scholarly journals Haploidentical Stem Cell Transplantation Can be Fully Conducted on an Outpatient Basis

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 4912-4912
Author(s):  
Iván Murrieta-Álvarez ◽  
Juan Carlos Olivares-Gazca ◽  
Yahveth Cantero-Fortiz ◽  
Andrés A. León-Peña ◽  
José Manuel Priesca-Marin ◽  
...  

Abstract Introduction Haploidentical hematopoietic stem cell transplantation (haplo-SCT) has become an acceptable approach for many patients; however, the suitability of this procedure and outcomes regarding efficacy and safety remain unclear. Here we present a single-center experience using a conditioning regimen based on cyclophosphamide and fludarabine under outpatient conditions. Methods: Retrospective study evaluating the performance of haplo-SCT in a single center. The conditioning regimen was fludarabine 50 mg on days -6 through -4, cyclophosphamide (Cy) 500 mg/m 2 on days -7 to -4 and tacrolimus 1000 mg and post-transplant Cy (50 mg/kg) on days +3 and +4 (Figure 1). Donor cell collection was accomplished through apheresis in all patients and fresh cells were administered on same day of collection. All the procedures were started on an outpatient basis. Overall survival was defined as mortality for any reason, starting to count from the day of transplantation to the date of death. The survival function was calculated according to the Kaplan-Meier estimation method. All patients signed a consent to participate after a thorough interview and the study protocol was approved by Clínica Ruiz IRB. Results: We grafted 20 patients, (14 adults and 6 children) with haploidentical cells and found that in 11 cases (55%), the full procedure could be completed totally as outpatients; the diagnosis of the grafted patients were: 10 acute lymphoblastic leukemia, 3 acute myelogenous leukemia, 3 paroxysmal nocturnal hemoglobinuria, and one each non-Hodgkin´s lymphoma, Blackfan-Diamond syndrome and multiple myeloma. Nine patients (3 children and 6 adults) were admitted to the hospital after completing the conditioning, 1 to 8 days after day 0: the causes for admission were neutropenic fever (5 cases), cytokine-release syndrome (3 cases), and intraabdominal abscess (1 case). Patients remained in the hospital for a median of 9 days. Four patients failed to engraft and recovered endogenous hematopoiesis and acute graft versus host-disease developed in 5 of 16 engrafted patients; two patients relapsed after the haplo-SCT. The transplant-related mortality was 35%, whereas the 2-year overall survival (OS) was 37.5% (Figure 2); the causes of the 7 deaths were: Four granulocytopenic sepsis, 2 graft-versus-host disease and one multiple organ failure. Conclusions Haplo-SCT procedures can be conducted safely and effectively on an outpatient basis; however, questions remain regarding the selection of patients and managing of complications, especially in outpatient conditions in which full and timely availability of specialized care could be the pivotal factor to improve short-term outcomes. Figure 1 Figure 1. Disclosures No relevant conflicts of interest to declare.

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 1327-1327 ◽  
Author(s):  
Nicolaus Kröger ◽  
Matthias Stelljes ◽  
Martin Bornhäuser ◽  
Wolfgang A. Bethge ◽  
Renate Arnold ◽  
...  

Abstract Abstract 1327 Total body irradiation (TBI) is an essential part of the conditioning regimen for acute lymphablastic leukaemia (ALL) patients who undergo allogeneic stem cell transplantation. Due to high toxicity and mortality induced by TBI, we investigate in a multicenter prospective phase II study the toxicity and efficacy of a non TBI-based regimen consisting of treosulfan (3 × 12 g/m2), etoposide (30 mg/kg), and cyclophosphamide (120 mg/kg) in patients with ALL who underwent allogeneic stem cell transplantation. Major inclusion criteria were complete remission, indication for allogeneic stem cell transplantation according GMALL, non eligibility for TBI or patient's wish to avoid TBI. Graft-versus host prophylaxis consisted of cyclosporine A and short course methotrexat and in case of unrelated donors also of anti-lymphocyte globulin (ATG). This preliminary analysis is based on 36 patients (female: n=17; male: n=19) with a median age of 42 years (range: 18–60y). Remission-status at transplantation was either 1. CR (n=27), 2.CR (n=7) or >2.CR (n=2). Donors were HLA-identical sibling (n=6), matched unrelated (n=21) or mismatched unrelated (n=9). 39% of the patients were Philadelphia chromosome positive. Primary graft failure was observed in 1 pts. The median time to achieve leukocyte (> 1×109/l)) and platelet (>20 × 109/l) engraftment was 22 (range, 11–47) and 24 days (range, 11–95), respectively. The toxicity was moderate including VOD (6%) and liver toxicity grade III (8%) and IV (3%). Acute graft versus host disease grade II-IV and grade III/IV was noted in 22% and 11%, respectively. Chronic graft versus host disease at 1 year was seen in 27%, which was extensive in 7% of the patients. After a median follow up of 12 months, the cumulative incidence of non-relapse mortality (NRM) at 1 year was only 9% (90% CI: 1–17%) and for relapse 37% (90% CI: 21–52%). The estimated 1-year disease free survival was 56% (90% CI:40-71%) and significantly better for patients transplanted in 1.CR vs 2. or higher CR (68% vs 15%, p=0.05). The estimated 1-year overall survival was 81% (90% CI: 69–92%). This preliminary results of a treosulfan, non TBI based conditioning regimen followed by allogeneic stem cell transplantation shows favourable toxicity profile with low non-relapse mortality. Longer follow up is needed to determine long-term freedom from disease. Disclosures: Kröger: MEDAC: Research Funding. Off Label Use: Treosulfan is not approved as conditioning regimen for stem cell transplantation.


Blood ◽  
2013 ◽  
Vol 121 (18) ◽  
pp. 3745-3758 ◽  
Author(s):  
Emily Blyth ◽  
Leighton Clancy ◽  
Renee Simms ◽  
Chun K. K. Ma ◽  
Jane Burgess ◽  
...  

Key Points Infusion of CMV-specific T cells early posttransplant does not increase acute or chronic graft-versus-host disease. CMV-specific T cells early posttransplant reduce the need for pharmacotherapy without increased rates of CMV-related organ damage.


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