scholarly journals Sirolimus-Based Immunosuppression as GvHD Prophylaxis after Bone Marrow Transplantation for Severe Aplastic Anaemia: A Case Report and Review of the Literature

2015 ◽  
Vol 2015 ◽  
pp. 1-3
Author(s):  
Katia Perruccio ◽  
Elena Mastrodicasa ◽  
Francesco Arcioni ◽  
Ilaria Capolsini ◽  
Carla Cerri ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Bone Marrow Transplantation ◽  
Aplastic Anaemia ◽  
Marrow Transplantation ◽  
Immunosuppressive Treatment ◽  
Conditioning Regimen ◽  
Severe Aplastic Anaemia ◽  
Leukocyte Antigen ◽  
Gvhd Prophylaxis ◽  
Grade Ii

Congenital or acquired severe aplastic anaemia (SAA) is cured by bone marrow transplantation (BMT) from a histocompatible leukocyte antigen- (HLA-) identical sibling. The best conditioning regimen is cyclophosphamide (CTX) with or without antithymocyte globulin (ATG), followed by short-term methotrexate (MTX) and cyclosporine A (CsA) to prevent graft-versus-host disease (GvHD). In our pediatric oncology-hematology unit, a 5-year-old girl with SAA was treated with two BMT from the same HLA-identical sibling donor. Severe CsA-induced adverse events (severe hypertension and PRES) after the first BMT led necessarily to CSA withdrawal. Alternative immunosuppressive treatment for GvHD prevention as tacrolimus and mycophenolate were not tolerated by our patient because toxicity > grade II. For this reason we decided to administrate sirolimus alone as GvHD prophylaxis and to prevent disease relapse after the rescue BMT. Here we report the successful use of sirolimus alone for GvHD prophylaxis after the second transplant in a pediatric BMT setting for SAA.

scholarly journals Bone marrow transplantation for children with severe aplastic anemia: use of donors other than HLA-identical siblings

Blood ◽  
1989 ◽  
Vol 74 (5) ◽  
pp. 1852-1857 ◽  
Author(s):  
B Camitta ◽  
R Ash ◽  
J Menitove ◽  
K Murray ◽  
C Lawton ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Bone Marrow Transplantation ◽  
Aplastic Anemia ◽  
Marrow Transplantation ◽  
Chronic Gvhd ◽  
Conditioning Regimen ◽  
Pneumocystis Pneumonia ◽  
Severe Aplastic Anemia ◽  
High Dose ◽  
Gvhd Prophylaxis

Abstract Eighty-five percent of untransfused and 70% of transfused patients with severe aplastic anemia (SAA) are cured with bone marrow transplants from histocompatible sibling donors. Use of partially matched family donors or unrelated donors has been relatively unsuccessful because of high incidences of graft rejection and graft-versus-host disease (GVHD). Thirteen children with SAA received marrow grafts from alternative donors (sibling 4, parent 5, unrelated 4). The first three patients were pretreated with cyclophosphamide (CYCLO) +/- irradiation and received methotrexate for GVHD prophylaxis. Subsequent children were pretreated with CYCLO + high-dose cytosine arabinoside + methylprednisolone + total body irradiation, had monoclonal antibody T- cell depletion of the donor marrow, and received cyclosporine for GVHD prophylaxis. Three heavily transfused patients with haploidentical- related donors failed to engraft and died. All 10 patients with more closely matched donors engrafted. Acute GVHD was grade II in only one patient (non-T-depleted); this patient is the only one with severe chronic GVHD. Three engrafted patients died (Pneumocystis pneumonia, systemic parainfluenza, venocclusive disease). Seven children are alive 33+ to 2,692+ days. Donors for the survivors were siblings 3, parent 1, unrelated 3. These data suggest that bone marrow transplantation from closely matched donors other than histocompatible siblings can be effective therapy for SAA if an intensive conditioning regimen is used. These results must be confirmed with larger numbers and longer follow- up.

Risk factors for interstitial pneumonia following bone marrow transplantation for severe aplastic anaemia

1989 ◽  
Vol 71 (4) ◽  
pp. 535-543 ◽  
Author(s):  
Roy S. Weiner ◽  
Mary M. Horowitz ◽  
Robert Peter Gale ◽  
Karel A. Dicke ◽  
Dirk W. Bekkum ◽  
...  
Keyword(s):  
Risk Factors ◽  
Bone Marrow ◽  
Bone Marrow Transplantation ◽  
Aplastic Anaemia ◽  
Interstitial Pneumonia ◽  
Marrow Transplantation ◽  
Severe Aplastic Anaemia

Bone marrow transplantation for severe aplastic anaemia a review of the Westminster experience of 24 cases

1983 ◽  
Vol 5 (2) ◽  
pp. 131-143 ◽  
Author(s):  
J.R. KENDRA ◽  
C.F. LUCAS ◽  
D. CHAMBERS ◽  
S. WILLIAMSON ◽  
N. BYROM ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Bone Marrow Transplantation ◽  
Aplastic Anaemia ◽  
Marrow Transplantation ◽  
Severe Aplastic Anaemia

Regimen-related toxicity in patients undergoing bone marrow transplantation.

1988 ◽  
Vol 6 (10) ◽  
pp. 1562-1568 ◽  
Author(s):  
S I Bearman ◽  
F R Appelbaum ◽  
C D Buckner ◽  
F B Petersen ◽  
L D Fisher ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Bone Marrow Transplantation ◽  
Marrow Transplantation ◽  
Disease Status ◽  
High Dose ◽  
Gvhd Prophylaxis ◽  
Preparative Regimen ◽  
Life Threatening ◽  
Grade Ii ◽  
Total Body

Bone marrow transplantation is associated with significant morbidity and mortality, some of which is due to high-dose chemoradiotherapy. In order to quantitate toxicity that was felt to be due to the preparative regimen (termed regimen-related toxicity [RRT]), a system was developed in which toxicities were graded from 0 (none) to 4 (fatal). One hundred ninety-five patients who underwent marrow transplantation for leukemia were studied retrospectively to determine whether toxicities that were clinically felt to be due to the preparative regimen were influenced by other factors such as disease status, graft-versus-host disease (GVHD) prophylaxis, and allogenicity. All patients developed grade I toxicity in at least one organ, and 30 developed grades III-IV (life-threatening or fatal) RRT. RRT was more common in relapsed patients v remission patients (P = .04), in those receiving 15.75 Gy total body irradiation (TBI) v 12.0 Gy TBI (P = .028), and in those receiving allogeneic marrow v autologous marrow (P = .0029). Autologous marrow recipients did not develop grades III-IV toxicity in this study. A multivariate analysis controlling for autologous marrow grafting showed that the dose of TBI was the only statistically significant predictor of grades III-IV RRT. Those patients who developed grade III RRT were unlikely to survive 100 days from transplant, though not all deaths could be attributed to RRT. Patients who developed grade II toxicity in three or more organs were more likely to die within 100 days than those developing grade II toxicity in two or less organs (P = .0027). This system was generally able to distinguish RRT from other toxicities observed in marrow recipients.

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