Similar Survival, but Better Quality of Life After Myeloablative Transplantation Using Unrelated Cord Blood Versus Identical Sibling Hematopoietic Cells in Adults with Hematologic Malignancies: A Single Institute Analysis.

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 3134-3134
Author(s):  
Huilan Liu ◽  
Liangquan Geng ◽  
Xingbing Wang ◽  
Kaiyang Ding ◽  
Juan Tong ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Cord Blood ◽  
Cumulative Incidence ◽  
Research Funding ◽  
Chronic Gvhd ◽  
Hematologic Malignancies ◽  
Hematopoietic Stem ◽  
Similar Survival ◽  
Unrelated Cord Blood

Abstract Abstract 3134 HLA-identical sibling allogeneic hematopoietic stem cell transplantation (HSCT) is the best therapeutic option for high-risk hematological disease, but it is limited by lack of availability of a suitable donor for most candidate patients. At our institution our center, unrelated cord blood transplantation (UCBT) was used as the first alternative option for patients who lack an HLA-matched related donor in the last decade. We retrospectively analyzed the clinical outcomes of UCBT versus HLA-identical sibling HSCT for adult patients with hematologic malignancies. Between April 2000 and May 2012, 194 consecutive adult patients with malignant hematological diseases received UCBT (77 patients; median age 21y, r 14–46; male 49) and related bone marrow transplantation (BMT) and/or peripheral blood stem cells transplantation (PBSCT) (117 patients; median age 30y, r 14–53; male 75). Diagnoses in two groups (UCBT/Sibling) included de novo AML (n = 23/32), ALL (n = 30/26), CML (n = 17/47), MDS/AML (n = 3/8), mixed phenotype acute leukemia (MPAL) (n = 3/1), NHL (n = 1/2), and MM (n = 0/1)). Overall rates of high-risk patients were 84.4% for UCBT recipients and 50.4% for BMT/PBSCT recipients(P<0.05). 11.6 percent of UCB grafts and 100% sibling grafts were HLA-identical (antigen level for HLA-A and B, allelic for DRB1, respectively). All patients received myeloablative conditioning regimens and CsA plus mycophenolate mofetil as GVHD prophylaxis. The median number of nucleated cells and CD34+ cells infused were 0.36 (r 0.19–0.69)×108 and 0.21(r 0.06–0.52)×106 per kilogram of the recipient's body weight for UCBT recipients, and 5.68 (r 2.02–12)×108 and 3.63(r 0.67∼14.99)×106 per kilogram for BMT/PBSCT recipients (P=0.000). The primary engraftment rate was 96.1% (UCBT) and 100% (BMT/PBSCT). The median time to 0.5×109/L ANC was 19 days (r 12–35) and 12 days(r 10–18)(P = 0.000) and the median time to 20×109/L platelets was 36 days(r 14–90)and 15 days(r 11–17)(P = 0.001); hematopoietic recovery was significantly delayed after UCBT. The cumulative incidence of total acute GVHD (47.3% vs.22.2%, P = 0.001), early transplantation-related mortality (TRM) before day +100 (13.0% vs.3.4%, P<0.05), and infection (58.4%vs. 12.0%, P<0.05) was higher in UCBT recipients. Median follow-up was 42.2 months (range, 4.5–123.2 months) for BMT/PBSCT recipients and 22.2 months (range, 3.0–110.2 months) for UCBT recipients. There was no significant difference in cumulative incidence of grades ?to ? acute GVHD (9.4% vs. 5.0% P = 0.247), relapse rate (14.3% vs. 14.5%, P=0.962), and 3 year over survival (OS) (59.6% vs. 64.1%, P=0.123). But the cumulative incidence of chronic GVHD (18.8% vs. 44.1%) and extensive chronic GVHD (0% vs. 18%) was lower in UCBT recipients (P<0.05). The estimated 9-M CI of discontinuing immunosuppressive therapy (IST) while alive was 80%, and long-term survivors of UCBT had better quality of life. Our data shows similar survival, but better quality of life after myeloablative transplantation using UCB versus identical sibling hematopoietic cells in adults with hematologic malignancies. HLA-mismatched cord blood should be considered an acceptable source of hematopoietic stem-cell grafts for adults in the absence of an HLA-matched adult donor. Disclosures: Sun: Anhui Provincial “115” Industrial Innovation Program (2009): Research Funding; the Fund of the Key Medical Project of Anhui Provincial Healthy Department (2010A005): Research Funding; Key Scientific and Technological Project of Anhui province “Twelfth Five-Year Plan” (11010402164): Research Funding.

Comparative Analysis of Single-Institute Transplantation From Unrelated Cord Blood and Related Adult Donors In Adults with Hematologic Malignancies.

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 4570-4570
Author(s):  
Zimin Sun ◽  
Huilan Liu ◽  
Liangquan Geng ◽  
Xingbing Wang ◽  
Kaiyang Ding ◽  
...  
Keyword(s):  
Cord Blood ◽  
Research Funding ◽  
Cord Blood Transplantation ◽  
Chronic Gvhd ◽  
Disease Free Survival ◽  
Hematologic Malignancies ◽  
Post Transplantation ◽  
Peripheral Blood Stem Cells ◽  
Hematopoietic Stem ◽  
Unrelated Cord Blood

Abstract Abstract 4570 We compared the clinical outcomes of allogeneic hematopoietic stem-cell transplantation among 126 adults with hematologic malignancies: 40 received unrelated cord blood transplantation (CBT) and 86 received related bone marrow transplantation (BMT) and/or peripheral blood stem cells transplantation (PBSCT). All patients received myeloablative transplantations which performed from 2001 through 2010.Recipients of unrelated CBT were younger (median, 23.5 vs. 30 years of age; P<0.001) and had more advanced disease at the time of transplantation (68% vs. 34%, P<0.001) than recipients of related BMT/PBSCT. All related transplants were HLA matched, whereas 77 percent of CBT grafts were HLA mismatched (P<0.001). The median number of nucleated cells infused was 0.37×108 per kilogram of the recipient's body weight for CBT and 5.39×108 per kilogram for BMT/PBSCT recipients (P<0.001). Neutrophil recovery was significantly delayed after CBT (median, 19 vs. 12 days post transplantation, P<0.001), however, overall engraftment rates were almost the same for both grafts. Multivariate analysis demonstrated no apparent differences in transplantation-related mortality (TRM) (33% in CBT and 21% in BMT/PBSCT recipients), relapse rate (15% in CBT and 15.1% in BMT/PBSCT recipients), the incidence of grades III to ‡W acute graft-versus-host disease (aGVHD) (7.5% in CBT and 5.8% in BMT/PBSCT recipients), 2 years disease free survival (DFS) (52.5% in CBT vs. 62.8% in BMT/PBSCT recipients) and over survival (OS) (62.5% in CBT vs. 67.4% in BMT/PBSCT recipients) between both groups, but extensive chronic GVHD (cGVHD) was lower in CBT recipients (2.5% vs. 17.4%). These data suggest that unrelated CBT could be as safe and effective as matched related BMT or mobilized PBSCT for adult patients with hematologic malignancies. Disclosures: Sun: International Cooperation Research Fund of Anhui Provincial Scientific and Technologic Committee (08080703026): Research Funding; Fund of the Key Medical Project of Anhui Provincial healthy department (2010A005): Research Funding; Anhui Provincial “115” Industrial Innovation Program: Research Funding.

Outcomes after Unrelated Cord Blood Transplantation in Children with Acute Lymphoblastic Leukemia. An Eurocord-Netcord Survey.

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 304-304 ◽  
Author(s):  
Vanderson Rocha ◽  
Gerard Michel ◽  
Nabil Kabbara ◽  
William Arcese ◽  
Juan Ortega ◽  
...  
Keyword(s):  
Cord Blood ◽  
Cumulative Incidence ◽  
Lymphoblastic Leukemia ◽  
Cord Blood Transplantation ◽  
Chronic Gvhd ◽  
Conditioning Regimen ◽  
Unrelated Cord Blood Transplantation ◽  
Blood Transplantation ◽  
Advanced Phase ◽  
Unrelated Cord Blood

Abstract Unrelated cord blood transplantation (UCBT) is an alternative option to treat children with haematological diseases without an HLA-identical donor. We have analyzed a total of 323 children with ALL receiving an UCBT, from 1994 to 2004 in 99 transplant centres in 24 countries, mostly in Europe. Cumulative incidence with competing risk and KM estimates were used to calculate outcomes. Seventy six children were transplanted in CR1, 136 in CR2 and 111 in more advanced phase of the disease. Among those children poor cytogenetics were observed in 89% of children in 1CR, 33% in 2CR and 42% in advanced phase. Twenty percent of children transplanted in advanced phase had been previously autografted. The median age was 6.5 years at UCBT, median cell dose infused was 4.1x107/kg and the median follow time was 22 months (3–96). The cord blood was HLA identical (6/6) in 12% of the cases, 5/6 in 46%, 4/6 in 39% and 3/6 in 3%. All children received myeloablative conditioning regimen (TBI in 66%) and the majority (67%) received CsA+corticoids as GVHD prophylaxis. Cumulative incidence of neutrophil recovery at day 60, platelets recovery (&gt;20.000) at day 180, acute (grade II–IV) and chronic GVHD were 76±5%, 54±5%, 42±3%, 14±2%, respectively. Overall 2 year-LFS was 36±3%. In a multivariate analysis, only CR1 or CR2 were associated with better LFS (HR=1.8; p&lt;0.0001). Outcomes CR1 (n=76) CR2 (n=136) Advanced (n=111) TRM at day 100 22+/−5% 25+/−4% 34+/−5% Relapse at 2 years 34+/−8% 37+/−5% 48+/−7% LFS at 2 years 42+/−6% 41+/−4% 24+/−4% For those patients transplanted with poor cytogenetics, LFS at 2 years was 32±6% and it was 37% for CR1, 43% for CR2 and 0% for advanced phase of the disease. In conclusion, in these large series of high risk ALL patients, these results show that UCBT should be proposed as alternative source of allogeneic transplantation for children lacking an HLA identical donor, in earlier status of the disease.

Irradiation-Based Myeloablative Cord Blood Transplantation for Hematologic Malignancies In Adults.

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 4583-4583
Author(s):  
Huilan Liu ◽  
Xingbing Wang ◽  
Liangquan Geng ◽  
Kaiyang Ding ◽  
Baolin Tang ◽  
...  
Keyword(s):  
Cord Blood ◽  
Total Dose ◽  
Research Funding ◽  
Cord Blood Transplantation ◽  
Chronic Gvhd ◽  
Hematologic Malignancies ◽  
Adult Patients ◽  
Pcr Analysis ◽  
Myeloablative Conditioning ◽  
Blood Transplantation

Abstract Abstract 4583 We retrospectively analyzed of the engraftment, Transplant-related complications and survival after unrelated cord blood transplantation (UCBT) using irradiation-based myeloablative conditioning in adult with hematologic malignancies. Between September 2006 and June 2010, 29 consecutive adult patients with hematological malignancies were treated with UCBT, Thirteen of them were advanced-stage disease and 11 of them were high risk or refractory disease. All patients received four fractionated 12 Gy TBI, total dose 12 g/m2 cytarabine and total dose 120 mg/kg cyclophosphamide as myeloablative conditioning. The median age was 21 years; the median weight was 57 kg. Double or multiple UCB grafts were used for 17 patients, while single UCB graft for 12 patients. the median number of nucleated cells was 3.83×107/kg and 5.25×107/kg. All patients were given a combination of cyclosporine A (CsA) and mycophenolate mofetil (MMF) for graft-versus-host disease (GVHD) prophylaxis. All patients obtained engraftment. Median time to neutrophil≥0.5×109/L was 20 days (range 14–37) and platelet engraftment in 25 patients (≥20×109/L) was 35 days (range 25–49). Chimerism was assessed by PCR analysis of short tandem repeat (STR) sequences on whole blood or bone marrow weekly in cases who were fully donor chimeric from 7 days to 21 days after transplantation. 16 cases developed acute GVHD, more than grade II in three cases. Six of twenty-four patients who survived more than 100 days developed limited chronic GVHD.17 cases were alive and in hematologic remission at a median follow-up of 504 days (range 49 ~ 1245). The probability of over survival at 3 years was 58.6%. Five cases relapsed. Nine of twelve cases died of transplant related complications and infection. These results suggest that UCBT after TBI-based myeloablative conditioning could be safely and effectively used for adult patients with hematologic malignancies. Disclosures: Sun: the Fund of the Key Medical Project of Anhui Provincial healthy department (2010A005): Research Funding; the Clinical Technology foundation of Anhui Provincial healthy department (2008A011): Research Funding; the Fund of Anhui Provincial “115” Industrial Innovation Program: Research Funding.

Long-Term Follow-up of Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Severe Alpastic Anemia After Cyclophosphamide Plus Antithymocyte Globulin Conditioning Regimen,

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 4090-4090
Author(s):  
Johanna Konopacki ◽  
Raphael Porcher ◽  
Marie Robin ◽  
Sabine Bieri ◽  
Jean Michel Cayuela ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Chronic Gvhd ◽  
Conditioning Regimen ◽  
Hematopoietic Stem ◽  
Gvhd Prophylaxis ◽  
Increased Risk ◽  
Long Term Follow Up

Abstract Abstract 4090 Background: Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) from an HLA- identical sibling is the treatment of choice for young patients with acquired severe aplastic anemia (SAA). Due to increased rates of secondary solid cancer in patients with SAA who received an irradiation-based conditioning regimen, we decided 2 decades ago to use the association of Cyclophosphamide (CY) and Antithymocyte globulin (ATG). We report here the long-term follow-up of patients who underwent HSCT from an HLA-identical related donor after this conditioning regimen. Patients and Methods: 61 consecutive patients with SAA who received a first transplantation from June 1991 to February 2010 in our center were included. Patients with Fanconi anemia or other congenital bone marrow failure were excluded. The conditioning regimen consisted in CY (200mg/Kg) and ATG (2.5 mg/kg/day × 5 days). The donors were HLA-identical siblings in 60 cases and family HLA-matched in 1 case. Graft-versus -host disease (GvHD) prophylaxis associated cyclosporine and methotrexate (days 1, 3, 6 and 11). Long-term clinical outcome, immune recovery and quality of life were assessed. Results: The median age was 21 years [range: 4–43], 41 being adults. Median duration of the disease before HSCT was 93 days. Most of the patients had idiopathic aplastic anemia (n=49, 80%). Median time from diagnosis to HSCT was 3 months (range, 1 to 140). All but 2 patients received bone marrow as source of stem cells and all but 2 engrafted (primary graft failure= 3.4%) with a neutrophils count > 0.5 G/L and a platelets count >20 G/L after a median of 23 (range, 19 to 43) and 21 days (range, 10 to 177), respectively. In patients who had achieved neutrophil recovery, no secondary graft failure was observed. Cumulative incidence (CI) of acute grade II-IV GvHD was 23% (95%CI, 13 to 34) and 18 patients developed chronic GvHD (CI: 32%, 95% CI, 20 to 46). In multivariate analysis, a higher number of infused CD3 cells was associated with an increased risk of developing chronic GvHD (p=0.017). With a median follow-up of 73 months (8 to 233), the estimated 6-year overall survival was 87% (95%CI, 78 to 97). At 72 months, the CI of secondary malignancies was 9%, 10 patients developed avascular necrosis (21% CI), 12 patients were diagnosed with endocrine dysfunctions (19% CI) and 5 presented cardiovascular complications (CI of 10%). The CI of bacterial, fungal and viral infections were 25% (95% CI, 15 to 36), 8% (95% CI, 3 to 17) and 61% (95% CI, 46 to 73) at 72 months, respectively. At 2 years post HSCT, the immune reconstitution was normal for CD3, CD8 T-cells, B-cell and NK-cell but still incomplete for CD4 T-cells. A FACT-BMT questionnaire of quality of life (QOL) was sent to all survivors (n= 53) of who 26 accepted to respond to the questionnaire. There was no evidence for a change in QOL perception with time after transplantation. Our data were compared with those obtained from HSCT recipients from a Swiss transplant center (n=125 patients), mainly transplanted for hematological malignancies. The perception of QOL in patients who were transplanted for SAA was similar to the group of patients who were transplanted for other reason than SAA. Conclusions: Our results confirm that HSCT from HLA-identical sibling donors after CY-ATG conditioning regimen is a curative treatment for patients with SAA, with an excellent long-term outcome. We found an increased risk of chronic GvHD associated with the number of infused CD3 cells. Furthermore, we also found non negligible late complications as well as a similar quality of life with patients transplanted for hematological malignancies. Improving long-term health conditions must thus be a priority field for research, exploring the use of new conditioning regimen as well as new GvHD prophylaxis to improve the quality of life post HSCT of such patients. Disclosures: Peffault de Latour: Alexion: Consultancy, Research Funding.

Unrelated Cord Blood Does Not Increase An Overall Risk Of Invasive Fungal Infections Compared To Unrelated Bone Marrow: A Single Center Retrospective Analysis For 749 Recipients

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 4541-4541 ◽  
Author(s):  
Yuki Asano-Mori ◽  
Hideki Araoka ◽  
Muneyoshi Kimura ◽  
Daisuke Kaji ◽  
Hikari Ota ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Cord Blood ◽  
Cumulative Incidence ◽  
Fungal Infections ◽  
Chronic Gvhd ◽  
Antifungal Prophylaxis ◽  
Invasive Fungal Infections ◽  
Systemic Corticosteroids ◽  
Grade Ii ◽  
Unrelated Cord Blood

Background Invasive fungal infections (IFIs) are of great concern after allogeneic hematopoietic stem cell transplantation (HSCT), the risk of which is considered to be particularly prominent among cord blood transplantation (CBT) recipients. Patients and Methods We retrospectively analysed the records of 749 adult patients who underwent CBT or unrelated bone marrow transplantation (uBMT) for the first time at the Toranomon Hospital between 2002 and 2012, and who had neither prior history nor suspicious findings of IFIs. As prophylaxis for IFIs, fluconazole (FLCZ) or itraconazole (ITCZ) capsules were conventionally used until around 2006, which were then changed to newer mold-active agents including ITCZ oral solution, voriconazole or micafungin after their approval in Japan, the choice of which was subjected to physician's discretion. Results Engraftment achieved in 418 CBT patients and 198 uBMT patients with a significantly longer neutropenic period in CBT patients (median 20 days vs 18 days, P<0.001), whereas 37 patients underwent re-transplantation and 96 died before engraftment. The incidences of grade II-IV acute graft-versus-host disease (GVHD) and extensive chronic GVHD were significantly less frequent after CBT compared to uBMT (40% vs 54% and 17% vs 46%, both P<0.001). Systemic corticosteroids at ≥0.5mg/kg/day was given in fewer CBT patients compared to uBMT patients (59% vs 66%, P=0.07). The median durations of immunosuppressants and antifungal prophylaxis were significantly shorter in CBT patients compared to uBMT patients (118 days vs 302 days and 315 days vs 491 days, both P<0.001). As prophylaxis for IFIs, 194 CBT patients and 91 uBMT patients received FLCZ/ITCZ capsules, while 341 CBT patients and 123 uBMT patients received newer mold-active agents. Seventy-nine patients (57 in CBT and 22 in uBMT) developed IFIs with a cumulative incidence of 12.2%, at a median of 27 (1-1646) days after HSCT. About 60% of the patients developed IFIs by day 50 and the percentage reached more than 90% until 1 year. The median onset was significantly earlier in CBT patients compared to uBMT patients (day 19 vs day 61, P=0.007). The cumulative incidence of IFIs was significantly higher in CBT patients compared to uBMT patients during 50 days after HSCT (7.9% vs 3.8%, P=0.04), but became significantly lower thereafter until 1 year (2.7% vs 6.9%, P=0.02), and an overall incidence was almost similar between the 2 groups (12.6% vs 11.6%, P=0.58) (Figure 1). Four patients had 2 infectious episodes caused by different fungal species, and a total of 83 infectious episodes were documented. Eighty-one cases were breakthrough infection during antifungal prophylaxis (FLCZ/ITCZ capsules in 28, and newer mold-active agents in 53). Invasive aspergillosis (IA) was the most common, accounting for 67.9% (proven in 11, probable in 46), followed by invasive candidiasis (IC), (19.3%; candidemia in 15, encephalitis in 1). Although the incidences of IA and IC were comparable between CBT and uBMT patients, relatively rare type of IFIs caused by Trichosporon (4 cases), Mucor (2 cases) and Rodotorula (1 case) exclusively occurred in CBT patients, except one case of Fusarium infection in a patient who relapsed after uBMT. Grade II-IV acute GVHD, extensive chronic GVHD and systemic corticosteroids at ≥0.5mg/kg/day were identified as significant risk factors of IFIs for both groups (HR 1.89, P=0.01, HR 4.16, P=0.006, and HR 1.83, P=0.02). However, the impact of all these were not apparent in CBT patients (HR 1.59, P=0.16, HR 2.18, P=0.29 and HR 1.48, P=0.22), in contrast with the powerful impact in uBMT patients (HR 2.51, P=0.049, HR 10.23, P=0.03 and HR 2.87, P=0.03). Although the cessation of antifungal prophylaxis significantly increased the risk of IFIs in uBMT patients (HR 5.95, P=0.01), it showed no impact in CBT patients (HR 0.87, P=0.89). IFIs were main causes of death in 21 patients, which significantly affected non-relapse mortality in both CBT and uBMT patients (HR 3.93 and HR 6.08, both P<0.001). Conclusion Unrelated cord blood did not increase an overall incidence of IFIs, because higher risk in the early post-transplant period was counterbalanced by the dramatically decreased risk due to lower frequencies of GVHD and its treatment in the later period. Particular attention might be required to the early-onset IFIs due to fungi other than Aspergillus or Candida species, in order to further decrease the risk of IFIs after CBT. Disclosures: No relevant conflicts of interest to declare.

scholarly journals Hematopoietic Stem Cell Transplantation at Home

Blood ◽  
2017 ◽  
Vol 130 (Suppl_1) ◽  
pp. 745-745
Author(s):  
Anthony D. Sung ◽  
Krista R. Nichols ◽  
Julia A. Messina ◽  
Kristi Romero ◽  
Tara Dalton ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Stem Cell ◽  
Gut Microbiome ◽  
Research Funding ◽  
Performance Status ◽  
Day Hospital ◽  
Hematopoietic Stem ◽  
House Calls ◽  
At Home

Abstract Introduction: Patients receiving autologous (auto) and allogeneic (allo) hematopoietic stem cell transplantation (HCT) typically undergo extensive inpatient hospitalizations, daily "day hospital" visits, or a combination of the two. This aspect of transplant poses a variety of challenges, ranging from nosocomial infections to hospital-induced delirium to lifestyle adjustments. By keeping patients at home, we may lower complications and improve quality of life, as well as potentially lower costs and resource utilization. Methods: We conducted a phase 1 study of home HCT to investigate the safety and feasibility of implementing this strategy in the United States. Patients interested in participating needed to live within a 30 minute drive of a transplant center and underwent a home inspection to assess suitability and safety (e.g. absence of black mold). Eligible patients would still receive pre-transplant conditioning at the hospital or day hospital, but they would be discharged home after receiving their stem cell infusion. The goal was to keep trial participants at home for the duration of transplant. In the morning, nurse practitioners or physician assistants made daily house calls to conduct assessments, examine patients, and draw blood for laboratory studies. These studies were run at the hospital, and in the afternoon a nurse returned to the patient's home to provide home blood transfusions, home intravenous fluids, electrolytes, or antibiotics, or other interventions. If an acute event occurred that could not be safely managed at home (e.g. first evaluation of febrile neutropenia), patients returned to the hospital or day hospital for further workup and care. Likewise, patients returned to the hospital or day hospital for certain routine procedures, such as intravenous methotrexate given as part of graft-versus-host disease prophylaxis or first blood transfusion to ensure there were no reactions. Transplant outcomes were monitored by the medical team throughout the patient's care and confirmed by a clinical research nurse or specialist by chart review. Stool samples, to assess changes in the gut microbiome, were collected at baseline and weekly for the first four weeks, at day 60, 100, and day 180. Samples will undergo 16s rRNA sequencing to identify the taxonomic groups of bacteria present in the gut. Results: Twenty-two patients received home HCT (Table 1). This included 6 allos and 16 autos. Ages ranged from 34-63 years for allos and 46-74 years for autos. In both groups, 2/3 or more of patients had a baseline Karnofsky Performance Status of 70 or 80. Patients in the allo group spent 72% of their days entirely at home, while patients in the auto group spent 52% of their days at home. The main reason for returning to the hospital or day hospital were febrile neutropenia (4 allos and 9 autos). Aside from CMV reactivation (3 of 6 allos, 50%), only 2 allos (33%) and 2 autos (13%) developed bloodstream infections. Three allos developed GVHD; interestingly, these were the three patients that spent the most time in the hospital/day hospital even prior to Day 30 (median 23 days vs. 11 days), even prior to development of GVHD. There was one case of treatment-related mortality (GVHD) in the entire cohort. Overall, patients and their caregivers endorsed the program, providing numerous expressions of appreciation and gratitude on exit interviews, and quality of life was well-preserved as assessed by FACT-BMT: median scores for autos stayed the same when comparing baseline (median 113) to day 30 (median 114) and increased on day 100 (median 124). Discussion: Our results suggest that home HCT is safe and feasible. Despite including mostly older adults with suboptimal performance status, patients did quite well at home. They were able to maintain their quality of life and had low rates of infectious complications. Though patients did have to return to the hospital or day hospital at various times during transplant, keeping patients out of the hospital for even half the duration of transplant could have tremendous cost savings that would offset the increased staffing and travel required for house calls. Studies of the gut microbiome are pending to test the hypothesis that home HCT will preserve the gut microbiome, thereby preventing GVHD. In addition, a randomized phase 2 study of home vs. standard transplant for allogeneic HCT is currently in progress. Disclosures Sung: Novartis: Research Funding; Merck: Research Funding; Cellective: Research Funding. Gasparetto: Janssen, BMS, Celgene: Consultancy; Janssen, BMS, Celgene: Other: Travel, accommodations, or other expenses paid or reimbursed; Celgene: Research Funding; Janssen, BMS, Celgene, Takeda: Honoraria. Rizzieri: Shire: Research Funding; Erytech: Research Funding.

scholarly journals Sequential Transplantation of Haploidentical Stem Cell and Unrelated Cord Blood With Using ATG/PTCY Increases Survival of Relapsed/Refractory Hematologic Malignancies

2021 ◽  
Vol 12 ◽  
Author(s):  
Hua Li ◽  
Xiaofan Li ◽  
Yiling Chen ◽  
Duihong Li ◽  
Xianling Chen ◽  
...  
Keyword(s):  
Cord Blood ◽  
Cord Blood Transplantation ◽  
Chronic Gvhd ◽  
Disease Free Survival ◽  
Hematologic Malignancies ◽  
Post Transplantation ◽  
Blood Transplantation ◽  
Gvhd Prophylaxis ◽  
Unrelated Donors ◽  
Unrelated Cord Blood

Allogeneic haploidentical HSCT (haplo-HSCT) and unrelated umbilical cord blood transplantation(UCBT)are used in patients lacking HLA-identical sibling or unrelated donors. With myeloablative condition and GVHD prophylaxis of using low-dose ATG and post-transplantation cyclophosphamide (PTCY), we conducted a prospective clinical trial. Of eligible 122 patients from February 2015 to December 2019 in the study, 113 patients were involved. Forty-eight patients were in the group of sequential haplo-cord transplantation (haplo-cord HSCT), and 65 patients were in the group of single UCBT. The primary endpoint of 2-year disease-free survival (DFS) was no statistical difference between groups (64.1 vs. 56.5%), p&gt;0.05. The analysis of subgroup patients with relapsed/refractory showed haplo-cord HSCT was associated with better OS (HR 0.348, 95% CI, 0.175–0.691; p=0.0025), DFS (HR 0.402, 95% CI, 0.208–0.779; p=0.0069), and GRFS (HR 0.235, 95% CI, 0.120–0.457, p&lt;0.0001) compared to the single cord group. The 2-year’s probability in OS, DFS, and GRFS was 64.9 vs. 31.6%, 64.5 vs. 31.6%, and 60.8 vs. 15.0% in the haplo-cord group and single cord group, respectively. III-IV acute GVHD 8.3 vs. 6.2%, chronic GVHD 25.8 vs. 13.7%, and extensive chronic GVHD 5.3 vs. 1.8% were shown in corresponding group, p&gt;0.05. The patients engrafted persistently with UCB showed better survival outcomes. Our sequential Haplo-cord HSCT with ATG/PTCY improved the survival of patients and might be an alternative transplantation approach for patients with relapsed/refractory hematologic malignancies.

Evaluation of Graft Versus Host Disease and Relapse Free Survival As Novel Endpoint in Allogeneic Hematopoietic Stem Cell Transplantation: A Retrospective Joint Naples-Paris Study

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 2285-2285
Author(s):  
Simona Pagliuca ◽  
Antonio M Risitano ◽  
Sylvie Chevret ◽  
Flore Sicre de Fontbrune ◽  
Alienor Xhaard ◽  
...  
Keyword(s):  
Stem Cell ◽  
Cumulative Incidence ◽  
Research Funding ◽  
Acute Gvhd ◽  
Chronic Gvhd ◽  
Relapse Free Survival ◽  
Hematopoietic Stem ◽  
Free Survival ◽  
Graft Versus Host

Abstract The cure of hematologic disorders by allogeneic hematopoietic stem cell transplantation (HSCT) is often associated with major complications resulting in poor outcome, including acute and chronic graft-versus-host disease (GVHD), relapse and death. Classical endpoints such as overall survival (OS), desease free survival (DFS) and non relapse-mortality (NRM) had become more and more unsuitable for transplant research because of their inability to a dynamic mesure of transplant-associated comorbidity. For this reason several composite endpoints taking into account also GVHD-associated comorbidity were proposed in the last years. GVHD free/relapse free survival (GRFS), proposed by Holtan et al (Blood 2015), includes grades 3-4 acute GVHD, systemic therapy requiring chronic GVHD, primary disease relapse , or death for any cause considered as events. This endpoint seems to completely characterize the survival without mortality or ongoing morbidity. With the intent to analyse the outcomes of our transplanted cohort, we retrospectively analysed GRFS of 959 consecutive patients receiving HSCT at Federico II University in Naples (n=119) and Saint-Louis Hospital (n=840) in Paris between 2007 and 2014, identifying prognostic factors associated with a better outcome and estimating the incidences of all components of this endpoint: rates of acute and chronic GVHD, disease relapse and death. Patient, disease and transplant characteristics are listed in table 1. Median duration of follow-up after HSCT was 22.1 months (IQR: 5.6-51 months). Cumulative incidence at day 100 of grade II-IV acute GVHD and grade III-IV were 42% and 16%, respectively. Cumulative incidence of chronic GVHD requiring systemic treatment at 1 and 5 years was 23% and 33%, respectively, diagnosed according to NIH criteria [14% of patients had score 1 (mild), 58% score 2 (moderate) and 27% score 3 (severe)cGVHD]. Cumulative incidence of relapse (considering all malignant and non-malignant diseases) was 26.7% (N=219) at 5 years. Overall survival for the whole population was 57% (95%CI, 53.3-60.8) at 5 years and Disease free survival (DFS) and non-relapse mortality (NRM) were respectively 50% (95%CI, 46.6-53.8) and 23% at 5 years. GRFS was 25% (95%CI, 21.8-28.5) at 5 years. Factors identified as influencing GRFS based on univariate analyses were age higher than 45 years (HR=1.64, 95%CI, 1.40-1.92), bone marrow (BM) as stem cell source (HR=0.40, 95%CI, 0.32-0.50); reduced intensity conditioning (RIC) (HR=0.63, 95%CI, 0.53-0.74); disease type [non-malignant disorders: HR=0.24, 95%CI, 0.17-0.33; myelodysplastic and myeloproliferative syndromes (MPN/CML/MDS): HR=1.34, 95%CI, 1.10-1.63; whereas other diagnosis did not influence GRFS] and than unrelated donor (matched: HR=1.71, 95%CI, 1.41-2.07;mismatch:HR=1.81, 95%CI, 1.48-2.23). Based on a multivariable Cox model, only diagnoses (non-malignancies, HR=0.27, 95%CI, 0.19-0.38 and MPN/CML/MDS, HR= 1.35, 95%CI, 1.11-1.65), and HLA unrelated graft (matched, HR=1.42, 95%CI, 1.17-1.73 and mismatched, HR=1.55, 95%CI, 1.26-1.92) remained associated with the outcome (Figure 1 and 2). GRFS could represent the ideal endpoint following HSCT. It differs significantly based upon type of disease and donor type, essentially. This composite indicator yields more information regarding complications of HSCT than the simpler measurement of OS or DFS. Its use willbetter compare these clinically important outcomes that accompany disparate HSCT techniques. All examined prognostic factors could enhance our ability to optimally judge the risk and the probability of true recovery after allogeneic HSCT. Our data support the use of this composite endpoint to describe HSCT outcome, and also pave the way for the investigation of novel endpoints, which may also track the dynamic changes of post-transplant events in the long-term. These retrospective data represent the background to investigate the impact of novel strategies of HSCT aiming to improve the outcome of HSCT, as detectable, by using more sensitive endpoints, tracking clinical events associated with detrimental long-term outcome. Figure 1 Figure 1. Figure 2 Figure 2. Disclosures Risitano: Alexion Pharmaceuticals: Other: lecture fees, Research Funding; Novartis: Research Funding; Alnylam: Research Funding; Rapharma: Research Funding. Peffault de Latour:Pfizer: Consultancy, Honoraria, Research Funding; Amgen: Research Funding; Novartis: Consultancy, Honoraria, Research Funding; Alexion: Consultancy, Honoraria, Research Funding.

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