| Vol. 6, No. 1: January/February 2006 |
Joint recommendations for long-term HCT recipients
A report on the recommended screening and preventative practices for long-term HCT recipients developed by a joint committee of the European Group for Blood and Marrow Transplantation (EBMT), the Center for International Blood and Marrow Transplant Research (CIBMTR), and the American Society for Blood and Marrow Transplantation (ASBMT). The joint committee developed these recommendations to assist health care providers provide long-term care for autologous and allogeneic HCT recipients. Topics covered include screening and preventative practices for infections and secondary malignancies, and rationale and testing for potential complications involving organs, the respiratory system, the endocrine system, the central nervous system, and the oral cavity.
Rizzo JD, et al. Biol Blood Marrow Transplant 2006; 12(2): 138-151. (More)
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ASBMT evidence-based review: HCT in adults with ALL
A systematic evidence-based review by the American Society for Blood and Marrow Transplantation (ASBMT) of the role of hematopoietic cell transplantation in adults with acute lymphoblastic leukemia (ALL). Treatment recommendations based on the evidence are presented and were unanimously agreed upon by a panel of ALL experts. The ASBMT reviews define current medical practice and specify the role of cytotoxic therapy with hematopoietic cell therapy in the treatment of diseases. The reviews also identify areas where evidence is insufficient and where additional research is needed.
Hahn T, et al. Biol Blood Marrow Transplant 2006; 12(1): 1-30. (More)
Editor's note: For additional evidence-based reviews and guidelines, visit the ASBMT Web site. |
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COBLT Report: Cord blood transplantation for storage diseases
This phase II Cord Blood Transplantation Study (COBLT) report presents the outcomes of 69 patients with lysosomal and peroxisomal storage diseases undergoing cord blood transplantation between 1999-2004. Patients (median age 1.8 yrs, range 0.1-11.7) had mucopolysaccharidoses I to III, mucolipidoses II (n=36), adrenoleukodystrophy (n=8), metachromatic leukodystrophy (n=6), Krabbe disease (n=16), and Tay-Sachs disease (n=3). Patients received a conditioning regimen of oral busulfan, ATG, and cyclophosphamide. Median cell dose was 8.7 ? 107/kg. Median time to neutrophil engraftment was 25 days, with 78% engrafting by day 42. Grade II-IV acute GVHD occurred in 36% of patients and one-year overall survival was 72%. Average time from the onset of a search to the identification of a matched cord blood unit was 15 days, and the authors note that a short search time is "extremely desirable" in these patients to prevent further disease progression. The authors conclude that cord blood transplantation should be considered for young patients diagnosed with lysosomal and peroxisomal storage diseases who lack a matched related bone marrow donor.
Martin PL, et al. Biol Blood Marrow Transplant 2006; 12(2): 184-194. (More) |
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PCR monitoring of BCR-ABL predicts survival in CML patients taking imatinib
A study of real-time quantitative BCR-ABL RT-PCR to predict the duration of continued complete cytogenetic response (CCR) in 85 CML patients treated with imatinib mesylate who achieved a CCR. With a median follow-up of 13 months after CCR (29 months after imatinib) 23 patients (27%) had disease progression (predominantly loss of CCR). At a median follow-up of 13 months after CCR, 42% of patients achieved at least a 2-log decrease from baseline of BCR-ABL mRNA. Patients who did not achieve a 2-log decrease had significantly worse progression-free survival (hazard ratio=5.8; p=0.005). The authors conclude that the achievement of a 2-log molecular response at the time of CCR is a significant and independent prognostic marker of subsequent progression-free survival. The authors also note that because cytogenetic methods lack adequate sensitivity for minimal residual disease detection after CCR, routine serial molecular monitoring would likely improve risk stratification and disease management by identifying patients with the highest risk of relapse.
Press RD, et al. Blood 2006; E pub ahead of print, Feb. 7. (More) |
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Comparable survival after related and unrelated donor transplant in pediatric acute leukemia
This study of hematopoietic cell transplantation in children with acute leukemia (ALL or AML) found no differences in long-term overall survival and disease-free survival (DFS) in children receiving related donors grafts as compared to children receiving unrelated donor grafts. Sources of unrelated donor grafts were marrow (n=85) and cord blood (n=81), and 101 patients received marrow from HLA-matched sibling donors. Three-year disease-free survival in patients transplanted in first complete remission (CR) was 49% (sibling) and 54% (unrelated donor). Overall survival and DFS were significantly higher in children transplanted in first CR compared with those transplanted in second remission or relapse. The authors conclude that unrelated donor transplantation "should be considered for infants with acute leukemia in first CR using the same eligibility criteria as are currently used for those with HLA matched sibling donors."
Eapen M, et al. J Clin Oncol 2006; 24(1): 145-151. (More) |
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Related and unrelated donor HCT for SCID
Severe combined immune deficiency (SCID) patients undergoing matched unrelated donor transplant have better survival than patients who receive mismatched related grafts, according to a study published in JAMA. This retrospective study analyzed the medical records of 94 infants with SCID who received marrow transplants between 1990-2004 at two pediatric referral centers. Donors were identical related (n=13), matched unrelated (n=41), and mismatched related (n=40). Two-year overall survival (OS) was best in patients receiving matched related transplants (92%). Two-year OS of matched unrelated and mismatched related transplant recipients was 81% and 53%, respectively. The survival benefit of matched unrelated donor transplantation over mismatched related transplantation occurred despite the higher incidence of acute GVHD disease in matched unrelated patients (73% vs. 45%; p=0.009).
Grunebaum E, et al. JAMA 2006; 295(5): 508-518. (More) |
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Low-dose, TBI conditioning and HCT for AML patients ineligible for conventional HCT
A study of 122 patients (median age 57.5 years) with acute myeloid leukemia ineligible for myeloablative transplantation due to age or other risk factors who underwent reduced-intensity transplantation using related or unrelated donors. All patients received 2 Gy total-body irradiation on day 0, and 103 received fludarabine (30 mg/m2/d) on days -4 to -2. Ninety-five percent of evaluable patients had sustained engraftment (6 patients (5%) rejected their graft). Fifty-one patients were alive at a median follow-up of 44 months. Two-year overall survival was 48% and disease-free survival was 44%, with no statistically significant differences between related and unrelated donors or between older (>60) and younger (<60) patients. Unrelated donor recipients in second complete remission had lower two-year relapse rates than recipients with advanced disease (34% vs. 63%).
Hegenbart U, et al. J Clin Oncol 2006; 24(3): 444-453. (More) |
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Reduced-intensity conditioning using treosulfan in patients older than 55
This prospective study examined the use of treosulfan in a reduced-intensity conditioning regimen in patients with secondary AML or MDS who were ineligible for standard myeloablative transplantation. Twenty-six patients (median age 60 years) received treosulfan and fludarabine before transplantation with grafts from matched or mismatched unrelated donors (n=20) or HLA-matched siblings (n=6). No patients experienced graft failure. Grade II-III acute GVHD was seen in six patients, and no patients experienced grade IV acute GVHD. The two-year estimated overall and disease-free survival was 36% and 34%, respectively, with no difference in survival between unrelated and related donors.
Kr?ger N, et al. Bone Marrow Transplant 2006; 37(4): 339-344. (More) |
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Review: Treatment of acute lymphoblastic leukemia
A review of the available treatment options for patients with acute lymphoblastic leukemia (ALL). Topics reviewed include risk assessment, factors predicting clinical outcome, clinical features of ALL, early response to treatment, remission induction, intensification (consolidation) therapy, and allogeneic hematopoietic cell transplantation.
Pui CH, et al. N Engl J Med 2006; 354(2): 166-178. (More) |
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Other journal articles of note:
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Advances in Transplantation is an electronic newsletter published six times a year by the Office of Professional Education of the National Marrow Donor Program (NMDP).
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NMDP to host CME audioconference on pediatric ALL
"Pediatric acute lymphoblastic leukemia: Advances in the management of high-risk patients." Wednesday, March 29, 2006, 11:30 a.m. Central Time Presenter: Farid Boulad, M.D. Program will be available online and on CD-ROM following the audioconference. Online registration |
DVD available for transplant patients over 50
Order your free copy of the NMDP DVD and workbook, Transplant as an Option When You are 50 and Older, which can assist patients 50 and older to learn more about the transplant process and how it relates to them. Order DVD |
NMDP symposium at ASH meeting
If you missed the NMDP's ASH Corporate Friday Symposium, Transplantation for the Older Patient, you may still get a copy of the program handout by e-mailing the NMDP at rryan@nmdp.org |
NMDP outcomes data now online at marrow.org/md
View the latest outcomes data on NMDP transplants:
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Survival by disease |
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Sources of cells for transplant |
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Age of transplant patients |
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Treatment-related mortality | More |
Online CME program on MDS
A CME program from the NMDP is now available online:
This program is also available on a free CD-ROM (no CME credit). More | |
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