Comparison of Outcomes after Transplantation of G-CSF–Stimulated Bone Marrow Grafts versus Bone Marrow or Peripheral Blood Grafts from HLA-Matched Sibling Donors for Patients with Severe Aplastic Anemia

Abstract

We compared outcomes of patients with severe aplastic anemia (SAA) who received G-CSF stimulated bone marrow (G-BM) (n=78), unstimulated bone marrow (BM) (n=547), or peripheral blood progenitor cells (PBPC) (n=134) from an HLA-matched sibling. Transplantations occurred in 1997–2003. Rates of neutrophil and platelet recovery were not different among the three treatment groups. Grade 2–4 acute graft-versus-host disease (GVHD) (RR 0.82, p=0.539), grade 3–4 acute GVHD (RR 0.74, p=0.535) and chronic GVHD (RR 1.56, p=0.229) were similar after G-BM and BM transplants. Grade 2–4 acute GVHD (RR 2.37, p=0.012) but not grade 3–4 acute GVHD (RR 1.66, p=0.323) and chronic GVHD (RR 5.09, p<0.001) were higher after PBPC transplants compared to G-BM. Grade 2–4 (RR 2.90, p<0.001), grade 3–4 (RR 2.24, p=0.009) acute GVHD and chronic GVHD (RR 3.26, p<0.001) were higher after PBPC transplants compared to BM. Mortality risks were lower after transplantation of BM compared to G-BM (RR 0.63, p=0.05). These data suggest no advantage to using G-BM and the observed higher rates of acute and chronic GVHD in PBPC recipients warrants cautious use of this graft source for SAA. Taken together, BM is the preferred graft for HLA matched sibling transplants for SAA.

Bone marrow versus peripheral blood as the stem cell source for sibling transplants in acquired aplastic anemia: survival advantage for bone marrow in all age groups

“…3 Grade II-IV and III-IV acute GvHD, and chronic GvHD were significantly more frequent after PB transplants than after BM ones. The mortality rate was lower after transplantation of unstimulated BM than after transplantation of G-CSF-stimulated BM or PB progenitor cells.…”

“…This study concluded that BM is the preferred stem cell source for HLA-matched sibling transplants for severe aplastic anemia. 3 Despite these results, PB is still being used as a source of stem cells for patients with acquired aplastic anemia: a recent survey by the Aplastic Anemia Working Party of the EBMT (WPSAA-EBMT) found that, in 2009, 40% of transplants were from PB (unpublished data). This is difficult to understand, particularly because of the significantly greater risks of both acute and chronic GvHD following PB grafts, 4 which may have short-and long-term consequences on morbidity and mortality, without the potential added value of a graft-versus-leukemia effect.…”

The EBMT activity survey: 1990–2010

“…After it has been shown in observational studies that peripheral blood was associated with more chronic GVHD and poor survival in patients with marrow failure, the trend is now to prefer marrow as a stem cell source for this indication. 14,15 The use of marrow has increased from 48% in 2009 to 59% in 2010 for nonmalignant disorders.…”

Hematopoietic Stem Cell Transplantation for Sickle Cell Disease: Current Practice and Emerging Trends

“…Although chronic GVHD rates are no different, adult patients have had higher mortality after related donor peripheral blood stem cell transplantations for severe aplastic anemia, and may also benefit from marrow as the stem cell source. 35 Haploidentical transplantations (preferably from the mother due to the possibility of tolerance to maternal antigens) are under investigation with CD34 selected products and early success has been reported. 33 With trials continuing to investigate and define outcomes in a controlled manner, a future reality will be the ability to offer HSCT to eligible patients based on a selection algorithm (Figure 1).…”