Similar Outcomes of Allogeneic Hematopoietic Cell Transplantation from Matched Sibling Donor and Haploidentical Donor for Refractory/Relapsed Acute Myeloid Leukemia

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 5511-5511
Author(s):  
Dai-Hong Liu ◽  
Li Yu ◽  
Wenrong Huang ◽  
Liping Dou ◽  
Honghua Li ◽  
...  
Keyword(s):  
Cohort Study ◽  
Stem Cell ◽  
Cell Transplantation ◽  
Conflicts Of Interest ◽  
Chronic Gvhd ◽  
Conditioning Regimen ◽  
Hematopoietic Stem ◽  
Platelet Recovery ◽  
Sibling Donor ◽  
Matched Sibling

Abstract Allogeneic hematopoietic stem cell transplantation (allo-HCT) is the only effective, even curative treatment for refractory/relapsed AML patients. Unmanipulated haploidentical HCT (haplo-HCT) resulted in encouraging outcomes for treatment of hematologic malignancies and become an alternative option in case of lacking HLA matched sibling donor (MSD). Unmanipulated haplo-HCT from G-CSF mobilized bone marrow and peripheral blood stem cell (PBSC) has shown similar results as that from MSD-HCT in leukemia. Here, we report the results of a cohort study on the efficacy and toxicity of haplo-PBSCT compared with MSD-PBSCT for treatment of refractory/relapsed AML. PATIENTS AND METHODS Among 419 newly diagnosed AML patients, 69 patients relapsed during CR1 and were planned to receive allo-HCT after re-induction. The order of preference of donors was MSD, matched unrelated (HLA 10/10 or 9/10 loci matched), or haploidentical donor. Thirty patients received haplo-PBSCT and 13 patients MSD-HCT (July, 2007 ~ June, 2014) at our unit. There was no difference of the characteristics of demography, disease or transplantation between these two groups (Table 1). High-resolution DNA techniques were used to evaluate the HLA-A, B, DRB1, DQB1, and C loci. Donors were treated with rhG-CSF (5 mg.kg-1.day-1) for consecutive days. The PBSCs were collected on day 5 - 6 and infused on the day of collection. The conditioning regimen consisted of Bu (9.6 mg.kg-1, intravenously, days -10 ~ -8), Carmustine, (250 mg.m-2, day -5), cytarabine (8 g.m-2, days -7 ~ -6), CY (120 mg kg-1, days -4 ~ -3), and ATG (rabbit; 10 mg.kg-1, days -5 ~ -2). MSD-HCT patients had the same conditioning regimen without ATG. All transplant recipients received cyclosporine A, mycophenolate mofetil, and short-term methotrexate for GVHD prophylaxis. The endpoint of the last follow-up for all surviving patients was June 30, 2015. RESULTS Sustained myeloid engraftment with full donor chimerism was achieved in both groups (100%) at a median of 16 (10 - 26) days. Twenty-six patients (86.7%) in haplo-PBSCT group and all patients in MSD-PBSCT group achieved platelet recovery. There was no difference of the cumulative incidence of acute GVHD grade 2-4 (Fig. 1), chronic GVHD (20% vs 33.3%, P=0.581), transplantation-related mortality (TRM) (16.7% vs 0%, P = 0.121), relapse (33.3% vs 38.5%, P = 0.578, Fig 2) between haplo-PBSCT and MSD-PBSCT group. Donor age of 41yr and older was an independent risk factor for inferior leukemia-free-survival (27.8% vs 37.2%, P = 0.004). CONCLUSION In this cohort study, haplo-PBSCT showed similar outcomes in patients with refractory/relapsed AML compared with MSD-PBSCT. It suggested the feasibility of G-CSF-primed PBSC as a graft source in unmanipulated haplo-HCT under myeloablative conditioning, which was effective and tolerable for treatment of poor risk leukemia. Table 1. Characteristics of patients and donors Haploidentical donor Matched sibling donor P value Cases % Cases % Gender, n (%) Receipt Male 22 73.3 8 61.5 0.485 Donor Male 22 73.3 7 53.8 0.292 Age,y, median(range) Patient ≤40 y, n (%) 21 70 6 46.2 0.178 Donor ≤41 y, n (%) 13 43.3 5 38.5 1.000 AML, n (%) 1.000 CR2 5 16.7 2 15.4 NR/beyond CR2 25 83.3 11 84.6 Time to transp 0.51 ≥7m 14 46.7 8 61.5 Conditioning Regimen, n (%) 0.675 BuCy 22 73.2 9 69.2 TBIcy 4 13.3 1 7.7 FB 4 13.3 3 23.1 CD34+ in graft (106/kg) 0.499 ≥4.77 17 56.7 5 41.7 Figure 1. Figure 1. Figure 2. Figure 2. Figure 3. Figure 3. Disclosures No relevant conflicts of interest to declare.

scholarly journals Similar Incidence of Severe Acute Gvhd and Less Extensive Chronic Gvhd in PBSCT from Unmanipulated Haploidentical Donor Compared with That from Matched Sibling Donor for Patients with Hematologic Malignancies

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 1964-1964
Author(s):  
Honghua Li ◽  
Wenrong Huang ◽  
Chunji Gao ◽  
Liping Dou ◽  
Fei Li ◽  
...  
Keyword(s):  
Cohort Study ◽  
Stem Cell ◽  
Acute Gvhd ◽  
Chronic Gvhd ◽  
Conditioning Regimen ◽  
Hematologic Malignancies ◽  
Hematopoietic Stem ◽  
Sibling Donor ◽  
Matched Sibling ◽  
Similar Incidence

Abstract Unmanipulated haploidentical hematopoietic stem cell transplantation (haplo-HCT) resulted in encouraging outcomes for treatment of hematologic malignancies and become an alternative option in case of lacking HLA matched sibling donor (MSD). Transplantation with G-CSF mobilized peripheral blood stem cell (PBSCT) has been a stable transplant setting with MSD. Unmanipulated haploidentical donor (haplo-PBSCT) has been applied in patients with hematologic malignancies. However, the characteristics of graft-versus-host disease (GVHD) in unmanipulated haplo-PBSCT are not clear. Here, we report the results of a cohort study on the clinical features of acute and chronic GVHD in haplo-PBSCT compared with PBSCT from MSD in patients with hematologic malignancies. PATIENTS AND METHODS Between July, 2007 and June, 2014, 94 patients with hematologic malignancies received haplo-PBSCT and 100 patients received PBSCT from MSD consecutively at our unit (Table 1). The PBSCs were collected on day 5 and 6 after 4 days of rhG-CSF (5 mg.kg¨C1 °¤day¨C1), then were infused on the day of collection. The conditioning regimen consisted of Bu (9.6 mg.kg-1, iv, days -10~-8), Carmustine, (250 mg.m-2, day -5), cytarabine (8 g.m-2, days -7~-6), CY (120mg kg-1, days -4~-3). Antithymoglobulin (ATG, rabbit; 10 mg.kg-1, days -5~-2) was used for haplo-PBSCT. All recipients received cyclosporine A, mycophenolate mofetil, and short-term methotrexate for GVHD prophylaxis. The endpoint of the last follow-up for all surviving patients was July 15, 2015. RESULTS Among the patients with acute GVHD, there was no difference of the rate of the involved organs between these two groups (skin: 67.4% vs 68.0%, p=1.000; liver: 15.2% vs 20.0%, p=0.742; gut: 33.3% vs 32%, p=1.000). Haplo-PBSCT was associated with higher incidence of acute GVHD grade 2-4 (HR: 3.04, 95% CI: 1.55-5.98, p=0.001) and lower incidence of extensive chronic GVHD (HR: 0.49, 95% CI: 0.24-0.99, p=0.047) compared with MSD PBSCT. There was no difference of the incidence of acute GVHD grade 3-4 between these two groups (haplo-PBSCT, 9.6% vs MSD PBSCT 8.9%, p=1.000). According to NIH criteria, the incidence of severe chronic GVHD was lower in haploidentical group (13.6%) compared with MSD group (40.5%, p=0.041). There was no difference of those for mild and moderate chronic GVHD (mild: 27.3% vs 13.5%, p=0.189; moderate: 59.1% vs 45.9%, p=0.422). CONCLUSION In this cohort study, haplo-PBSCT was associated with similar incidence of severe acute GVHD, lower extensive chronic GVHD and lower severe chronic GVHD compared with MSD-PBSCT. It suggested the potential advantage of ATG in improvement of long-term quality of life of the transplant recipients. Table 1. Characteristics of patients and donors Haploidentical donor, n = 94 Matched sibling donor, n = 100 P value Gender, n (%) Receipt, male 73 (77.7) 64 (64.0) 0.041 Donor, male 63 (67.0) 60 (60.0) 0.371 Age Patient, y, median 27 38 0.055 Donor, y, median 38 39 0.364 Hematologic malignances, n (%) Acute leukemia 72 (76.6) 61 (61.0) MDS 3 (3.2) 21 (21.0) 0.000 CML 5 (5.3) 10 (10.0) Lymphoma 14 (14.9) 8 (8.0) Status of disease, n (%) 0.000 CR1 42 (44.7) 76 (76) CR2 14 (14.9) 5 (5) NR/beyond CR2 38 (40.4) 19 (19) Time to transplant (d) 361 299 0.946 Conditioning Regimen, n (%) 0.354 BuCy 60 (63.8) 66 (66.0) TBIcy 28 (29.8) 23 (23.0) FB 6 (6.4) 11 (11.0) CD34+ in graft (106/kg) 5.86 4.77 0.057 ≥4.60 51 (54.3) 41 (41.0) 0.084 Disclosures No relevant conflicts of interest to declare.

scholarly journals HLA-Haploidentical Donors Seem to be Not Inferior to HLA-Identical Sibling Donors for Hematopoietic Stem Cell Transplantation of Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)?

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 5867-5867
Author(s):  
Feng Chen ◽  
Depei Wu ◽  
XiaoWen Tang ◽  
Miao Miao ◽  
Chengcheng Fu ◽  
...  
Keyword(s):  
Stem Cells ◽  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Conflicts Of Interest ◽  
Chronic Gvhd ◽  
Conditioning Regimen ◽  
Short Term ◽  
Hematopoietic Stem ◽  
Alternative Option

Abstract Background PNH is an acquired clonal disorder of the hemopoietic stem cells for which the only curative treatment is allogeneic hematopietic stem cell transplantation (allo-HSCT). But allo-HSCT is challenging for those who have no HLA-matched donors.Several recent studies have shown that haploidentical HSCT for patients with hematological malignancy can achieved comparable outcomes with HLA-identical sibling transplantation . There are very few reports on the use of haploidentical HSCT for PNH . Is haploidentical HSCT a valid alternative option for patients with PNH? Methods 19 PNH patients received allo-HSCT between Dec 2007 and Oct 2015 at our institution. 12 donors were HLA-haploidentical and 7 were HLA-matched siblings. The patients were aged 8 to 54 years (median 28 years) . Of the 12 haploidentical donors, 6 were siblings, 2 fathers,2 mothers,1 son and 1 daughter. 12 patients with haploidentical donors received a myeloablative conditioning regimen consisting of busulfan, cyclophosphamide and ATG (anti-thymocyte globulin), 7 patients with identical siblings were given a reduced intensity conditioning. G-CSF-mobilized bone marrow and peripheral blood stem cells were transplanted as graft. Prophylaxis for graft-versus-host disease (GVHD) consisting of cyclosporine or tacrolimus + short-term methotrexate + mycophenolate mofetil was used for 12 patients with haploidentical donors, and cyclosporine + short-term methotrexate for 7 with identical siblings. Results All 19 patients were engrafted successfully. The median time of neutrophils (ANC) reached to 0.5×109/L and platelets (PLT) reached to 20×109/L was 12 days and 15 days in haploidentical group , and that to identical group was 11 days and 13 days ,respectively. There were 2 patients developed grade Ⅱ acute GVHD in haploidentical group while 1 patients with grade Ⅳ aGVHD in identical group . Limited chronic GVHD was observed in 2/12 patients in haploidentical group and 1/7 patients in identical group. After a median follow-up time of 22.0 (range 4.0-42.0) months, the 3-year OS probability was 77.8±13.9% and 85.7±13.2% for haploidentical and identical group,respectively (P=0.03). 2 patients died of treatment-related mortality in haploidentical group, including severe pulmonary infection (n=1) and transplant-associated thrombotic microangiopathy (n=1) ,and 1 died of severe aGVHD in identical group. No patients were documented to have a recurrence of a PNH clone after HSCT in both groups. Conclusion This report seemed that long-term outcomes of HLA- haploidentical HSCT in patients with PNH were comparable to that of HLA- matched donor at our institution . Haploidentical HSCT should be considered as a valid alternative option for PNH patients without HLA- matched donors . Disclosures No relevant conflicts of interest to declare.

Outcome of Allogeneic Stem Cell Transplantation for Patients with Severe Aplastic Anemia after Conditioning with Procarbazine, ATG and Cyclophosphamide: A Single Center Experience.

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 2027-2027
Author(s):  
Ki Seong ◽  
Jong Wook Lee ◽  
Yoo Jin Kim ◽  
Hee Je Kim ◽  
Chang Ki ◽  
...  
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Aplastic Anemia ◽  
Cell Transplantation ◽  
Chronic Gvhd ◽  
Univariate Analysis ◽  
Conditioning Regimen ◽  
Severe Aplastic Anemia ◽  
Platelet Recovery ◽  
Matched Sibling

Abstract Background: Cyclophosphamide (CY, 200 mg/kg) plus ATG seems to be accepted as a standard conditioning regimen in HLA-matched sibling stem cell transplantation (SCT) for severe aplastic anemia (SAA). We report herein the outcome of allogeneic SCT for the patients with SAA conditioned with procarbazine (PCB), ATG and CY. Methods: Between January 1995 and March 2004, consecutive one hundred and sixty two patients with SAA received matched sibling SCT after conditioning with PCB, ATG and CY. The median age of patients was 28.5 (16~50) and median interval between diagnosis and SCT was 10.5 months (1~216). Fifty-five patients (34%) had a history of immunosuppressive therapy before SCT. The conditioning regimen consisted of PCB (6.25 mg/kg/day, 6 days), CY (50 mg/kg/day, 4 days) and ATG (1.25 mg/kg/day, 3 days). No graft manipulation was performed in 92 patients (52%), and donor marrow expansion and megadose transplantation (bone marrow plus CD34+ isolated PB as a stem cell source) were done in 35 patients (21.6%). All patients received of cyclosporine and methotrexate as GVHD prophylaxis. Results: The median dose of CD34+ and CD3+ cells infused were 4.8 x 106/kg (0.8~49.6) and 5.1 x 107/kg (0 ~ 38), respectively. Median time to neutrophil recovery more than 500/μL and platelet recovery more than 20,000/μL were 13 days (0~49) and 19 days (0~150), respectively. All patients achieved successful primary engraftment, but delayed engraftment failure developed in 18 patients (11%). Ten among 18 patients who developed graft failure received second SCT, resulting survival of 9 patients. The incidence of acute (more than grade II) and chronic GVHD were 9.3% (n=15) and 13.0 % (n=21), respectively. DFS and overall survival (OS) at 6 years was also 90%. Factors influencing on DFS on univariate analysis were patient and donor’s age, development of acute and chronic GVHD, and times of pregnancy (over 2 times). Development of acute and chronic GVHD, and occurrence of rejection were factors that influence DFS on multivariate analysis. Causes of death (n=15) were pneumonia (n=4), rejection (n=4), GVHD±infection (n=2), chronic GVHD (n=1), MOF (n=1), CVA (n=1), autoimmune hemolytic anemia (n=1), and unknown (n=1). Conclusions: These data suggest that the conditioning regimen used in this study is feasible for patients with SAA who received matched sibling SCT. GVHD and rejection were the most important factors that influence DFS.

Stem Cell Transplantation from HLA-Identical Siblings Versus Alternative Donors in β-Thalassemia Diseases.

Blood ◽  
2006 ◽  
Vol 108 (11) ◽  
pp. 3157-3157
Author(s):  
Preeda Vanichsetakul ◽  
Panya Seksarn ◽  
Issarang Nuchprayoon
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Chronic Gvhd ◽  
Conditioning Regimen ◽  
Disease Free Survival ◽  
Thalassemia Major ◽  
Hematopoietic Stem ◽  
Unrelated Donors ◽  
Matched Sibling

Abstract Background: Allogeneic hematopoietic stem cell transplantation (SCT) has been established as a curative therapy for β-thalassemia major which is prevalent among Thais. Donors are usually patients’ siblings but the chance to find an optimal HLA-identical is just about 30%. Thus clinical trial using alternative donors such as partially-mismatched related donors or matched unrelated donors could be a good choice. Methods: To study the efficacy and results, we retrospectively review our experience with β-thalassemia pediatric patients undergoing transplantation from July 1999 to July 2006. Results: There were 43 patients categorized into HLA-identical siblings group (n=26, 13 male and 13 female) and alternative donors group (n=17, 14 male and 3 female). Median age and weight were 7.2 years (1.2–14.8 years), 19.8 kg (9.8–34 kg) and 7.7 years (1.4–14.8 years), 21.5 kg (11–33 kg), respectively. Amongst 26 matched siblings there were 17 bone marrow (BM), 4 peripheral blood stem cell (PBSC), and 5 cord blood (CB) donors. Of the alternative group stem cells derived from 12 matched unrelated, 2 two-antigen mismatched sibling CB, 2 one-antigen mismatched unrelated CB, and 1 one-allele mismatched unrelated BM donors. Myeloablative conditioning regimen consisted of busulfan (oral or intravenous) and cyclophosphamide for matched siblings SCT. Anti-thymocyte globulin was added for alternative donors SCT. Fludarabine was also used in addition for unrelated donors SCT. Graft-vs-host disease (GvHD) prophylaxis consisted of cyclosporine plus methotrexate except for unrelated SCT that tacrolimus plus methotrexate were used instead. Median numbers of infused CD34+ cells of the matched siblings group were 11.1x106/kg (3.7–35.4) in BMT (n=17), 9.3x106/kg (7.1–12.8) in PBSCT (n=4), and 0.94x105/kg (0.2–5.3) in CBT (n=5), compared to 5.5x106/kg (1.5–18.1) in BMT (n=10), 11.1x106/kg (11–17.2) in PBSCT (n=3), and 2.1x105/kg (1.5–3) in CBT (n=4) of the alternative group. Median times to neutrophil and platelet engraftments in each groups were 15 and 33 days, compared to 17 and 41 days, respectively. 39 successful donor engraftments were achieved from 38 HLA-matched (25 related and 13 unrelated) donors and from 1 mismatched unrelated CB unit. The remaining 1 of the matched related group and 3 of the alternative group did not engrafted, all but one case subsequently resumed autologous recovery. Acute GvHD occurred in 4 matched-sibling and 5 unrelated SCT recipients. After immunosuppressive therapy the lesions were completely resolved in all but two patients, one who suffered from severe grade IV intestinal GvHD and another who later developed extensive chronic GvHD which required treatment longer than 4 years. There were 4 mortalities only in the alternative group: 3 patients died post-engraftment due to severe fatal GvHD (n=1), cerebral aspergillosis (n=1), severe veno-occlusive disease with liver failure (n=1; Pesaro class 3); and one died from neutropenic sepsis 3-week after 4/6 matched related CBT. Median follow-up time for surviving patients was 53.5 months (2–84 months). Overall and disease-free survival in HLA-matched SCT (n=38) recipients were 94.7% (n=36) and 92.1% (n=35), compared to in HLA-mismatched (n=5) 60% (n=3) and 20% (n=1), respectively. Conclusions: SCT for β-thalassemia major using HLA-matched donors, either related or unrelated, had superior results than HLA-mismatched. In situation of unavailable matched sibling donors, the search for an appropriate complete HLA-matched unrelated volunteer donor using high resolution DNA typing is essential for favorable outcomes.

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 ◽  
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.

Haploidentical Stem Cell Transplantation for Patients Relapsing after First Allogeneic Transplantation

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 4618-4618
Author(s):  
Virginie Lavoipierre ◽  
Samia Harbi ◽  
Luca Castagna ◽  
Angela Granata ◽  
Sabine Fürst ◽  
...  
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Cumulative Incidence ◽  
Conflicts Of Interest ◽  
Chronic Gvhd ◽  
Conditioning Regimen ◽  
Good Survival ◽  
Hematopoietic Stem ◽  
Second Transplantation

Abstract INTRODUCTION: Relapse after allogeneic hematopoietic stem cell transplantation (alloHSCT) remains a major therapeutic challenge: outcome is very poor, without curative option in most cases. Second alloHSCT may be considered in few selected patients because of anticipated limitations: 1) donor availability; 2) high toxicity due to previous treatments; 3) low efficacy considering the very advanced disease situation. We hypothesized that the use of post transplantation Cyclophosphamide (pCY) haplo-SCT may be an interesting alternative to overcome these limitations. In particular, the presence of full haplotype HLA mismatch could provide a decisive antileukemic effect relative to alloreactivity. In absence of large series in this setting, we report here the outcome after HaploSCT for patients who relapse after a first alloHSCT. METHODS: We retrospectively studied adult patients, who received a second pCy Haplo-SCT for hematological malignancies. Patients were treated between 2009 and 2016. The objective was to assess both the feasibility and the efficacy of HaploSCT in this setting. RESULTS: Twenty seven patients were included: median time between first alloHSCT and relapse was 11 months (range: 1-82). Median age at second transplantation was 49 years old (range: 21-61). Most of patients had acute myeloid leukemia (n=12, 44%) or Hodgkin lymphoma (n=6 patients, 22%). Fifteen patients (55%) were in complete remission at the time of pCY Haplo-SCT. Hematopoietic cell transplantation-comorbidity index was ≥ 3 in 20 patients (74%). Thirteen patients (48 %) received non-myeloablative conditioning regimen (as Baltimore schema, Luznik et al. BBMT 2008) prior to HaploSCT while remaining patients received busulfan-based regimen. Day+100 cumulative incidence of grade 2 to 4 and 3 to 4 acute GVHD was 15% and 7%. 2-year cumulative incidence of chronic GVHD was 12%. The cumulative incidence of non-relapse mortality and relapse at 2 years were 38% and 27%, respectively. With a median follow up of 25 months (range: 4-63), 2-year progression-free and overall survivals were 36% and 39%, respectively. Disease status at the time of HaploSCT was a major determinant for outcome. Indeed, 2-year NRM and OS were 58% and 25% in patients transplanted with active disease, respectively, while corresponding values in patients transplanted in CR were 21% (p=0.036) and 49% (p=0.041), respectively (Figure 1A and 1B). CONCLUSION: We can conclude that in selected patients who could be candidate for second transplantation, HaploSCT is feasible and may represent a curative option. The overall incidence of relapse of 27% is promising in this situation for which no alternative for cure is available, with relatively good survival in patients transplanted in CR. However, the very high NRM (58%) in refractory patients should make us consider second transplant with caution in this setting. For these patients, specific developments are needed to avoid procedure-related toxicity. Disclosures No relevant conflicts of interest to declare.

Allogeneic hematopoietic stem cell transplantation in thalassemia major: results of a reduced-toxicity conditioning regimen based on the use of treosulfan

Blood ◽  
2012 ◽  
Vol 120 (2) ◽  
pp. 473-476 ◽  
Author(s):  
Maria Ester Bernardo ◽  
Eugenia Piras ◽  
Adriana Vacca ◽  
Giovanna Giorgiani ◽  
Marco Zecca ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Cumulative Incidence ◽  
Chronic Gvhd ◽  
Conditioning Regimen ◽  
Unrelated Donor ◽  
Hematopoietic Stem

Abstract Sixty thalassemia patients (median age, 7 years; range, 1-37) underwent allogeneic hematopoietic stem cell transplantation (HSCT) after a preparation combining thiotepa, treosulfan, and fludarabine. Before HSCT, 27 children were assigned to risk class 1 of the Pesaro classification, 17 to class 2, and 4 to class 3; 12 patients were adults. Twenty patients were transplanted from an HLA-identical sibling and 40 from an unrelated donor. The cumulative incidence of graft failure and transplantation-related mortality was 9% and 7%, respectively. Eight patients experienced grade II-IV acute GVHD, the cumulative incidence being 14%. Among 56 patients at risk, 1 developed limited chronic GVHD. With a median follow-up of 36 months (range, 4-72), the 5-year probability of survival and thalassemia-free survival are 93% and 84%, respectively. Neither the class of risk nor the donor used influenced outcome. This treosulfan-based preparation proved to be safe and effective for thalassemia patients given allogeneic HSCT.

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

Blood ◽  
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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