MDS Transplant Outcomes

For some people with myelodysplastic syndromes (MDS), a bone marrow, peripheral blood or cord blood transplant (also called a BMT) may offer the best chance for long-term remission of their disease. This page provides some data on patients' outcomes after a transplant to treat MDS. For a more complete overview of MDS and how it may be treated, see Myelodysplastic Syndromes (MDS).

The National Marrow Donor Program^® (NMDP) and its research arm, the Center for International Blood and Marrow Transplant Research^® (CIBMTR) collect and study data to learn how to improve patient outcomes. The NMDP also operates Be The Match^®.

Allogeneic transplant outcomes

The standard transplant for MDS is allogeneic, which uses blood-forming cells from a family member, an unrelated donor or a cord blood unit. For allogeneic transplants, the blood-forming cells can be collected from the donor’s marrow or from the bloodstream (peripheral blood stem cells, or PBSC). The figures below showing data from the NMDP state whether patients received marrow or PBSC.

Though the outcomes for marrow and PBSC transplants may appear different below, there could be many reasons for this. For example, the patient groups may not have the same risk factors (such as age or past treatments). Doctors are still trying to find out whether one works better than the other.

Figure 1.
Probability of Survival after Allogeneic Transplants for Myelodysplastic Syndromes, 2000-2010, by disease status and donor type. (CIBMTR data)

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Figure 2.
Myelodysplastic syndromes: Survival of adult (age ≥18 years) marrow recipients, by WHO disease classification, unrelated donor transplants facilitated by the NMDP, 2000-2009. (NMDP data)

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Figure 3.
Myelodysplastic syndromes: Survival of adult (age ≥18 years) PBSC recipients, by WHO disease classification, unrelated donor transplants facilitated by the NMDP, 2000-2009.  (NMDP data)

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Figure 4.
Myelodysplastic syndromes: Survival of adult (age ≥18 years) marrow and PBSC recipients, by age at transplant, unrelated donor transplants facilitated by the NMDP, 2000-2009. (NMDP data)

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Factors that affect allogeneic transplant outcomes

Two major risks for people with MDS who are treated with transplant are complications of the transplant and relapse of the disease. Advances in transplant are reducing life-threatening complications of transplant. These advances include changes in the preparative regimen used to prepare patients for transplant and improved matching of donors to patients. Some important factors that can affect transplant survival are discussed below.

The patient's age

In general, younger patients have a better chance of survival than older patients. Figures 1 and 2 show much higher survival rates for children (age 20 or younger). However, most patients with MDS are adults over age 60. The risks of MDS and the risks of transplant both tend to increase with a person's age. However, a person's overall health and other factors are also important and a transplant can offer some older adults a chance at long-term survival. For example, one small study that focused on people age 55 to 66 showed an estimated 3-year survival without relapse of 33% to 53%, depending on the type of MDS. [1]

The type of MDS and the IPSS risk score

In general (and as shown above in Figures 3 and 4), people with refractory anemia (RA) or refractory anemia with ringed sideroblasts (RARS) have a better chance of long-term survival than those with refractory anemia with excess blasts (RAEB1/RAEB2). (For an explanation of MDS types and risk scores, see Myelodysplastic Syndromes.)

Although IPSS risk score data were not available for the figures above, other studies have shown that risk score can help predict the chances of long-term survival after transplant for MDS. People with a low or intermediate-1 risk score tend to have a better chance of long-term survival than people with an intermediate-2 or high risk score. In one study of 109 patients, after transplant the estimated 3-year survival without relapse ranged from 80% for patients with a low risk score to 29% for those with a high risk score. [2]

The closeness of the donor match

In general, a matched sibling donor is the optimal match and can give a better chance of survival than an unrelated donor. However, because of advances in unrelated donor transplant, some recent studies show similar outcomes for transplants using sibling donors and transplants using unrelated donors, especially for patients with early disease status (Figures 1-2). For example, in the study of 109 patients described above, the estimated 3-year survival without relapse was 56% for patients who had sibling donors and 59% for those who had unrelated donors. [2]

Timing of the transplant

Some studies have shown better outcomes for transplants done soon after diagnosis than those done later. One multi-center study of patients who received a transplant using a matched sibling donor between 1990 and 1999 focused on the timing of transplant for MDS. It found that for patients with low and intermediate-1 risk scores, waiting and transplanting only when the disease progressed was the strategy that maximized survival. However, it was important to receive a transplant before AML developed. For patients with intermediate-2 and high-risk scores, the study showed that a transplant soon after diagnosis gave a better chance of a longer life. [3]

The preparative regimen — high-dose or reduced-intensity

The preparative regimen is treatment to prepare a person for transplant. The high-dose preparative regimen (myeloablative conditioning) uses high-dose chemotherapy with or without radiation therapy. This regimen is very intense. Many patients with MDS are older and have other health problems that may make them unable to tolerate this regimen. However, they may be able to tolerate a reduced-intensity regimen (non-myeloablative), which uses lower doses of chemotherapy and low-dose or no radiation therapy.

Reduced-intensity transplant is a newer approach to transplant for MDS, and its use is growing. A reduced-intensity transplant may offer some patients with MDS the chance for long-term survival. A recent study included 62 patients who received a reduced-intensity transplant. [4] In these patients, the rate of early complications was lower than that seen after high-dose transplants, showing that the reduced-intensity regimen was better tolerated. At one year the rate of survival without relapse was 61% for patients who had a sibling donor and 59% for those who had an unrelated donor. [4] These early results appear promising when compared to those for high-dose (myeloablative) transplants. [1, 2] Longer follow-up is needed to find out how survival rates and the risk of MDS relapse after reduced-intensity transplants compare to those after high-dose transplants over the long term.

Autologous transplant outcomes

The standard transplant for MDS is allogeneic, which uses blood-forming cells from a family member, an unrelated donor or a cord blood unit. Another type of transplant is an autologous transplant, which uses the patient's own blood-forming cells. An autologous transplant is a standard treatment for some diseases and is being studied in clinical trials as a treatment for MDS. An autologous transplant may be an option for patients who do not have a suitable donor for an allogeneic transplant.

An autologous transplant has a lower risk of complications than an allogeneic transplant. However, the risk of disease relapse may be higher. To be able to undergo an autologous transplant, a person with MDS must:

• Reach a complete remission after chemotherapy
• Produce enough blood-forming cells to collect for a transplant
• Be healthy enough to tolerate a transplant

In one study, treatment with an autologous transplant was planned for 83 patients with high-risk MDS. Forty-two of these patients reached a complete remission after chemotherapy, and 24 of those patients were able to go on to receive an autologous transplant. At the time of the report, 12 of those 24 patients were alive without relapse 8 to 55 months after transplant, and 9 patients had relapsed. [5] Another study included 79 patients who received an autologous transplant to treat MDS or AML that developed out of MDS. The 2-year survival without relapse was 39%, and the rate of relapse was 64%. [6]

Making treatment choices

For more information about myelodysplastic syndrome treatment options and things to think about when making treatment choices, see Myelodysplastic Syndromes (MDS) — Making Treatment Choices.

References

1. Deeg HJ, Shulman HM, Anderson JE, et al. Allogeneic and syngeneic marrow transplantation for myelodysplastic syndrome in patients 55 to 66 years of age. Blood. 2000; 95(4):1188-1194.
   http://www.bloodjournal.org/cgi/content/full/95/4/1188  
2. Deeg HJ, Storer B, Slattery JT, et al. Conditioning with targeted busulfan and cyclophosphamide for hemopoietic stem cell transplantation from related and unrelated donors in patients with myelodysplastic syndrome. Blood. 2002; 100(4):1201-1207.
   http://www.bloodjournal.org/cgi/content/full/100/4/1201  
3. Cutler CS, Lee SJ, Greenberg P, et al. A decision analysis of allogeneic bone marrow transplantation for the myelodysplastic syndromes: delayed transplantation for low-risk myelodysplasia is associated with improved outcome. Blood. 2004; 104(2):579-585.
   http://www.bloodjournal.org/cgi/content/full/104/2/579  
4. Ho AYL, Pagliuca A, Kenyon M, et al. Reduced-intensity allogeneic hematopoietic stem cell transplantation for myelodysplastic syndrome and acute myeloid leukemia with multilineage dysplasia using fludarabine, busulphan, and alemtuzumab (FBC) conditioning. Blood. 2004; 104(6):1616-1623.
   http://www.bloodjournal.org/cgi/content/full/104/6/1616  
5. Wattel E, Solary E, Leleu X, et al. A prospective study of autologous bone marrow or peripheral blood stem cell transplantation after intensive chemotherapy in myelodysplastic syndromes. Leukemia. 1999; 13(4):524-529.
   http://www.nature.com/leu/journal/v13/n4/abs/2401387a.html  
6. De Witte T, Van Biezen A, Hermans J, et al. Autologous bone marrow transplantation for patients with myelodysplastic syndrome (MDS) or acute myeloid leukemia following MDS. Blood. 1997; 90(10):3853-3857.
   http://www.bloodjournal.org/cgi/content/full/90/10/3853  

Contributing Editors

C. F. LeMaistre, M.D., Southwest Texas Methodist Hospital, San Antonio, Texas
Willis Navarro, M.D., National Marrow Donor Program, Minneapolis, Minn.
Paul Shaughnessy, M.D., Texas Transplant Institute, San Antonio, Texas
Anthony S. Stein, M.D., City of Hope National Medical Center, Duarte, Calif.