scholarly journals Preemptive Administration of Ruxolitinib Rapidly Ameliorate Gvhd after Allo-Hematopoietic Stem Cell Transplantation: A Single-Center Retrospective Study

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 3293-3293
Author(s):  
Kangni Lin ◽  
Minmin Chen ◽  
Qian Li ◽  
Jinhua Ren ◽  
Haojie Zhu ◽  
...  
Keyword(s):  
Stem Cell ◽  
Retrospective Study ◽  
Hematopoietic Stem Cell Transplantation ◽  
Complete Remission ◽  
Stem Cell Transplantation ◽  
Overall Response Rate ◽  
Response Rate ◽  
Single Center ◽  
Hematopoietic Stem ◽  
Conditioning Regimens

Objective: Clinical benefit with ruxolitinib as a salvage therapeutic agent in steroid-refractory acute graft-versus-host disease (aGVHD) has been reported. The purpose of this study is to explore the efficacy and safety profile of preemptive administration of ruxolitinib for patients with GVHD after hematopoietic stem cell transplantation (HSCT), and to determine the optimal timing of start of ruxolitinib treatment. Methods: We conducted a single-center retrospective study to analyze the overall response rate at day 28 and the amelioration of GVHD after preemptive treatment of ruxolitinib in patients with GVHD post-HSCT. A total of 35 patients (26 males/ 9 females) with GVHD (30 aGVHD/ 5 cGVHD) treated at Fujian Medical University Union Hospital (Fuzhou, China) from July 2016 to April 2019 were enrolled. All patients received conditioning regimens including ATG, cyclosporine A, methylprednisolone, methotrexate mycophenolate mofetil w/o basiliximab for prevention of GVHD. Ruxolitinib was administered orally at 10 mg twice daily for patients ≥ 40 kg or 5 mg twice daily if <40 kg. The patients were divided into 3 groups according to the starting time of GVHD treatment with the preemptive ruxolitinib: shorter than 3 days (≤3 day,13, 37.1%), between days 3-7 (3-7 days,10, 28.6%) and more than 7 days(>7 days,12, 34.3%). Treatment responses of ruxolitinib and the safety are compared among groups. Results: These three groups were well matched demographically. There were no significant differences in conditioning regimens, donor types, CD34+ cells infusion, GVHD prophylaxis regimens and primary treatment among 3 groups. The median time to response of ≤3 days group, 3-7 days group and >7 days group was 20.5 (6-43) , 19.5 (2-107) and 20.5 (9-60) days, respectively. The ≤3 d group showed a best overall response rate (ORR) and complete remission rate (CRR) at day 28 after treatment than 3-7 d group and >7 d group (ORR: 92.3% vs 90.0% vs 83.3%, P=0.361; CRR: 46.2% vs 40.0% vs 25.0%, P=0.512 ). The cumulative complete remission rates among 3 groups were 58.5% (≤3 d group), 86.7% (3-7 d group) and 25% (>7 d group), respectively (P=0.096). The severity of GVHD were significantly ameliorated at day 28 in ≤3 d group than the other 2 groups ( 92.3% vs 70.0% vs 50.0%, P=0.064), and moreover, the clinical GVHD scores dropped significantly in ≤3 d group than >7 d group (-1.54 grade vs -0.75 grade, P=0.041) at day 28 after treatment. Incidence and severity of adverse reactions did not differ significantly between groups (P>0.05). Conclusion: When preemptive administration ruxolitinib is initiated rapidly, remarkable efficacy on amelioration of GVHD can be achieved without significant adverse effects. Larger randomized trials are needed to confirm efficacy. Disclosures No relevant conflicts of interest to declare.

scholarly journals Phase 2 trial of clofarabine in combination with etoposide and cyclophosphamide in pediatric patients with refractory or relapsed acute lymphoblastic leukemia

Blood ◽  
2011 ◽  
Vol 118 (23) ◽  
pp. 6043-6049 ◽  
Author(s):  
Nobuko Hijiya ◽  
Blythe Thomson ◽  
Michael S. Isakoff ◽  
Lewis B. Silverman ◽  
Peter G. Steinherz ◽  
...  
Keyword(s):  
Stem Cell ◽  
Acute Lymphoblastic Leukemia ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Pediatric Patients ◽  
Overall Response Rate ◽  
Response Rate ◽  
Lymphoblastic Leukemia ◽  
Phase 2 ◽  
Hematopoietic Stem

Abstract The outcomes in children with refractory/relapsed (R/R) acute lymphoblastic leukemia (ALL) are dismal. The efficacy and safety of intravenous clofarabine 40 mg/m2 per day, cyclophosphamide 440 mg/m2 per day, and etoposide 100 mg/m2 per day for 5 consecutive days in pediatric patients with R/R ALL was evaluated in this phase 2 study. The primary endpoint was overall response rate (complete remission [CR] plus CR without platelet recovery [CRp]). Among the 25 patients (median age, 14 years; pre-B cell ALL, 84%; ≥ 2 prior regimens: 84%; refractory to previous regimen: 60%), the overall response rate was 44% (7 CR, 4 CRp) with a 67.3-week median duration or remission censored at last follow-up. Most patients proceeded to alternative therapy, and 10 patients (40%) received hematopoietic stem cell transplantation. Six patients (24%) died because of treatment-related adverse events associated with infection, hepatotoxicity, and/or multiorgan failure. The study protocol was amended to exclude patients with prior hematopoietic stem cell transplantation after 4 of the first 8 patients developed severe hepatotoxicity suggestive of veno-occlusive disease. No additional cases of veno-occlusive disease occurred. The regimen offered encouraging response rates and sustained remission in R/R patients. Future investigation should include exploration of patient selection, dosing, and supportive care. This trial was registered at www.clinicaltrials.gov as #NCT00315705.

Allogeneic hematopoietic stem cell transplantation for primary myelofibrosis: A single-center experience.

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. e19522-e19522
Author(s):  
Taner Demirer ◽  
Guldane Cengiz Seval ◽  
Sinem Civriz Bozdag ◽  
Selami Kocak Toprak ◽  
Meltem Kurt Yuksel ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Acute Gvhd ◽  
Single Center ◽  
Hematopoietic Stem ◽  
Number Of Patients ◽  
Conditioning Regimens

e19522 Background: Allogeneic hematopoietic stem cell transplantation (Allo-HSCT) is a well-established treatment modality for patients with Myelofibrosis (MF) and still remains the only potentially curative therapy. The aim of this study was to determine overall outcomes of myelofibrosis patients treated with allo-HSCT in our center. Methods: This is a retrospective single-center analysis of 26 patients (female/male: 8/18) suffering from MF who underwent allogeneic HSCT at our center between 2002-2019. Forty-two percentages of the patients were at least MF intermediate-2 status based on DIPSS score (calculated at the time of transplantation). Results: All patients were in chronic phase of PMF at the time of the transplant. The median age at diagnosis was 48.2 years (range, 32-63 years). Median follow up of the patients were 15 months (range, 3-213 mo) and median time from diagnosis to HSCT was 25 months. Three of the patients had splenectomy before allo-HSCT. Splenomegaly was found in 20 patients and resolved completely in 6 patients. Myeloablative and RIC regimens were used for 8 (36.4%) and 18 (69.2%) of the 26 transplant procedures. All patients experienced engraftments of neutrophil and platelet except the five who died in aplasia period. Neutrophil and platelet engraftments occurred at median of 16 days (12-39 d) and 20 days (range, 11-78 d). There were no statistically significant differences in engraftment time between the types of conditioning regimens. Acute GVHD occurred with 11 of 26 transplanted allografts, with 6 of these being grade 3-4 acute GVHD. Chronic GVHD was seen 7 (26.9%) of the patients and 3 of these being extensive. A total of 26 out of the 11 patients died and 9 of the these 15 patients were alive and GVHD free at the end of our study. Relapse occurred in 3 patients after a median of 12 months and was treated with DLI in one case. The probability of 3-year progression free survival (PFS) and overall survival (OS) in all patients were 86.2%±9.1% and 58.7±11.4%, respectively. No statistical significance was observed for mean estimated 3-year OS in terms of the conditioning regimens. Conclusions: Despite the small number of patients, our results suggest that allo-HSCT may provide a curative treatment for patients with PMF.

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