Perfect Engraftment after Fludarabine, Cyclophosphamide Plus Thymoglobulin Conditioning Regimen for Unrelated Transplantation in Severe Aplastic Anemia: Phase II Prospective Multi-Center Study in Korea

Blood ◽  
2008 ◽  
Vol 112 (11) ◽  
pp. 3003-3003
Author(s):  
Hyoung Jin Kang ◽  
Hee Young Shin ◽  
Jun Eun Park ◽  
Young Tak Lim ◽  
Nak Gyun Chung ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Aplastic Anemia ◽  
Phase Ii ◽  
Peripheral Blood ◽  
Promising Result ◽  
Conditioning Regimen ◽  
Severe Aplastic Anemia ◽  
Once Daily ◽  
Donor Type ◽  
Mobilized Peripheral Blood

Abstract Anti-thymocyte globulin (ATG) has been used in severe aplastic anemia (SAA) as a part of the conditioning regimen. Among the many kinds of ATG preparations, thymoglobulin had been found to be more effective in preventing graft-versus-host disease (GVHD) and rejection of organ transplants. After the promising result of the pilot study (Bone Marrow Transplant. 2004. 34; 939), phase II prospective multi-center clinical trial was performed with fludarabine, cyclophosphamide plus thymoglobulin conditioning regimen to allow good engraftment in unrelated transplantation for SAA. Twenty-eight patients underwent bone marrow (N=15) or mobilized peripheral blood (N=13) transplantation with cyclophosphamide (50 mg/kg once daily i.v. on days −9, −8, −7 & −6), fludarabine (30 mg/m^2 once daily i.v. on days −5, −4, −3 & −2) and thymoglobulin (2.5 mg/kg once daily i.v. on days −3, −2 & −1) from HLA matched unrelated donors. GVHD prophylaxis regimen was composed of cyclosporine (or tacrolimus), methotrexate, with or without low dose thymoglobulin (1.25 mg/kg once daily i.v. on days 7, 9 and 11). The median infused cell dose of nucleated cells and CD34 positive cells were 6.8×10^8/kg (1.3– 39.9×10^8/kg) and 5.2×10^6/kg (1.2–27.0×10^6/kg), respectively. The median number of days required for ANC of more than 0.5×10^9/l and 1.0×10^9/l were 14 days (10–35 days) and 15 days (11–40 days), respectively. The spontaneous platelet recovery to more than 20×10^9/l required a median of 22 days (22–182 days). Donor type hematologic recovery (donor type chimerism more than 90%) was achieved in all patients. Fourteen patient developed grade II–IV acute GVHD. The event free survival (EFS) was 73% and all events were transplantation related mortality (TRM) which included coagulopathy (N=3), PTLD (N=2), pneumonia (N=1), and myocardiac infarction (N=1). The EFS of patients who received bone marrow (65%) was not different from that of patients who received mobilized peripheral blood (82%) (P=0.37), but the EFS of patients who received immunosuppressive therapy (IST) previously (55%) was lower than that of patients who didn’t receive IST (92%), significantly (P=0.04). Fludarabine, cyclophosphamide plus thymoglobulin conditioning allows for the promising result of very good engraftment, although serious events occurred in some patients. We are now planning to start new multicenter study to decrease TRM by reducing the dose of cyclophosphamide.

Improved Outcome of Reduced Toxicity Fludarabine, Cyclophosphamide Plus Thymoglobulin Conditioning Regimen for Unrelated Donor Transplantation in Severe Aplastic Anemia: Comparison of Two Multicenter Prospective Studies

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 220-220
Author(s):  
Hyoung Jin Kang ◽  
Ji Won Lee ◽  
Hyery Kim ◽  
Kyung Duk Park ◽  
Hee Young Shin ◽  
...  
Keyword(s):  
Aplastic Anemia ◽  
Phase Ii ◽  
Graft Failure ◽  
Phase Ii Study ◽  
Conditioning Regimen ◽  
Severe Aplastic Anemia ◽  
Unrelated Donor ◽  
Once Daily ◽  
Conditioning Regimens ◽  
Reduced Toxicity

Abstract Abstract 220 Introduction: Hematopoietic stem cell transplantation (HSCT) is a curative therapeutic modality for severe aplastic anemia, but optimal conditioning regimen for the HSCT with an unrelated donor has not been defined yet. As the thymoglobulin had been found to be more effective among many kinds of anti-thymocyte globulins, and fludarabine based conditioning regimens without total body irradiation have been reported to be promising for transplantation from unrelated donors in SAA, combination of fludarabine, cyclophosphamide and thymoglobulin conditioning regimens had been tried to reduce GVHD and to allow good engraftment. Our previous phase II study (study 1) of fludarabine, cyclophosphamide plus thymoglobulin conditioning regimen resulted in successful engraftment (100%), but treatment-related mortality (TRM) occurred in 9 (32.1%) patients (NCT00737685, Biol Blood Marrow Transplant. 2010.16;1582). As cyclophosphamide is more toxic than fludarabine with similar effect, then we performed a new phase II study (study 2) with reduced toxicity fludarabine, cyclophosphamide plus thymoglobulin conditioning regimen by reducing dosage of cyclophosphamide and increasing dosage of fludarabine (NCT00882323). Patients and Methods: Twenty-eight and 31 patients were enrolled in study 1 and 2, respectively. In study 1, cyclophosphamide (50 mg/kg once daily i.v. on days −9, −8, −7 & −6), fludarabine (30 mg/m2̂ once daily i.v. on days −5, −4, −3 & −2) and thymoglobulin (2.5 mg/kg once daily i.v. on days −3, −2 & −1) were used for the conditioning regimen. For study 2, cyclophosphamide was reduced to 60 mg/kg once daily i.v. on days −8 & −7, and fludarabine was increased to 40 mg/m2̂ once daily i.v. on days −6, −5, −4, −3 & −2. Thymoglobulin (2.5 mg/kg once daily i.v. on days −4, −3 & −2) was also used. Results: Donor type hematologic recovery was achieved in all patients of study 1 (100%) and study 2 (100%). Events were occurred in 10 patients of study 1. Nine patients developed TRM, which included thrombotic microangiopathy (N=2), pneumonia (N=1), myocardiac infarction (N=1), post-transplantation lymphoprolifarative disease (N=3), and chronic GVHD-associated complications (N=2). Delayed graft failure occurred in 1 patient at 37 months after HSCT. In study 2, 2 patients had events. One patient developed TRM (pneumonia) and delayed graft failure occurred in 1 patient at 4 months after HSCT. Overall survival rate of study 2 (96.7%) was significantly higher than that of study 1 (67.9%) (P=0.005). Event free survival of patients was significantly better in study 2 (93.3%) compared to that of study 1 (64.3%) (P=0.014). Conclusions: Reduced toxicity fludarabine, cyclophosphamide plus thymoglobulin conditioning regimen showed promising results with same successful engraftment and less TRM compared to the previous combination and was optimal for the unrelated donor transplantation in severe aplastic anemia. Disclosures: No relevant conflicts of interest to declare.

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.

scholarly journals Increased incidence of solid malignant tumors after bone marrow transplantation for severe aplastic anemia [see comments]

Blood ◽  
1991 ◽  
Vol 78 (2) ◽  
pp. 277-279 ◽  
Author(s):  
G Socie ◽  
M Henry-Amar ◽  
JM Cosset ◽  
A Devergie ◽  
T Girinsky ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Bone Marrow Transplantation ◽  
Aplastic Anemia ◽  
Marrow Transplantation ◽  
Malignant Tumors ◽  
Conditioning Regimen ◽  
Severe Aplastic Anemia ◽  
Male Patients

Abstract From May 1980 to December 1989, 107 consecutive patients with non- constitutional severe aplastic anemia underwent bone marrow transplantation at our institution using cyclophosphamide and thoraco- abdominal irradiation as conditioning regimen. During the same period, 40 patients with Fanconi anemia were also grafted after a similar conditioning, giving a total series of 147 patients. With a mean follow- up of 64 months, four male patients developed a solid malignant tumor, a number that leads to an 8-year cumulative incidence rate of 22% (eg, relative risk to general population = 41, P less than .001). These results should be considered as a warning to clinicians who follow these successfully grafted long-term patients.

Fludarabine Based Conditioning for Allogeneic Transplantation in Severe Aplastic Anemia.

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 2029-2029
Author(s):  
Mammen Chandy ◽  
Biju George ◽  
Auro Viswabandya ◽  
Vikram Mathews ◽  
Ashish Bajel ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Aplastic Anemia ◽  
Chronic Gvhd ◽  
Conditioning Regimen ◽  
Allogeneic Transplantation ◽  
Severe Aplastic Anemia ◽  
Peripheral Blood Stem Cells ◽  
Allogeneic Bmt ◽  
Gvhd Prophylaxis ◽  
Blood Stem Cells

Abstract Patients with severe aplastic anemia (SAA) who are multiply transfused or septic have a poor outcome after allogeneic stem cell transplantation. Forty three patients (31 males and 12 females) with SAA underwent allogeneic BMT using a fludarabine based conditioning regimen between 1998 and 2005. The median age was 20 years (range: 4–38) with 11 children and 32 adults. All donors were 6 antigen matched HLA identical sibling or family donors. Co morbidities seen included bacterial sepsis in 15 patients, fungal pneumonia in 4 and a recent intracranial bleed in 5 patients. Seven patients had failed Antithymocyte or antilymphocyte globulin (ATG/ALG) and two patients had failed their first transplant. The median time from diagnosis to transplant was 12 months (range: 2 – 96) and the median transfusions prior to BMT was 28 (range: 2 – 380). Conditioning therapy consisted of: Fludarabine (Flu) 180 mg/m2 over 6d + Busulfan (Bu) 8 mg/kg over 2d + ATG 40 mg/kg/day over 4 d in 17 patients, Flu 180 mg/m2 over 6d + Cyclophosphamide (Cy) 120 mg/kg over 2d ± ATG 40 mg/kg/day over 4d in 17 patients, Flu/TBI/OKT3 in 4, and Cy 120 + Flu 150mg/m2 in 5 patients. Graft versus host disease (GVHD) prophylaxis consisted of Cyclosporine and mini methotrexate. Graft source was peripheral blood stem cells in 39 patients and G-CSF stimulated bone marrow in 4. The median cell dose was 5.2 x 108 MNC/kg (range: 2.1 – 13.6) for PBSC and 5.2 x 108 TNC/kg (range: 3.7 – 6.8) for bone marrow. Five patients expired within the first 10 days due to sepsis. Thirty seven patients engrafted with a mean time to ANC > 500 of 11.6 days (range: 8 – 18) and median platelet engraftment time of 13 days (range: 8 – 32). One patient had primary graft failure and expired on day 64 due to fungal pneumonia despite a second transplant. Acute GVHD was seen in 14 patients (38%) with Grade III–IV GVHD in 4 (10.5%). Chronic GVHD was seen in 10 patients with 6 having limited and 4 with extensive GVHD. Two patients had secondary graft rejection on day + 24 and +60 respectively and expired due to fungal pneumonia. At a median follow up of 17 months (range: 5 – 78); 29 patients (67.7%) are alive and well. Among patients treated with Flu/Bu/ATG, 12/17 (70.5%) are alive and well while the DFS is 82% (14/17) in patients treated with Flu/Cy ± ATG. Comparison with patients conditioned with Cy/ATG during 1990–2004 is given in the table. This comparison suggests that a fludarabine based conditioning regimen may be better, with less rejection, than Cy/ATG for allogeneic BMT in sick patients with SAA who are infected and multiply transfused at the time of BMT. Comparative data Fludarabine Cy/ATG Number 43 26 Rejection 3 (7%) 7 (27%) DFS 29(67.7%) 11 (46%)

Unrelated Cord Blood Cell Transplantation for Acquired Severe Aplastic Anemia: The Report from the Japan Cord Blood Bank Network.

Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 2020-2020
Author(s):  
Seiji Kojima ◽  
Ayami Yoshimi ◽  
Shuichi Taniguchi ◽  
Junichi Hara ◽  
Toshimitsu Matsui ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Cord Blood ◽  
Aplastic Anemia ◽  
Cumulative Incidence ◽  
Cord Blood Transplantation ◽  
Conditioning Regimen ◽  
Severe Aplastic Anemia ◽  
Bone Marrow Donor ◽  
Grade Ii ◽  
Unrelated Cord Blood

Abstract Treatment approaches for patients with severe aplastic anemia (SAA), who failed immunosuppressive therapy and lack a bone marrow donor remains a great challenge. Unrelated cord blood transplantation (UCBT) has not been recommended for SAA because of historical poor outcome with high rate of engraftment failure. To evaluate the current feasibility of UCBT in SAA, we retrospectively analyzed the outcomes of 31 patients (median age 28 years old; ranged 0.9–72.3 years old) with SAA, who received UCBT as the first graft between 1998 and 2006 in Japan. Median disease duration before UCBT was 337 day (31–5063 days). By serology, HLA loci were matched in 4 recipient-donor pairs and mismatched (1–2 loci) in 27 patients. A minimum cell count of 2 × 107 nucleated cells/kg body weight was infused in all patients. Engraftment was observed in 17 of 24 evaluable patients. The median times to achieve a neutrophil count ≥ 0.5 × 109/l and a platelet count ≥ 50 × 109/l were 19 days (range 12–35 days) and 59 days (range 39–145 days), respectively. The results of chimerism analysis were available in 9 of them and all of them showed complete donor chimerism (>99%) except one with autologous recovery. Late rejection was seen in one patient. Acute GVHD (≥ grade II) was observed in 5 of 18 evaluable patients (grade II; n=4, grade III; n=1) (cumulative incidence =17.1%) and chronic GVHD was observed in 4 of 14 evaluable patients (extensive: n=1, limited: n=3) (cumulative incidence =19.7%). Currently, 13 patients are alive, having survived for median 22.5 months (ranged 3 to 77 months) after UCBT (overall survival at 2 years=40%). Causes of death of 18 patients were following: graft failure (n=7), bacterial/fungal infections (n=3), hepatic veno-occlusive disease (n=3), and others (n=5). The conditioning regimen appeared to be the most important factor for the outcome and low dose total body irradiation (2–4 Gy) + fludarabine (90–250/ mg/m2) and cyclophosphamide (50–100 mg/kg or 2250/mg/m2) (n=5) gave the best outcome with 80% of survival. The GVHD prophylaxis with single agent (cyclosporine or tacrolimus) related with a better engraftment rate than 2 or more agents (84.4% vs 47.3%, p=0.02). These results suggest that UCBT can be a salvage treatment for patients without a bone marrow donor and warrant further evaluation in prospective studies. Optimization of conditioning regimen will improve the engraftment and outcome of UCBT.

Outcome of Alternative Donor Transplantation for Severe Aplastic Anemia Can Be Comparable to Outcome with Matched Related Donors.

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 2052-2052
Author(s):  
Alana A. Kennedy-Nasser ◽  
Kathryn Leung ◽  
Steven Gottschalk ◽  
Dean A. Lee ◽  
George Carrum ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Overall Survival ◽  
Aplastic Anemia ◽  
Pediatric Patients ◽  
Chronic Gvhd ◽  
Conditioning Regimen ◽  
Severe Aplastic Anemia ◽  
Unrelated Donor ◽  
Alternative Donor

Abstract Matched related donor (MRD) bone marrow transplantation is the treatment of choice for pediatric patients with severe aplastic anemia; however, only 25% of patients will have an HLA-identical sibling. Alternative donor transplants may be an option for these patients, but such therapies have been associated with greater incidences of graft failures and graft-versus-host disease (GVHD). We retrospectively analyzed 32 pediatric patients who have undergone either bone marrow or peripheral blood stem cell transplant for severe aplastic anemia at our institution from April 1997 to April 2005. These patients had a total of 34 transplants. One patient had a MRD transplant followed by a matched unrelated donor (MUD) transplant eight years later, while another patient had a HLA-mismatched unrelated donor (MMUD) transplant followed by a transplant from a haplo-identical parent. Of the remaining 30 patients, 12 received MRD transplants, whereas 18 patients received alternative donor transplants - 11 MUD, 3 haplo-identical donors, and 4 MMUD. The median age at transplant was 9 years (range 1.5 to 18.4 yrs). All patients who received alternative donor transplants had previously failed therapy, including antithymocyte globulin (ATG) and cyclosporine. For MRD transplants, the conditioning regimen most often utilized cyclophosphamide 50 mg/kg x 4 days and ATG 30 mg/kg x 3 days. For alternative donor transplants, the conditioning regimen most often utilized cyclophosphamide 50 mg/kg x 4 days, Campath 3–10 mg x 4 days (dependent upon patient’s weight) or ATG 30 mg/kg x 3 days, and TBI (single fraction 200 cGy for MUD; two fractions 200 cGy for MMUD). Alternative donor recipients who received ATG in their preparative regimen were transplanted between December 1997 and March 2001 (n=9), whereas patients who received Campath were treated between November 2001 and April 2005 (n=11). GVHD prophylaxis was either FK506 or cyclosporine +/− mini-methotrexate. The overall survival for MRD patients was 91.7% versus 80% for alternative donor patients at a median follow-up of 47 months (range 3 to 100 months). Of the 32 patients, there were 5 deaths: pulmonary failure with extensive, chronic GVHD (n=1); poor graft function with infection (n=1); and infection (n=3). For patients receiving alternative donor transplants, the overall survival for the Campath group was 81.8% vs. 77.8% in the ATG group. None of the Campath patients developed extensive, chronic GVHD compared to 3/9 ATG patients. In conclusion, alternative donor transplantation using Campath or ATG in the preparatory regimens can establish donor engraftment and offers a curative therapy for pediatric severe aplastic anemia patients with survival similar to that of patients receiving matched sibling transplants. Although follow-up is shorter, Campath may be associated with a reduced incidence of extensive, chronic GVHD and further investigation is warranted.

scholarly journals Morphology in patients with severe aplastic anemia treated with antilymphocyte globulin

Blood ◽  
1992 ◽  
Vol 80 (2) ◽  
pp. 337-345 ◽  
Author(s):  
A Tichelli ◽  
A Gratwohl ◽  
C Nissen ◽  
E Signer ◽  
C Stebler Gysi ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Aplastic Anemia ◽  
Peripheral Blood ◽  
Clonal Evolution ◽  
Cumulative Risk ◽  
Fetal Hemoglobin ◽  
Severe Aplastic Anemia ◽  
Antilymphocyte Globulin ◽  
Probability Of Survival ◽  
Patients At Risk

One hundred and seventeen patients with severe aplastic anemia (SAA) were treated at our institution between 1976 and 1990 with antilymphocyte globulin (ALG) therapy. Seventy-nine (68%) are alive and probability of survival at 14 years, according to Kaplan and Meier, is 62% +/- 12%. Twenty-six patients developed a late clonal complication: 11 had a myelodysplastic syndrome (MDS) and 17 had paroxysmal nocturnal hemoglobinuria (PNH); two patients had both. The cumulative risk at 10 years is 42%. The development of MDS/PNH after SAA directly affects survival. The probability of being alive at 14 years is 81% +/- 10% for patients with stable disease and 36% +/- 13% for those with clonal evolution (P = .001). To look for predictive signs, we reevaluated peripheral blood and bone marrow cytomorphology at presentation, during regeneration, and in remission. We examined the peripheral blood values for hemoglobin, reticulocytes, granulocytes, thrombocytes, mean corpuscular volume (MCV), and fetal hemoglobin, as well as bone marrow for cellularity, erythropoiesis, myelopoiesis, and megakaryopoiesis. ALG therapy induces slow and incomplete recovery. Although in “remission,” ALG patients have lower hemoglobin values, higher reticulocyte counts, lower granulocyte and platelet values, and a higher MCV and fetal hemoglobin than normal controls. They retain a reduced number of megakaryocytes and a persistence of atypical monocytes in bone marrow morphology as stigmata of their disease. Patients with late clonal complications show distinct morphologic abnormalities: patients with PNH have higher MCVs, higher granulocyte and reticulocyte counts, and more dyserythropoiesis at diagnosis and a lower hemoglobin with an increased proportion of erythroblasts in the bone marrow in “remission.” Patients who later developed MDS are not different from the total patient population at diagnosis. After therapy, these patients are characterized by the presence of ring sideroblasts and atypical monocytes during regeneration and by a persistent increase in MCV, a higher fetal hemoglobin, lower granulocyte values, and megakaryocytic dysplasia during “remission.” Thus, routine morphologic follow-up examination of blood and bone marrow can discover patients at risk for late hematologic complications after ALG therapy.

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

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.

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