scholarly journals Aplastic anemia treated by allogeneic bone marrow transplantation: a report on 49 new cases from Seattle

Blood ◽  
1976 ◽  
Vol 48 (6) ◽  
pp. 817-841 ◽  
Author(s):  
R Storb ◽  
ED Thomas ◽  
PL Weiden ◽  
CD Buckner ◽  
RA Clift ◽  
...  
Keyword(s):  
Aplastic Anemia ◽  
Graft Rejection ◽  
Marrow Transplantation ◽  
Fungal Infections ◽  
Allogeneic Bone Marrow Transplantation ◽  
Severe Aplastic Anemia ◽  
Allogeneic Bone ◽  
Marrow Graft ◽  
Term Survival ◽  
Total Body

Forty-nine patients with severe aplastic anemia, 33 due to unknown cause, 11 drug or chemical related, 2 associated with hepatitis, 1 with paroxysmal nocturnal hemoglobinuria, and 2 possibly associated with Fanconi syndrome did not show recovery after 0.5–96 (median 2) mo of conventional therapy. Twenty-two were infected and 21 were refractory to random platelet transfusions at the time of admission. All were given marrow grafts from HLA-identical siblings. Forty-five were conditioned for grafting by cyclophosphamide (CY), 50 mg/kg on each of 4 successive days, and four by 1000 rad total body irradiation. All were given intermittent methotrexate therapy within the first 100 days of grafting to modify graft-versus-host disease (GVHD). Three patients died from infection too early to evaluate (days 1–8). Forty-six had marrow engraftment. Of these, 20 are surviving with good peripheral blood counts between 186 and 999 days, and 18 have returned to normal activities. Chronic GCHD is a problem in five. Twelve patients died of infection following rejection of the marrow graft. Twelve patients died with bacterial or fungal infections or interstitial pneumonia and active GVHD or soon following resolution of GVHD. Two patients died with marrow engraftment and no GVHD, one with an interstitial, and the other with a bacterial pneumonia. Thirty-six patients who had received random donor blood transfusions were randomly assigned to receive either CY or procarbazine-antithymocyte globulin-CY as conditioning regimens to test whether the incidence of graft rejection could be decreased. There was no difference in the incidence of graft rejection between the two regimens. In 13 patients with rejection, second transplants were attempted either with the original marrow donor (9 patients) or another HLA-identical sibling (4 patients). Three of these transplants were not evaluable, seven were unsuccessful and three were successful with only one of the three surviving for more than 468 days. In conclusion, the long-term survival of 41% of the patients in the present study is similar to that achieved in our first 24 patients, and confirms the importance of marrow transplantation for the treatment of severe aplastic anemia. Marrow graft rejection, GVHD, and infections continue to be the major causes of failure.

scholarly journals Aplastic anemia treated by allogeneic bone marrow transplantation: a report on 49 new cases from Seattle

Blood ◽  
1976 ◽  
Vol 48 (6) ◽  
pp. 817-841 ◽  
Author(s):  
R Storb ◽  
ED Thomas ◽  
PL Weiden ◽  
CD Buckner ◽  
RA Clift ◽  
...  
Keyword(s):  
Aplastic Anemia ◽  
Graft Rejection ◽  
Marrow Transplantation ◽  
Fungal Infections ◽  
Allogeneic Bone Marrow Transplantation ◽  
Severe Aplastic Anemia ◽  
Allogeneic Bone ◽  
Marrow Graft ◽  
Term Survival ◽  
Total Body

Abstract Forty-nine patients with severe aplastic anemia, 33 due to unknown cause, 11 drug or chemical related, 2 associated with hepatitis, 1 with paroxysmal nocturnal hemoglobinuria, and 2 possibly associated with Fanconi syndrome did not show recovery after 0.5–96 (median 2) mo of conventional therapy. Twenty-two were infected and 21 were refractory to random platelet transfusions at the time of admission. All were given marrow grafts from HLA-identical siblings. Forty-five were conditioned for grafting by cyclophosphamide (CY), 50 mg/kg on each of 4 successive days, and four by 1000 rad total body irradiation. All were given intermittent methotrexate therapy within the first 100 days of grafting to modify graft-versus-host disease (GVHD). Three patients died from infection too early to evaluate (days 1–8). Forty-six had marrow engraftment. Of these, 20 are surviving with good peripheral blood counts between 186 and 999 days, and 18 have returned to normal activities. Chronic GCHD is a problem in five. Twelve patients died of infection following rejection of the marrow graft. Twelve patients died with bacterial or fungal infections or interstitial pneumonia and active GVHD or soon following resolution of GVHD. Two patients died with marrow engraftment and no GVHD, one with an interstitial, and the other with a bacterial pneumonia. Thirty-six patients who had received random donor blood transfusions were randomly assigned to receive either CY or procarbazine-antithymocyte globulin-CY as conditioning regimens to test whether the incidence of graft rejection could be decreased. There was no difference in the incidence of graft rejection between the two regimens. In 13 patients with rejection, second transplants were attempted either with the original marrow donor (9 patients) or another HLA-identical sibling (4 patients). Three of these transplants were not evaluable, seven were unsuccessful and three were successful with only one of the three surviving for more than 468 days. In conclusion, the long-term survival of 41% of the patients in the present study is similar to that achieved in our first 24 patients, and confirms the importance of marrow transplantation for the treatment of severe aplastic anemia. Marrow graft rejection, GVHD, and infections continue to be the major causes of failure.

Allogeneic Bone Marrow Transplantation in a Child with Hemophilia A and Severe Aplastic Anemia.

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 5421-5421
Author(s):  
Mauricio Ostronoff ◽  
Rodrigo Florencio ◽  
Fabiana Ostronoff ◽  
Gilma Campos ◽  
Sandra Arruda ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Bone Marrow Transplantation ◽  
Aplastic Anemia ◽  
Marrow Transplantation ◽  
Hemophilia A ◽  
Allogeneic Bone Marrow Transplantation ◽  
Allogeneic Bone Marrow ◽  
Severe Aplastic Anemia ◽  
Allogeneic Bone ◽  
Catheter Implantation

Abstract Hemophilia A is a blood clotting disorder characterized by a mutation of the factor VIII (FVIII) gene, leading to a deficiency in FVIII. It is the most common type of hemophilia. Inheritance is X-linked; hence males are affected while females are carriers or very rarely display a mild phenotype. 1:10,000 males are affected. We describe a child with a rare association of congenital hemophilia A and acquired severe aplastic anemia (SAA) who was successfully treated with allogeneic bone marrow transplantation (allo-BMT). A 6-year old male, SG, was diagnosed with hemophilia A (FVIII 3%) at 6-month old when he presented with right knee hemarthroses and psoas muscle hematoma. His maternal grandfather was hemophiliac. Since his diagnose, FVIII was infused on average once a month, generally due to hemarthroses and bleeding secondary to trauma. In July 2004, pt presented with progressive pancytopenia. In January 2005 CBC showed: Hb 7g/dl, Ht 22%, WBC 1000/μl (absolute neutrophil count 250/μl) and PLT 23,000/μl. Bone marrow aspirate and biopsy were consistent with SAA. DEB test was negative. Since then he was poly-transfused with PLT and RBC due to increased bleeding tendency, especially hematoma and petechiae. In July 2005 he underwent an allo-BMT from his HLA-identical non-hemophiliac 5-year old brother. For catheter implantation pt received PLT transfusion to achieve a PLT level greater than 50,000/mm3 and 60 units/kg of FVIII to achieve an initial level of 100% followed by 30 units/kg every 12 hours to maintain it continuously at greater than 50% for 3 days. Conditioning consisted of cyclophosphamide 50mg/day from D-5 to D-2 and thymoglobulin 5mg/kg from D-3 to D-1. Cyclosporine and a short course of methotrexate were given for GVHD prophylaxis. 3,5x108 mononuclear cells/kg were infused. Allo-BMT was uneventful. Neutrophil engrafted on D+15 and megakaryocyte (>50,000/mm3) on D+38. During the transplant PLT were kept at greater than 30,000/mm3 with PLT transfusion every 2 days. FVIII was infused when there was more than 2 cm increase in the knee perimeter or any other abnormal bleeding, especially at the catheter implantation site. 6 months after allo-BMT the child is well, with a normal CBC and coagulation tests similar to pre-BMT. Hemophilia and AA association is rare, and to our knowledge, this is the first allo-BMT in a hemophiliac patient to be reported.

scholarly journals Bone marrow transplantation for severe aplastic anemia: a randomized controlled study of conditioning regimens

Blood ◽  
2007 ◽  
Vol 109 (10) ◽  
pp. 4582-4585 ◽  
Author(s):  
Richard E. Champlin ◽  
Waleska S. Perez ◽  
Jakob R. Passweg ◽  
John P. Klein ◽  
Bruce M. Camitta ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Bone Marrow Transplantation ◽  
Aplastic Anemia ◽  
Marrow Transplantation ◽  
Allogeneic Bone Marrow Transplantation ◽  
Survival Rates ◽  
Severe Aplastic Anemia ◽  
Controlled Study ◽  
High Dose ◽  
Allogeneic Bone

AbstractThe addition of antithymocyte globulin (ATG) to a regimen of high-dose cyclophosphamide has been advocated to enhance engraftment after allogeneic bone marrow transplantation (BMT) for severe aplastic anemia (SAA). In a prospective clinical trial, 134 patients were randomly assigned to receive cyclophosphamide alone or in combination with ATG. All patients received T-cell–replete bone marrow from an HLA-matched sibling. With a median follow-up of 6 years, the 5-year probabilities of survival were 74% for the cyclophosphamide alone group and 80% for the cyclophosphamide plus ATG group (P = .44). Graft failure and graft-versus-host disease (GVHD) rates were similar in both groups. With the survival rates achieved, this study is not adequately powered to detect significant differences between the 2 treatment groups. In conclusion, the results of allogeneic BMT for SAA have improved over time related to advances in supportive care. The addition of ATG to the preparative regimen did not significantly improve the outcome.

Pediatric Bone Marrow Transplantation: Current Progress and Future Prospects

PEDIATRICS ◽  
1983 ◽  
Vol 72 (6) ◽  
pp. 818-822
Author(s):  
PAUL M. SONDEL ◽  
MICHAEL E. TRIGG ◽  
RICHARD HONG ◽  
JONATHAN L. FINLAY ◽  
MAREK J. BOZDECH
Keyword(s):  
Immune Deficiency ◽  
Bone Marrow ◽  
Bone Marrow Transplantation ◽  
Aplastic Anemia ◽  
Marrow Transplantation ◽  
Allogeneic Bone Marrow Transplantation ◽  
Allogeneic Bone ◽  
Acute Leukemias ◽  
Term Survival ◽  
And Function

Allogeneic bone marrow transplantation (BMT) has been applied with increasing frequency and success to the treatment of children with severe immune deficiency disease,1,2 aplastic anemia,3,4 and the acute leukemias.5,8 Patients with these otherwise rapidly fatal diseases receive an intravenous infusion of marrow from a healthy donor. The healthy marrow either "replaces" the deficient marrow of children with immune deficiency or aplastic anemia, or "rescues" the marrow of children who have received ablative antileukemic therapy. The resultant engraftment makes the patient a chimera, with reconstitution of mature hematopoietic and immunologic cells of donor origin. When successful, this results in long-term survival with normal marrow function, no recurrence of the original disease, and a return to normal childhood activity and function with only a few irreversible but major side effects (such as infertility following total body irradiation).9

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