types of research. You may be asked to think about joining a clinical trial for a new drug or some other part of your treatment. Your doctor may also ask you to allow him or her to share the results of your transplant with the NMDP and the CIBMTR. For either type of research, you will be included only if you agree (give informed consent).

The NMDP and CIBMTR and other researchers publish the results of their studies in medical journals. Doctors then use the information to better treat patients. Being part of a clinical trial may not help you directly, but it is an important way for doctors to gain knowledge that may help other patients in the future.

Resources for your doctor

Resources for doctors are available in the Physician Resources section of this Web site. Some resources you might want to share with your doctor include:

• Advances in Auto & Allo Transplants
• Diseases Treatable by Hematopoietic Cell Transplant
• Changing Trends in Diseases & Patients Treated
• Recommended Timing for Transplant Consultation