Allogeneic Stem Cell Transplantation - A Long-Term Curative Approach In Patients ≥ 60 Years With Advanced Disease AML/MDS, - The Freiburg Experience Of 250 Consecutive Patients

Abstract Since the introduction of reduced-toxicity conditioning prior to allogeneic hematopoietic stem cell transplantation (alloSCT) we transplanted from 1999 to 2012, 250 consecutive patients (pts) with myeloid malignancies (AML, MDS) aged ≥ 60 years (yrs). The 144 male and 106 female pts with a median age of 66 yrs (range 60-77) were transplanted for de novo AML (n=95), s/tAML (n=104) and MDS (n=51) with 89% unfavorable cytogenetics (CALGB). Since 2004 pts received a prospective fitness assessment (Deschler et al., Haematologica 2013). In 74% the donor was matched/mismatched unrelated and in 26% related. Only 16% were transplanted in CR1/2, 84 % with advanced or untreated disease. The conditioning regimen was the FBM protocol (fludarabine, carmustine, melphalan; Bertz et al., JCO 2003) in 98%, and 97% of the pts received PBSC. For GVHD prophylaxis in 91% a combination of cyclosporine plus alemtuzumab or ATG-F™ was used. At day +30, 94% of the pts had achieved CR by standard measures. With a median follow up of 57 months (3-157) 37% of the pts are alive; main causes of death were relapse (n=62), infection (n=35) and age-related diseases (n=13). The probability of OS/DFS was at 1yr 61%/49%, at 2 yrs 49%/41% and at 5 yrs 37%/34%, respectively. The probability for NRM at 1 yr is 24%. Nineteen known prognostic factors for outcome were evaluated: e.g. patient and donor age, graft size, days between diagnosis and alloHCT, CMV, early/advanced disease, cytogenetics, Sorror and Gratwohl score, donor type, HLA-identity. In the multivariate analysis a better OS (factors with p<0.1; table) was seen with a matched donor; a better DFS with a related donor, and high CD34+ graft content; in contrast, a mismatched donor is a risk factor for reduced DFS.TableMultivariate analysis of prognostic factors* for OS and DFSvariablevalueHazard Ratio95% CI lower limit95% CI upper limitP valueOverall survivalRemission at alloHCTadvanced1.370.862.160.1825HLA mismatchyes1.401.011.960.0463HCT-CI (Sorror)>= 21.311.011.960.1007Peripheral blood blastsyes1.210.841.760.3034Disease-free survivalRemission at alloHCTadvanced1.290.722.300.3946Donorrelated0.640.430.950.0258HLA mismatchyes1.440.992.090.0561CD34+ cells> median0.760.551.040.0867Bone marrow blasts> 5%1.210.781.880.3915*in univariate analysis p<0.157 (AIC criterion; Sauerbrei W, 1999 Applied Statistics,48:313-329.70.) In conclusion, this unique large cohort of older pts with AML/MDS with mainly advanced disease and unfavorable cytogenetics shows a high feasibility, safety and efficacy of alloHCT after the FBM protocol. AML/MDS pts in their 7th and 8th decade of life fit for transplant should be evaluated for alloHCT as very important long-term curative option. Disclosures: No relevant conflicts of interest to declare.

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Is HLA-Haploidentical Hematopoietic Stem Cell Transplantation a Valid Alternative Option for Patients with Combined Paroxysmal Nocturnal Hemoglobinuria and Aplastic Anemia (AA/PNH Syndrome)?

Blood ◽

10.1182/blood.v126.23.5535.5535 ◽

2015 ◽

Vol 126 (23) ◽

pp. 5535-5535

Author(s):

Feng Chen ◽

Wu Depei ◽

Xiaowen Tang ◽

Miao Miao ◽

Chengcheng Fu ◽

...

Keyword(s):

Stem Cell ◽

Stem Cell Transplantation ◽

Cell Transplantation ◽

Conflicts Of Interest ◽

Therapeutic Option ◽

Cord Blood Transplantation ◽

Chronic Gvhd ◽

Conditioning Regimen ◽

Hematopoietic Stem

Abstract Background In the era of eculizumab, indentifying patients with PNH who may benefit from allogeneic stem cell transplantation(SCT) is challenging, especially for those who have no HLA-matched donors. Several recent studies have shown that HLA-haploidentical SCT for patients with hematological malignancy can achieved comparable outcomes with HLA-identical sibling transplantation. There are very few reports on the use of HLA-haploidentical SCT for AA/PNH. The aim of the present study was to assess the long-term clinical outcome of HLA-haploidentical SCT in patients with AA/PNH. Methods Total of 9 AA/PNH patients received HLA-haploidentical SCT between Oct 2010 and Oct 2014 at our institution. The patients were aged 13 to 54(median 24 years). The median interval from the diagnosis to transplantation was 48 months (range 2-180). Of the 9 HLA-haploidentical donors, 3 were siblings, 2 fathers, 2 mothers, 1 son and 1 daughter. 8 patients received myeloablative conditioning regimen consisting of busulfan, cyclophosphamide and ATG (anti-thymocyte globulin), 1 patient who underwent salvage HLA-haploidentical SCT after the graft failure of double umbilical cord blood transplantation received conditioning including reduced-intensity total body irradiation, cyclophosphomide and ATG. G-CSF-mobilized bone marrow and peripheral blood stem cells were transplanted as graft. Prophylaxis for graft-versus-host disease(GVHD) consisted of cyclosporine or tacrolimus + mycophenolate mofetil + short-term methotrexate. Results All 9 patients were engrafted successfully. The median time of neutrophils (ANC) reached to 0.5×109/L and platelets (PLT) reached to 20×109/L were 12 (range 11-26) days and 15 (range 11-120) days, respectively. 2 patients developed grade Ⅱ acute GVHD, 2 patients developed limited chronic GVHD. After a median follow-up time of 14.0 (range 4.0-30.8) months, the 2-year OS probability was 72.9±16.5%. 2 patients died of treatment-related mortality, including severe pulmonary infection (n=1) and transplant-associated thrombotic microangiopathy (n=1),respectively. No patients were documented to have a recurrence of a PNH clone after SCT. Conclusion This study showed that long-term outcomes of HLA-haploidentical SCT in patients with AA/PNH were comparable to that of HLA-matched donor SCT ( the 2-year OS probability was 80.5±10.2%, P=0.02) at our institution. HLA-haploidentical SCT should be considered as a valid alternative therapeutic option for AA/PNH patients without HLA-matched donors. Disclosures No relevant conflicts of interest to declare.

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Stem Cell Transplantation for Relapsed and Refractory Burkitt Lymphoma.

Blood ◽

10.1182/blood.v116.21.4589.4589 ◽

2010 ◽

Vol 116 (21) ◽

pp. 4589-4589

Author(s):

Steven Kwon ◽

Amir Steinberg ◽

Catherine Bresee ◽

Mercedes Franco ◽

Angela M. Lopez ◽

...

Keyword(s):

Stem Cell ◽

Stem Cell Transplantation ◽

Cell Transplantation ◽

Burkitt Lymphoma ◽

Bone Marrow Involvement ◽

Conditioning Regimen ◽

Small Sample ◽

Refractory Disease ◽

Hematopoietic Stem

Abstract Abstract 4589 Background: Burkitt lymphoma is a highly aggressive and rapidly proliferating hematologic malignancy. Various chemotherapy regimens (HyperCVAD, CODOX-M/IVAC, REPOCH) have been shown to improve overall survival rate. However, in instances of relapsed or refractory disease, there is no clear-cut standard of care. Hematopoietic stem cell transplantation (HCT) has shown benefit in these patients. Here, we report our institutional experience using HCT for relapsed/refractory Burkitt lymphoma. Methods: Between February 1994 and January 2010, 13 patients (10 males, 3 females) with refractory or relapsed Burkitt lymphoma were retrospectively evaluated. The patients in question were administered a particular conditioning regimen followed by stem cell transplantation (11 autologous, 2 allogeneic). The average age at transplant was 41.8± 3.4 years (range 24 to 67). Stem cell source was from peripheral blood in all transplants including allogeneic (2 HLA-identical sibling transplants). The conditioning regimens for HCT consisted of the following: TBI/Rituximab/Cytoxan/VP16, TBI/Cytoxan/VP16, BEAM, Cranial Spinal boost/TBI/Cytoxan/VP16 or Cranial boost/TBI/Cytoxan. Results: On average, patients received transplant 299 ± 36 days after initial diagnosis (range 153 to 582). All patients achieved engraftment. For the 5/13 deceased patients (38%), cause of death was attributed to relapsed/refractory disease (n=2) and treatment related causes (n=3). Using the Kaplan-Meier method, the average survival time after transplant was computed at 27.7 ± 5.3 months and 75% were alive 2-years after transplant. Likely due in part to the small sample size, no covariates (age, gender, conditioning regimen, HIV status, presence of B symptoms, and CNS or bone marrow involvement) were found to be predictive of survival rates. Conclusion: Patients with Burkitt lymphoma, known for its rapid growth, can achieve long-term complete remission (CR) with intense, combination chemotherapy. Despite successful long-term remission rates, a substantial portion of patients die from uncontrolled disease. At the first sign of refractory or relapsed disease, resources should be mobilized to proceed with HCT. Our study, though limited in size, provides further compelling evidence that long-term CR, and potentially cure, may be achieved using HCT as a treatment modality. Disclosures: No relevant conflicts of interest to declare.

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Femoral Head Necrosis In Three Patients After Allogeneic Hematopoietic Stem Cell Transplantation

Blood ◽

10.1182/blood.v122.21.5489.5489 ◽

2013 ◽

Vol 122 (21) ◽

pp. 5489-5489

Author(s):

Gang Zhao ◽

Zhi Li ◽

Jiahua Ding ◽

Jun Wang ◽

Zhengping Yu ◽

...

Keyword(s):

Stem Cell ◽

Femoral Head ◽

Hematopoietic Stem Cell Transplantation ◽

Stem Cell Transplantation ◽

Cell Transplantation ◽

Hematopoietic Stem Cell ◽

Conflicts Of Interest ◽

Conditioning Regimen ◽

Femoral Head Necrosis ◽

Hematopoietic Stem

Abstract Femoral head necrosis (FHN) is one of common complications after hematopoietic stem cell transplantation (HSCT). It impacts on patients' normal life with severe pain. To investigate FHN after allogeneic HSCT, We performed retrospective analyses. Since 2003, our department has conducted 98 cases of allogeneic hematopoietic stem cell transplantation for patients with hematologic diseases. Chemotherapy regimens and transplant conditioning regimen before transplantation were steroid-free. FHN occurred in 3 out of 98 cases. The 3 patients were treated with steroid for preventing graft versus host disease (GVHD) after transplantation. However, all the three patients suffered from GVHD, which was cured with steroidal medication subsequently. Then, symptoms of FHN come out and were significantly improved after conservative treatment in all the three patients. The occurrence of FHN might be associated with GVHD and corticosteroids prescription. Early prevention might be helpful in reducing the incidence and improving outcomes. Disclosures: No relevant conflicts of interest to declare.

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Outcomes of second allogeneic hematopoietic stem cell transplantation (SCT) for patients with acute lymphoblastic leukemia (ALL).

Journal of Clinical Oncology ◽

10.1200/jco.2012.30.15_suppl.6540 ◽

2012 ◽

Vol 30 (15_suppl) ◽

pp. 6540-6540

Author(s):

LM Poon ◽

Roland Bassett ◽

Gabriela Rondon ◽

Laura L Worth ◽

Laurence Cooper ◽

...

Keyword(s):

Stem Cell ◽

Acute Lymphoblastic Leukemia ◽

Hematopoietic Stem Cell Transplantation ◽

Stem Cell Transplantation ◽

Cell Transplantation ◽

Lymphoblastic Leukemia ◽

Univariate Analysis ◽

Hematopoietic Stem ◽

Active Disease

6540 Background: Disease relapse is the major cause of failure after allogeneic hematopoietic stem cell transplantation (SCT) for acute lymphoblastic leukemia (ALL). Treatment options for these patients (pts) are limited and only a second SCT provides a realistic chance for long term disease remission. Methods: We retrospectively analyzed the outcomes of 31 pts with ALL who relapsed following their first allogeneic SCT, and went on to receive a second allogeneic SCT. Univariate analysis was used to assess for risk factors which influenced treatment-related mortality (TRM), and progression free survival (PFS) following second SCT. Results: 31 pts were evaluable for response with 2 early deaths within 30 days of SCT. The median age of the pts was 26 years (range 7- 49), and the median duration between their first SCT (SCT1) to relapse was 9.5 months (range 2.2-32.6 months). 39% of pts were transplanted in active disease (n=12). The complete remission (CR) rate post SCT was 89%; 83% of pts transplanted with active disease attained CR. With a median follow-up of 3 years among survivors, PFS, and OS rates at 1 year were estimated at 23%. The TRM rate was 41% at 12 months. We did not identify any factors that impacted TRM through univariate analysis. We found a significant relationship between the time to progression following SCT 1 and PFS following SCT 2 (p=0.02, HR=0.93/month). Conclusions: In summary, a second transplant remains an effective method for achieving response in a highly refractory patient population. Pts with longer PFS following SCT1 have a better PFS following SCT2. While long-term survival is limited, a significant proportion of pts remain disease-free for up to one year following SCT2, providing a window of time to administer preventive interventions. Notably, our 4 long-term survivors, received novel therapies in the form of a single umbilical cord blood unit in addition to the PBSC to augment the immune response (n=2), a change in the stem cell source for the SCT from cord to mismatched adult unrelated (n=1), and post SCT maintenance therapy with 5-azacytidine (n=1), underscoring the need for a fundamental change with the methods for the second transplant to improve outcome.

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Monosomal Karyotype in Myeloid Malignancies Is Prognostically Worse Even After Allogeneic Hematopoietc Stem Cell Transplantaion; Results From Two Transplant Centers,

Blood ◽

10.1182/blood.v118.21.4154.4154 ◽

2011 ◽

Vol 118 (21) ◽

pp. 4154-4154 ◽

Cited By ~ 1

Author(s):

Minauchi Koichiro ◽

Akio Shigematsu ◽

Masanobu Nakata ◽

Toshihiro Matsukawa ◽

Koh Ebata ◽

...

Keyword(s):

Stem Cell ◽

Stem Cell Transplantation ◽

Cell Transplantation ◽

Cox Regression ◽

Conflicts Of Interest ◽

Conditioning Regimen ◽

Structural Abnormality ◽

Hematopoietic Stem ◽

Myeloid Malignancies ◽

Monosomal Karyotype

Abstract Abstract 4154 Background: Monosomal karyotype (MK) has been defined as the presence of two or more autosomal monosomies or of a single monosomy associated with at least one structural abnormality (Breems et al, JCO 2008). The presence of MK has been associated with extremely poor prognosis in patients with not only acute myeloid leukemia (AML) but myelodysplastic syndrome (MDS) (Patnaik et al, Leukemia 2011). Our goal was to investigate the efficacy of allogeneic hematopoietic stem cell transplantation (HSCT) for myeloid malignancies with MK. Patients and methods: We combined data from two transplant centers, Sapporo Hokuyu Hospital and Hokkaido University Hospital, and analyzed consecutive patients who underwent allogeneic transplantation for AML and MDS between January 2003 and July 2010. Patients were divided according to MK classification scheme into four groups (Oran et al, BBMT 2011), CN; cytogenetically normal, MK; monosomal karyotype, CBF; core binding factor abnormalities, Oth (Others); non-CBF and non-MK abnormalities. Patients with acute promyelocytic leukemia were excluded. Resuts: One-hundred eighty three out of 229 patients were analyzed with a median age of 48 years(15–68). Sixty one (33%) were from HLA-matched related donors, 86 (47%) from unrelated and 36 (20%) were cord blood.Conditioning regimens were myeloablative (MAC, n=102, 56%) or reduced intensity(RIC, n=81, 44%). Seventy patients (38%) were cytogenetically normal, 27 (15%)had CBF abnormalities, 70(38%) had non-CBF and non-MK abnormalities and 16(9%) had monosomal karyotype. There was no statistically difference between four groups in age, donor source and conditioning regimen. In the MK group, the proportion of MDS and non-remission state at stem cell transplantation were significantly higher than other groups (p=0.002, p<0.001). Four-year over all survival in patients with MK was 0%, which was significantly inferior to other groups; 50% for CN, 30.4% for CBF, 29.4% for Oth(p<0.001). Cox regression modeling showed that the disease status at stem cell transplantation (p=0.026) and the existence of MK (p=0.012) had prognostic value. Seven of 16 patients with MK died within the first 50 days after transplantation, and 9 patients died within 120 days. Five patients died of infection and 2 died of complicated organ failure and 2 died of progression disease. Three patients who underwent transplantation at non-remission setting, survived more than 1-year experienced chronic graft-versus-host disease, suggesting the existence of GVL effect to myeloid malignancies with MK. Conclusion: This retrospective analysis revealed the dismal prognosis of myeloid malignancies with MK, even after allogeneic HSCT. Novel therapies and strategies are urgently needed for this very poor prognostic group. Disclosures: No relevant conflicts of interest to declare.

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Relevance and Factors Predicting for Early Lymphocyte Recovery After Allogeneic Bone Marrow Stem Cell Transplantation (BMT).

Blood ◽

10.1182/blood.v120.21.3053.3053 ◽

2012 ◽

Vol 120 (21) ◽

pp. 3053-3053

Author(s):

Ulas D. Bayraktar ◽

Rima M Saliba ◽

Gabriela Rondon ◽

Antonio Di Stasi ◽

Partow Kebriaei ◽

...

Keyword(s):

Stem Cell ◽

Stem Cell Transplantation ◽

Cell Transplantation ◽

Proportional Hazards ◽

Conflicts Of Interest ◽

Univariate Analysis ◽

Conditioning Regimen ◽

Allogeneic Bone ◽

Limited Information ◽

Grade Ii

Abstract Abstract 3053 Recovery of the immune system is critical for the success of allogeneic stem cell transplantation. Several groups, including ours, reported that faster lymphocyte recovery was associated with improved outcomes. There is limited information however, on factors affecting lymphocyte recovery and its influence on outcomes specifically after BMT. We retrospectively assessed 536 consecutive patients (pts) with acute leukemia (452 AML/MDS and 102 ALL) who underwent BMT (and engrafted) at our institution between 01/1999 and 12/2010 to determine 1) predictors of early lymphocyte recovery (ELR), and 2) influence on outcomes of ELR defined as achieving absolute lymphocyte count of 1000/μL (ALC1000) by day 100 after BMT. Characteristics of the study population including demographics, graft, disease, and transplant characteristics assessed are described in the Table. Conditioning regimens were classified as reduced intensity (RI), or high intensity (including TBI-, busulfan-, or melphalan-based) conditioning, as previously described. Cox's proportional hazards regression analysis was used to assess predictors of ELR and NRM on univariate and multivariate (MV) analysis. Pts who developed grade II-IV acute GVHD (aGVHD), received a second graft infusion, or relapsed before achieving ALC1000 were censored at the time of these events for the assessment of predictors of ELR. Only pts who were alive progression-free, and had not developed grade II-IV aGVHD by day 100 were eligible for the assessment of predictors of outcomes in landmark analysis starting on day 100 after BMT. Outcomes were assessed at the median follow-up in surviving pts of 40 months. On multivariate analysis, significant predictors of lower rate of ELR included TBI- or melphalan-based ablative conditioning (HR=0.5, p 0.003) (compared to RI or busulfan-based), and a haploidentical donor (HR=0.4, p 0.05). Preliminary analyses assessing CMV reactivation and grade II-IV aGVHD as time dependent variables showed these factors to be associated with higher and lower ELR, respectively. MV analyses incorporating these factors in prognostic model are ongoing and final results will be presented at the meeting. Among the 246 pts eligible for the outcomes assessment, ELR (HR=0.2, P <0.001) and remission status (CR1/CR2) at transplant (HR=0.3, P 0.004) were the only significant predictors of the rate of NRM on MV. The lower NRM rate in pts with ELR translated into higher overall survival on univariate analysis, yet this association was only significant at 1 year (HR1year 0.5, p=0.02; HR40 mo 0.7, p=0.2) after transplant. There was no impact for ELR on the rate of disease relapse at either one of these time points after BMT. In conclusion, early lymphocyte recovery is an independent prognostic factor for NRM after BMT. Lymphocyte recovery was influenced by the conditioning regimen, the use of a haploidentical donor and the development of aGVHD but not by the infused CD34 cell numbers or disease status at transplant. Disclosures: No relevant conflicts of interest to declare.

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Effect of Melphalan 140 Mg/m2 Versus 200 Mg/m2 on Toxicities and Outcomes in Multiple Myeloma Patients Undergoing Single Autologous Stem Cell Transplantation

Blood ◽

10.1182/blood.v126.23.1988.1988 ◽

2015 ◽

Vol 126 (23) ◽

pp. 1988-1988

Author(s):

Lakshmikanth Katragadda ◽

Lindsay McCullough ◽

Yunfeng Dai ◽

Jack W Hsu ◽

John W Hiemenz ◽

...

Keyword(s):

Multiple Myeloma ◽

Stem Cell ◽

Stem Cell Transplantation ◽

Cell Transplantation ◽

Conflicts Of Interest ◽

Conditioning Regimen ◽

Disease Status ◽

Cox Proportional Hazards Model ◽

Hematopoietic Stem ◽

Preparative Regimen

Abstract Introduction: Although melphalan at a dose of 140 mg/m2 (MEL-140) is known to be an effective preparative regimen for autologous hematopoietic stem cell transplantation (ASCT) in multiple myeloma (MM) patients, there are very few studies comparing it to the most commonly used dose of 200 mg/m2 ( MEL-200). Methods: We retrospectively reviewed the records of all myeloma patients who underwent an ASCT between 2001 and 2010 at our institution. We then identified patients who received melphalan as their preparative regimen at doses of 140 mg/m2 or 200 mg/m2. Patients who received any other drug as conditioning regimen or had more than one ASCT or had documented amyloidosis were excluded. Data were collected for variables known to possibly affect prognosis of MM patients. We assessed effect of melphalan dose on toxicities and outcomes. Results: A total of 129 eligible patients were identified, with 33 receiving MEL-140 and 96 receiving MEL-200. As was expected significantly higher percentage of patients in the MEL-140 arm were older than 65 years (P=<0.001) or had cardiac ejection fraction < 50 (P=0.0001) or had Karnofsky score < 80 (P=0.01) or had creatinine > 2 either at diagnosis (P=0.004) or the time of ASCT (P=0.001). Rest of the patient and disease characteristics including Durie-Salmon stage, myeloma subtype and disease status at ASCT were not significantly different between the 2 arms. Patients in MEL-140 needed significantly longer time to ANC engraftment (P=0.037) and also had significantly higher frequency of neutropenic fever (P=0.003). There were no significant differences in mucositis (including grade), nausea, vomiting, diarrhea, bacteremia, or length of hospital stay and frequency of repeat hospitalizations among both groups. There was no treatment related mortality in either group. At a median follow up of 74 months (range, 52-140) from ASCT, there were no significant differences in relapse free survival (RFS) (P=0.4988) and overall survival (OS) (P=0.6936) between the two groups. Five year OS for MEL-140 and MEL-200 is 71.6% and 78.9%, while RFS is 23.9% and 34%, respectively. Proportion of patients whose myeloma status improved to ≥ VGPR at 3 months post ASCT was also not different (P=0.385). Importantly, similar proportions of patients received various post ASCT maintenance therapy (P=0.605). In multivariate cox proportional hazards model only disease status of ≥VGPR at the time of ASCT significantly affected RFS (P=0.024) but did not impact OS (P=0.104). Conclusion: MM patients who received MEL-140 had similar long term outcomes as those who received MEL-200 despite their older age, lower performance status and renal insufficiency. Disclosures No relevant conflicts of interest to declare.

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Haploidentical Stem Cell Transplantation for Rare Pediatric Diseases at Peking University People's Hospital

Blood ◽

10.1182/blood.v128.22.5848.5848 ◽

2016 ◽

Vol 128 (22) ◽

pp. 5848-5848

Author(s):

Yao Chen ◽

Huan Chen ◽

Lan-Ping Xu ◽

Yu-Hong Chen ◽

Jing-Zhi Wang ◽

...

Keyword(s):

Stem Cell ◽

Stem Cell Transplantation ◽

Cell Transplantation ◽

Conflicts Of Interest ◽

Conditioning Regimen ◽

Disease Patient ◽

Rare Disorders ◽

Hematopoietic Stem ◽

Pediatric Diseases ◽

Conditioning Regimens

Abstract Background:Rare diseases requiring hematopoietic stem cell transplantation (SCT) present a challenge for BMT centers. The patients present at a low frequency, and there are often no established conditioning regimens for these disorders, especially for children without HLA matched sibling donors. We report the conditioning regimen using haploidentical related donors, and outcomes for the following rare disorders transplanted at our center from January 2013 through December 2015. During the past three years we had patients presented with the following: Fanconi anemia (FA), dyskeratosis congenital (DC), adrenal leukodystrophy (ALD) and Wiskott-Aldrich syndrome (WAS). Methods:We treated these children with rare disorders that required SCT at our center, and are reporting the transplant outcomes for these rare diseases. Results: All children were transplanted with haploidentical related donors using G-CSF mobilized bone marrow and peripheral blood stem cell. Conditioning regimens and treatment plans were mainly following Beijing protocol and varied with type of disease. Patient characteristics and outcomes are presented in Table 1. No graft failure occurred and three of 5 patients (60%) are alive and well with no evidence of disease. Conclusions: Our program has treated a variety of rare disorders with an event-free-survival rate of 60%. Our data suggests haploidentical SCT for rare pediatric diseases seems to be challenging and promising. Disclosures No relevant conflicts of interest to declare.

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Anti-CD25 instead of ATG for the Conditioning of HLA-Mismtched Hematopoietic Stem Cell Transplantation.

Blood ◽

10.1182/blood.v114.22.4315.4315 ◽

2009 ◽

Vol 114 (22) ◽

pp. 4315-4315

Author(s):

Jun Wang ◽

Qingxiang Meng ◽

Nailan Guo

Keyword(s):

Stem Cell ◽

Hematopoietic Stem Cell Transplantation ◽

Stem Cell Transplantation ◽

Cell Transplantation ◽

Hematopoietic Stem Cell ◽

Conflicts Of Interest ◽

Conditioning Regimen ◽

Hla Typing ◽

Peripheral Blood Stem Cells ◽

Hematopoietic Stem

Abstract Abstract 4315 Purpose To analysis the effect of Anti-CD25 instead of ATG for the conditioning of HLA-mismatched hematopoietic stem cell transplantation (HSCT). Methods 21 cases underwent HLA-mismatched HSCT in our hospital from Mar. 2006 to May 2009. Diagnosis included ANLL(n=9)(2 in CR,7 in Relapse)?ALL(n=2), AHL(n=2), CML(n=4), MDS (n=2) and SAA (n=1), HLA Typing : All patients receive transplants from HLA-mismatched related donors. Among whom, 1,2 and 3 antigen mismatched were 11,8 and 2 cases respectively. Conditioning regimen consisted of modified BU/CY (n=12), BU/CY (n=6), FLU/BU (n=2) and CY/ATG for SAA(n=1). In addition, ATG(Thymoglobuline, 5-7.5mg/Kg, iv, divided into three days, -8∼-6d) was given for 8 patients. Anti-CD25 was given for 13 patients, including Daclizumab (50mg/d, -1?,03?,+4?,+11d) for 7 patients and Basiliximab (20mg/d, -1?,02?,+2?,+7d) for 6 patients. GVHD prophylaxis consisted of CSA and short course of MTX. In addition, MMF was given in all patients at the dosage 1.0 g/d for one month. The donors were given granulocyte colony-stimulating factor (G-CSF) at a dosage of 5 μg/Kg.d subcutaneously for 5 days, bone marrow was collected on day 3, peripheral blood stem cells were collected at day 4 and 5. Results Engraftment was obtained in all 21 patients, the median time to WBC >0.5×109/L and BPC>20×109/L in ATG and Anti-CD25 group were 14, 13d and 13, 12d, respectively (p>0.05), I∼II aGVHD accurred in 3 cases(37.5%) in ATG group and 5 cases(23.1%) in Anti-CD25 group,III∼IV aGVHD was observed in 1 case (12.5%) in ATG group and 2 cases (15.3%) in Anti-CD25 group. cGVHD was evaluable in 14 patients who survived after day +100, Extensive cGVHD developed in 1 (20%) of 5 patients in ATG group, and in 4 (44%) of 9 patients in Anti-CD25 group. Daclizumab and Basiliximab were not cause any infusion-related toxicity. Up to now, 13 patients survived in complete remission follow up 3-59 months, 4 cases (50%) died (3 in TRM and 1 in relapse) in ATG group, 4 cases (30.7%) died (1 in TRM and 3 in relapse) in Anti-CD25 group. Conclusion The replacement of ATG with Anti-CD25 seems to be more safety and effective for the conditioning of HLA –mismatched HSCT. Disclosures: No relevant conflicts of interest to declare.

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