Abstract
Thirty-eight patients who met the diagnostic criteria for severe aplastic anemia underwent allogeneic hematopoietic stem cell transplantation (HSCT). The median patient age was 20 years (range, 14-36 years). Twenty-four patients were treatment-naïve, 11 had failed one or more previous courses of immunosuppressive therapy, and 3 had failed a previous HSCT. The conditioning regimen included fludarabine 30 mg/m2/day for 3 days (days -9, -8, and -7) and cyclophosphamide 50 mg/kg/day for 4 days (days -5, -4, -3, and -2). Graft-versus-host disease (GVHD) prophylaxis consisted of cyclosporine and short-course methotrexate. All patients underwent transplantation with unmanipulated bone marrow as the stem cell source. The median total nucleated cell (TNC) dose was 2.43 × 108/kg (range, 0.60-6.7 × 108/ kg). The conditioning regimen was well tolerated, with minimal treatment-related mortality. Engraftment was observed in all patients after transplantation; the median time to engraftment of neutrophils and platelets was 18 and 23 days, respectively. Twenty-five of the 27 patients with available chimeric studies at day 180 maintained donor chimerism. Acute GVHD grade ≥II was diagnosed in 4 patients (11%). Extensive chronic GVHD was observed in 8 patients (25%) who survived beyond day +100, at a median observation time of 43 months. Graft rejection with relapse of aplais was observed in one patient. The overall survival (OS) for the whole group was 79%. A trend toward improved OS was observed in the treatment-naïve patients (83% vs 71%), but this was statistically insignificant (P = .384). The fludarabine-based conditioning regimen used in this study with relatively young cohort of patients was well tolerated, with a low rate of rejection and treatment outcomes comparable to those seen in other, more intense and potentially more toxic conditioning regimens. Our results await validation in a larger study, optimally in a randomized controlled manner.
| Original language | English |
|---|---|
| Pages (from-to) | 717-722 |
| Number of pages | 6 |
| Journal | Biology of Blood and Marrow Transplantation |
| Volume | 17 |
| Issue number | 5 |
| DOIs | |
| Publication status | Published - May 2011 |
| Externally published | Yes |
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Keywords
- Aplastic anemia
- Fludarabine
- Transplantation
ASJC Scopus subject areas
- Hematology
- Transplantation
Cite this
Fludarabine-Based Conditioning Chemotherapy for Allogeneic Hematopoietic Stem Cell Transplantation in Acquired Severe Aplastic Anemia. / Al-Zahrani, Hazzaa; Nassar, Amr; Al-Mohareb, Fahad; Al-Sharif, Fahad; Mohamed, Said; Al-Anazi, Khalid; Patel, Moosa; Rasheed, Walid; Saleh, Abu Jafar Mohammed; Bakr, Mahmoud; Ahmed, Shad; Ibrahim, Khalid; Hussain, Fazal; Elkum, Naser; Elhassan, Tusneem; Nurgat, Zubeir; Chaudhri, Naeem; Aljurf, Mahmoud.
In: Biology of Blood and Marrow Transplantation, Vol. 17, No. 5, 05.2011, p. 717-722.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - Fludarabine-Based Conditioning Chemotherapy for Allogeneic Hematopoietic Stem Cell Transplantation in Acquired Severe Aplastic Anemia
AU - Al-Zahrani, Hazzaa
AU - Nassar, Amr
AU - Al-Mohareb, Fahad
AU - Al-Sharif, Fahad
AU - Mohamed, Said
AU - Al-Anazi, Khalid
AU - Patel, Moosa
AU - Rasheed, Walid
AU - Saleh, Abu Jafar Mohammed
AU - Bakr, Mahmoud
AU - Ahmed, Shad
AU - Ibrahim, Khalid
AU - Hussain, Fazal
AU - Elkum, Naser
AU - Elhassan, Tusneem
AU - Nurgat, Zubeir
AU - Chaudhri, Naeem
AU - Aljurf, Mahmoud
PY - 2011/5
Y1 - 2011/5
N2 - Thirty-eight patients who met the diagnostic criteria for severe aplastic anemia underwent allogeneic hematopoietic stem cell transplantation (HSCT). The median patient age was 20 years (range, 14-36 years). Twenty-four patients were treatment-naïve, 11 had failed one or more previous courses of immunosuppressive therapy, and 3 had failed a previous HSCT. The conditioning regimen included fludarabine 30 mg/m2/day for 3 days (days -9, -8, and -7) and cyclophosphamide 50 mg/kg/day for 4 days (days -5, -4, -3, and -2). Graft-versus-host disease (GVHD) prophylaxis consisted of cyclosporine and short-course methotrexate. All patients underwent transplantation with unmanipulated bone marrow as the stem cell source. The median total nucleated cell (TNC) dose was 2.43 × 108/kg (range, 0.60-6.7 × 108/ kg). The conditioning regimen was well tolerated, with minimal treatment-related mortality. Engraftment was observed in all patients after transplantation; the median time to engraftment of neutrophils and platelets was 18 and 23 days, respectively. Twenty-five of the 27 patients with available chimeric studies at day 180 maintained donor chimerism. Acute GVHD grade ≥II was diagnosed in 4 patients (11%). Extensive chronic GVHD was observed in 8 patients (25%) who survived beyond day +100, at a median observation time of 43 months. Graft rejection with relapse of aplais was observed in one patient. The overall survival (OS) for the whole group was 79%. A trend toward improved OS was observed in the treatment-naïve patients (83% vs 71%), but this was statistically insignificant (P = .384). The fludarabine-based conditioning regimen used in this study with relatively young cohort of patients was well tolerated, with a low rate of rejection and treatment outcomes comparable to those seen in other, more intense and potentially more toxic conditioning regimens. Our results await validation in a larger study, optimally in a randomized controlled manner.
AB - Thirty-eight patients who met the diagnostic criteria for severe aplastic anemia underwent allogeneic hematopoietic stem cell transplantation (HSCT). The median patient age was 20 years (range, 14-36 years). Twenty-four patients were treatment-naïve, 11 had failed one or more previous courses of immunosuppressive therapy, and 3 had failed a previous HSCT. The conditioning regimen included fludarabine 30 mg/m2/day for 3 days (days -9, -8, and -7) and cyclophosphamide 50 mg/kg/day for 4 days (days -5, -4, -3, and -2). Graft-versus-host disease (GVHD) prophylaxis consisted of cyclosporine and short-course methotrexate. All patients underwent transplantation with unmanipulated bone marrow as the stem cell source. The median total nucleated cell (TNC) dose was 2.43 × 108/kg (range, 0.60-6.7 × 108/ kg). The conditioning regimen was well tolerated, with minimal treatment-related mortality. Engraftment was observed in all patients after transplantation; the median time to engraftment of neutrophils and platelets was 18 and 23 days, respectively. Twenty-five of the 27 patients with available chimeric studies at day 180 maintained donor chimerism. Acute GVHD grade ≥II was diagnosed in 4 patients (11%). Extensive chronic GVHD was observed in 8 patients (25%) who survived beyond day +100, at a median observation time of 43 months. Graft rejection with relapse of aplais was observed in one patient. The overall survival (OS) for the whole group was 79%. A trend toward improved OS was observed in the treatment-naïve patients (83% vs 71%), but this was statistically insignificant (P = .384). The fludarabine-based conditioning regimen used in this study with relatively young cohort of patients was well tolerated, with a low rate of rejection and treatment outcomes comparable to those seen in other, more intense and potentially more toxic conditioning regimens. Our results await validation in a larger study, optimally in a randomized controlled manner.
KW - Aplastic anemia
KW - Fludarabine
KW - Transplantation
UR - http://www.scopus.com/inward/record.url?scp=79954627091&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=79954627091&partnerID=8YFLogxK
U2 - 10.1016/j.bbmt.2010.08.013
DO - 10.1016/j.bbmt.2010.08.013
M3 - Article
VL - 17
SP - 717
EP - 722
JO - Biology of Blood and Marrow Transplantation
JF - Biology of Blood and Marrow Transplantation
SN - 1083-8791
IS - 5
ER -