Patients who underwent hematopoietic stem cell transplantation with bone marrow from unrelated donors reported better 5-year outcomes than those who received peripheral blood, according to long-term follow-up data from a randomized trial.
Bone marrow recipients reported improved psychological well-being and less troublesome graft-versus-host disease (GVHD) symptoms. They also appeared more likely to return to work.
Stephanie J. Lee
More transplants are performed from unrelated donors than related donors, according statistics from the Center for International Blood and Marrow Transplant Research. More than 80% of transplants from unrelated donors use peripheral blood rather than bone marrow.
Results from a large randomized trial published in 2012 showed that both graft sources produced similar survival, nonrelapse mortality and relapse rates. Transplant recipients who received bone marrow had higher rates of graft failure (P = .002), whereas those who received peripheral blood had higher rates of chronic GVHD (P = .01).
Stephanie J. Lee, MD, MPH, PhD, professor of medicine at University of Washington School of Medicine and member of the Fred Hutchinson Cancer Research Center, and colleagues conducted a long-term analysis of patient-reported outcomes from this trial.
Study participants had diagnoses of acute or chronic leukemia, myelodysplasia or myelofibrosis.
The researchers collected preliminary data at the time of enrollment, with follow-up data collected 6 months, 1 year, 2 years and 5 years after transplantation.
Patients completed several validated self-assessments. These included the Functional Assessment of Cancer Therapy–Bone Marrow Transplant, which measures physical, social, emotional and functional domains; the Mental Health Inventory, which measures psychological well-being, psychological distress, anxiety, depression, positive affect, and loss of behavior and personal control; and the Lee Chronic GVHD symptom scale, which measures symptoms in the skin, lungs, eyes and mouth, as well as energy, nutrition and overall psychological status.
Additionally, the researchers collected data on self-assessed performance status, overall health and quality of life.
The investigators collected occupational data through questions about job status, type of work, number of hours of paid and unpaid labor, school attendance, work importance, and changes to work goals.
A total of 195 study participants (bone marrow, n = 102; peripheral blood, n = 93) remained alive 5 years after transplantation. More than half of the participants (53.5%; n = 104) were aged 40 years or older, and men comprised 51.8% (n = 101) of the cohort.
Approximately 70% of study participants completed baseline assessments. At baseline, patients assigned to HSCT with bone marrow reported higher functional well-being (P = .01), total Functional Assessment of Cancer Therapy scores (P = .02) and Health Rating Scale scores (P = .03).
Response rates at predetermined assessment time points during the first 2 years after transplant ranged from 28% to 43%.
Participants in the bone marrow group had higher chronic GVHD skin scores at 6 months (P = .009), whereas participants in the peripheral blood group had higher chronic GVHD mouth scores at 2 years (P = .03). No other differences occurred during the first 2 years.
Factors associated with missing outcomes data at 5 years included younger age and high-risk disease; however, graft source was not associated with missing data (OR = 1.05; 95% CI, 0.52-2.14).
The patient-reported outcome response rate for each instrument ranged between 74% and 78% at the 5-year interval.
Overall mean scores favored the bone marrow group for the Functional Assessment of Cancer Therapy survey (mean and standard deviation, 76.7 [1.6] vs. 70.5 [1.9]; P = .01), the Mental Health Inventory Psychological Well-Being survey (78.9 [1.7] vs. 72.2 [1.9]; P = .01) and Lee Chronic GVHD symptoms scores (13.1 [1.5] vs. 19.3 [1.6]; P = .004).
Seventy-one percent (n = 72) of responding survivors in the bone marrow group had no chronic GVHD at 5 years, compared with 49% (n = 46) of the peripheral blood group. Bone marrow recipients also had lower rates of mild GVHD (17% vs. 23%), moderate GVHD (9% vs. 17%) and severe GVHD (4% vs. 9%; P = .03).
Participants in the bone marrow group had a higher 5-year likelihood of returning to full- or part-time work (OR = 1.5; 95% CI, 1.2-2). Fifty-two percent (n = 42) of 5-year survivors in the bone marrow group reported returning to work, compared with 40% (n = 29) of the peripheral blood group.
Undergoing transplantation with matched unrelated bone marrow correlated with better functional well-being (P = .02), higher health rating scale scores (P = .02) and perceptions of overall quality of life (P = .04).
This cohort also had better GVHD symptom scores in the eyes (P < .001) and lungs (P = .004), as well as higher energy scores (P = .003).
Both cohorts had similar rates of OS (bone marrow vs. peripheral blood, 40% vs. 39%), relapse (32% vs. 29%) and treatment-related mortality (29% vs. 32%).
The researchers acknowledged study limitations, including the overall lack of data from approximately 25% of study participants, as well as the low response rates at 6 months, 1 year and 2 years.
“The failure to see an increase in the proportion of [HSCTs] using bone marrow means that the clinical results published were not compelling enough to change treatment of these patients,” Lee and colleagues wrote. “It remains to be seen whether the additional information provided by this study will be judged sufficient to recommend bone marrow instead of peripheral blood for unrelated donor transplantation when performed for the indications and using the approaches included in this study.” – by Cameron Kelsall
Disclosure: The researchers report no relevant financial disclosures.