scholarly journals Association of early donor chimerism status with survival outcomes in post allogeneic hematopoietic stem cell transplant patients of nonmalignant diseases

2021 ◽  
Author(s):  
Nadia Sial ◽  
Parvez Ahmed ◽  
Feroza Hamid Wattoo ◽  
Nadir Ali
Keyword(s):  
Bone Marrow ◽  
Overall Survival ◽  
Bone Marrow Failure ◽  
Disease Free Survival ◽  
Beta Thalassemia ◽  
Bone Marrow Failure Syndrome ◽  
Hematopoietic Stem ◽  
Free Survival ◽  
Donor Chimerism ◽  
Disease Free

Objective: To highlight the association of early donor chimerism status at 2nd month with various survival outcomes. Method: The retrospective study was conducted at the Armed Forces Bone Marrow Transplant Centre, Rawalpindi, Pakistan, and comprised patient  data from January 2011 to July 2016. Data related to participants who underwent human leukocyte antigen-matched transplants for bone marrow failure syndrome and beta thalassemia major. Short tandem repeat-based polymerase chain reaction was used to assess donor chimerism status. Overall survival, disease-free survival, relapse-free survival, and graft versus host disease-free survival rates were noted. Data was analysed using SPSS 23. Results: Of the 106, 64(60.4%) had bone marrow failure syndrome and 42(39.6%) had beta thalassemia major. The overall median follow-up was 13.53 months (range: 1.81-62.73 months). Early donor chimerism status was associated with overall survival (p=0.02) and disease-free survival (p=0.01). Mixed donor chimerism was less hazardous in terms of overall survival (p=0.04) and disease-free survival (p=0.02). Conclusion: Early mixed donor chimerism contributed to optimal survival in nonmalignant disease. Key Words: Hematopoietic stem cell transplantation, Nonmalignant diseases, Survival outcome, Conditioning regimen.

Induction Therapy with Dasatinib and Prednisone for Patients with Philadelphia Positive ALL

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 4907-4907
Author(s):  
Abhishek Chilkulwar ◽  
Salman Fazal ◽  
Jocelyn T. De Yao ◽  
Parik Padhi ◽  
Cyrus Khan ◽  
...  
Keyword(s):  
Overall Survival ◽  
Induction Therapy ◽  
Disease Free Survival ◽  
Hematopoietic Stem ◽  
Free Survival ◽  
Comorbidity Index ◽  
High Comorbidity ◽  
Disease Free ◽  
Matched Donor

Abstract Background: The addition of a Tyrosine Kinase Inhibitor (TKI) to induction chemotherapy has improved the outcome of patients with Philadelphia chromosome positive (Ph+) acute lymphoblastic leukemia (ALL). However, the treatment related mortality and morbidity of intensive treatment increases with age. The use of a TKI alone for induction is less toxic and yields CR rates comparable to combined therapy. Eligibility for post remission hematopoietic stem cell transplantation is less likely to be compromised with TKI induction. We present a retrospective review of patients with Ph+ ALL treated at our institution with dasatinib and prednisone induction who subsequently underwent allogeneic hematopoietic stem cell transplant (allo-HSCT) as post remission therapy. Methods: We retrospectively identified 15 patients with Ph+ ALL treated at our institution between February 2012 and June 2015. Patients received induction therapy with dasatinib at 100 mg or 140mg daily till complete hematological response. Prednisone 60 mg/m2/day (capped at 120 mg daily) was administered until day 24 and then tapered and stopped at day 32. Intrathecal chemotherapy with MTX and Ara-C were administered twice during the induction period. Dasatinib dose reduction/discontinuation was permitted for non-hematological toxicity. Patients who achieved remission proceeded to allo-HSCT if a suitable HLA-matched donor was available. Patients who did not have a suitable HLA matched donor received TKI + POMP maintenance. We calculated CHR, CCyR, disease-free survival (DFS) and overall survival (OS). Results: The median age of patients treated with dasatinib plus prednisone was 62 years (range: 19-73). Baseline patient and disease characteristics are summarized in Table 1. Median WBC count was 22.5 x 109/L. Fourteen of 15 patients treated with dasatinib achieved a CHR (93.3%), 1 patient did not undergo a bone marrow biopsy but had normal blood counts. Median time to CHR was 42 days (range: 22-69). CCYR was obtained in 11 patients (73%) and MMR was achieved in 5 patients (33%). No patient died during induction therapy. The 14 patients who were in CHR after induction, underwent allo-HSCT (n=7), are being evaluated for allo-HSCT (n=3), were unable to undergo allo-HSCT due to a high comorbidity index and/or lack of a suitable donor (n=3) or were lost to follow-up (n=1). Of the 3 patients who were unable to undergo allo-HSCT, 2 patients continue on dasatinib maintenance and 1 patient takes ponatinib. Of 8 patients not yet transplanted 3 relapsed, while only 1 relapse was seen in 7 patients who underwent allo-HSCT. Median DFS was 315 days (range: 57-1061) and median OS was 354 days (range: 107-1082) corresponding Kaplan Meier curves for OS and DFS are shown below. Conclusions: In our adult Ph+ ALL patients induction therapy with dasatinib and prednisone was effective and well tolerated. Patients achieving CHR were able to undergo allo-HSCT with curative intent. This strategy retrospectively appears equal or better than results with induction chemotherapy of conventional variety. Table 1. Patient characteristics Male sex, n (%) 5 (33.3) Age <20, n (%) 1 (6.7) 20-49, n (%) 1 (6.7) ³50, n (%) 13 (86.6) Median (range) 62 (19-73) Median follow-up in months (range) 11.7 (4.1-40) Presenting WBC x 10 9/L < 30, n (%) 8 (53.3) ³ 30, n (%) 7 (46.7) Median (range) 22 (2.8-358.4) Bcr-Abl type p190, n (%) 12 (80) p210, n (%) 2 (13.3) P190 and p210, n (%) 1 (6.7) Bcr-Abl level (1 unknown)* Mean (range) 35.1 (1.8-194.4) Median time to CHR in days (1 unknown), (range) 41.5 (22-69) Induction dose of dasatinib 70mg BID, n (%) 1 (6.7) 100mg daily, n (%) 8 (53.3) 140mg daily, n (%) 6 (40) CCyR after induction achieved, n (%) 11 (73.3) MMR achieved after induction, n (%) 5 (33.3) Dasatinib Dosing after Induction None, n(%) 1 (6.7) 70mg BID, n(%) 1 (6.7) 100mg/day, n(%) 12 (80) 140mg/day, n(%) 1 (6.7) POMP + TKI post induction, n(%) 4 (26.7) Post remission therapy (3 being evaluated for transplant, 1 never achieved CHR, 1 lost to ff-up)+ Transplant, n (%) 7 (46.7) Ponatinib, n (%) 1 (6.7) Dasatinib, n (%) 1 (6.7) HyperCVAD±, n (%) 1 (6.7) TKI maintenance after transplant, n (% of transplanted) 3 (42.9) M351T mutation, n (%) 1 (6.7) F317L mutation, n (%) 1 (6.7) Bcr-Abl detection by PCR with unit in ratio (international scale), +poor performance status or high comorbidity index is the reason for no transplant, ±hyperCVAD initiated but not tolerated. Figure 1. Overall Survival. Figure 1. Overall Survival. Figure 2. Disease Free Survival Figure 2. Disease Free Survival Disclosures Fazal: Novartis: Honoraria, Speakers Bureau; Bristol Myers Squibb: Consultancy, Honoraria, Speakers Bureau; Ariad: Consultancy, Honoraria, Speakers Bureau; Pfizer: Honoraria, Speakers Bureau. Off Label Use: Dasatinib use for newly diagnosed Ph+ ALL.

scholarly journals Wilms’ tumor gene 1 is an independent prognostic factor for pediatric acute myeloid leukemia following allogeneic hematopoietic stem cell transplantation

BMC Cancer ◽  
2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Dao-Xing Deng ◽  
Juan-Juan Wen ◽  
Yi-Fei Cheng ◽  
Xiao-Hui Zhang ◽  
Lan-Ping Xu ◽  
...  
Keyword(s):  
Overall Survival ◽  
Myeloid Leukemia ◽  
Wilms Tumor ◽  
Disease Free Survival ◽  
Hematopoietic Stem ◽  
Free Survival ◽  
Pediatric Aml ◽  
Disease Free ◽  
Tumor Gene ◽  
Acute Myeloid

Abstract Background Sequential monitoring of Wilms’ tumor gene 1 (WT1) after allogeneic hematopoietic stem cell transplantation (allo-HSCT) could predict relapse in adult acute myeloid leukemia (AML). However, the prognostic role of WT1 in pediatric AML after allo-HSCT is unclear. Thus, we determined to see whether sequential monitoring of WT1 after allo-HSCT could predict relapse in AML children. Methods Pediatric AML patients receiving allo-HSCT from January 21, 2012 to December 20, 2018 at the Peking University Institute of Hematology were included in this study. WT1 expression level was determined by TaqMan-based reverse transcription-polymerase chain reaction. WT1 sequential monitoring was performed 1, 2, 3, 4.5, 6, 9, and 12 months post-transplantation and at 6-month intervals thereafter. The primary end point was relapse. The secondary end points included disease-free survival (DFS), overall survival (OS), and non-relapse mortality (NRM). Kaplan–Meier analysis was used for DFS and OS estimates, while competing risk analysis was used for estimating relapse and NRM. Results Of the 151 consecutive patients included, the median age was 10 years (range, 1–17). The optimal cutoff value of WT1 within 1 year after allo-HSCT to predict relapse was 0.8% (80 WT1 copies/104 ABL copies), with a sensitivity of 60% and specificity of 79%. Compared with WT1 expression < 0.8%, WT1 expression ≥0.8% indicated significantly higher 5-year cumulative incidence of relapse (CIR, 35.1% vs. 11.3%; P = 0.001), lower 5-year disease-free survival (DFS, 60.4% vs. 80.8%; P = 0.009), and lower 5-year overall survival (OS, 64.9% vs. 81.6%; P = 0.038) rates. Multivariate analyses showed that WT1 was an independent risk factor for relapse (HR 2.89; 95% confidence interval (CI), 1.25–6.71; P = 0.014). Both the CIR (5-year CIR: 8.3% vs. 11.3%; P = 0.513) and DFS (5-year DFS: 91.7% vs. 80.8%; P = 0.208) were comparable between patients achieving minimal residual disease (MRD) negativity after preemptive interferon-α (IFN-α) treatment and those without MRD after allo-HSCT, which were better than those of MRD-positive patients without preemptive therapies. Conclusions Sequential monitoring of WT1 could predict relapse in pediatric AML after allo-HSCT. WT1-directed immunotherapy may have the potential to prevent relapse and improve survival.

Impact of Chronic Graft Versus Host Disease in the Overall Survival and Disease Free Survival of Patients with Hematologic Disorders Submitted to Allogeneic Bone Marrow Transplantation

Blood ◽  
2008 ◽  
Vol 112 (11) ◽  
pp. 4305-4305
Author(s):  
Emmerson de Sousa Eulalio ◽  
Elizabeth Schulz ◽  
José Salvador Rodrigues de Oliveira
Keyword(s):  
Bone Marrow ◽  
Overall Survival ◽  
Graft Versus Host Disease ◽  
De Novo ◽  
Disease Free Survival ◽  
Extensive Form ◽  
Free Survival ◽  
Graft Versus Host ◽  
The Mean ◽  
Disease Free

Abstract The management of the chronic graft versus host disease (C-GVHD) in the allogeneic hematopoietic stem cell transplantation (allo-HSCT) is still challenging and does not follow a consensus. This disease can manifest as auto-immune phenomena, often affecting multiple organs, and it is considered as a determinant factor for higher post transplant mortality and detrimental quality of life, although conversely associated with a strong graft versus tumor effect which determines a beneficial biological response in patients with haematological pathologies. The aim of this study was to determine the impact of the C-GVHD in the overall survival (OS) and disease free survival (DFS) in patients with hematologic pathologies who underwent allo-HSCT with HLA identical donors in the BMT Hospitals Sao Paulo and Santa Marcelina, in Sao Paulo, Brazil. We performed a retrospective study of historical cohort including 233 allo-HSC transplants, 151 patients aged 9 to 63 years old (median 31) with survival superior to 100 days, from August 1993 to December 2004. The classification of DECH-C followed the criteria of Seattle (Shulman et al. 1980) revised by Lee et al (2003). Our series was composed of 97 patients with chronic myeloid leukemia (CML), 54 with acute leukemias (AL), 42 with marrow failure (MF) and 40 with lymphoproliferative diseases (LPD). Forty one percent of the patients exhibited the advanced disease at HSCT time and the main source of HSC was bone marrow (n=174, 74.7%). The diagnosis of C-GVHD was performed in 166 procedures (71.2%). Seventy one patients (42.8%) were classified as C-GVHD quiescent, 69 (41.6%) as de novo, and 25 (15.1%) as progressive. The extensive form occurred in 95 cases (57.2%) and the limited form in 70 cases (42.2%). The mean OS for our cohort was 34.5 months (m) and the mean DFS was 29.5 m. The mean OS of patients with C-GVHD was 42 m, significantly higher than than 33.8m OS of patients without C-GVHD (p=0.05). Similarly, the DFS was 38.6m for C-GVHD patients, against 22.6m of DFS for patients without C-GVHD (p=0.0001). The OS of those patients with C-GVHD de novo (47m) and quiescent (44.8m) were superior than the OS of patients without C-GVHD (33.8%) or with the progressive form of C-GVHD (20.8m), p=0.002. The same effect was observed for the DFS in the de novo form (42.6m) and quiescent form (41.4m) of C-GVHD, with a DFS twice superior than the progressive form (19.8m) and than the C-GVHD absent (22.6m), p=0.001. When compared with cases without C-GVHD, the clinical severity of the C-GVHD (limited or extensive) showed a strong correlation with a higher DFS. Patients with limited C-GVHD had a DFS of 38.3m and the extensive form a DFS of 38.9m, significantly superior than the DFS of patients without C-GVHD with a DFS of 22.6m (p=0.002). Hence, this study shows the existence of a correlation between the presence of C-GVHD and higher OS and DFS in patients with haematological disorders submitted to allo-HCT. The diagnosis of progressive C-GVHD was associated to a deleterious effect over the outcome of allo-HCT, therefore inactivating the benefits of the graft versus tumour effect observed in the clinical presentations de novo and quiescent of C-GVHD.

Intensive Treatment Versus Azacitidine for Acute Myeloid Leukemia in Elderly: Retrospective Evaluation

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 4926-4926
Author(s):  
Zahit Bolaman
Keyword(s):  
Bone Marrow ◽  
Overall Survival ◽  
Complete Remission ◽  
Cytosine Arabinoside ◽  
Disease Free Survival ◽  
Intensive Treatment ◽  
Free Survival ◽  
Elderly Aml ◽  
Disease Free ◽  
Acute Myeloid

Backround: Most of patients with acute myeloid leukaemia (AML) are elderly and older age is an negative prognostic factor for AML. There is no consensus about treatment of elderly patients with AML. In this study, we evaluated intensive treatment with cytosine arabinoside and idarubicine versus azacitidine treatment. Primary objectives were evaluation of disease free (DFS) and overall survival (OS), the rate of complete remission and the remission duration. Methods: 45 patients with AML were evaluated retrospectively. We examined peripheral blood smear, bone marrow aspirate and trephine, immunophenotyping and cytogenetic analysis, biochemical evaluation of renal and liver function and coagulation profile in pre-treatment period. Intensive treatment consisted of cytosine arabinoside 200 mg/m2/day for 7 days and idarubicin 12 mg/m2/ for 3 days. Bone marrow examination was done between 14-28 days for intensive treatment and 2-6 cyclus for azacitidine treatment. Consolidation treatment (1-3) was done after intensive treatment if the patients were remission. All patients were ≤ECOG 2. Growth factors were not routinely administered before, during or after chemotherapy. Statistical analyses, differences in baseline characteristics between groups were analyzed using the Student's t-test for continuous variables and differences in OS between groups were then analyzed using the likelihood ratio or log-rank tests. Table 1. The main properties of patients with elderly AML Treatment arm Mean age Sex M/F Leucocyte mean Hb gr/dl FAB (n) MDS history(n) LDH Complete remission Total 73±9 26/19 34.029 ±6870 8.53±1,2 M0:15, M1:6, M2:15,M4:4,M5:2 Yes:21 No:24 276±151 15 Intensive 72±7 18/10 44.176±12390 6.91±2 M0:6, M1:4, M2:12, M4:3, M5:1 yes: 14 299±101 13 Azacitidine 74±8 8/9 31.289±4013 8,95±1.6 M0:9, M1:2, M2:3, M4:1, M5:1 yes:7 256±72 2 Results: Total patient number were 45 and they were ≥ 65 years (yrs). Male/female ratio was 29/16. Two patients rejected leukemia treatment. One patient were not evaluated for treatment response. Intensive arm consisted of 26 patients and azacitidine arm 16 patients. Disease free survival and overall survival for intensive arm and azacitidine arm were 12,1±9,4, 16,7±22,4, respectively. The most importance factors were leucocyte counts, hb level. Picture 1-2: Disease free survival and overall survival for intensive arm and azacitidine arm Conclusions: These results showed that intensive treatment is more effective than azacitidine in elderly AML patients with ECOC≤2. There is need for large studies which include higher number of patients with AML in order to evaluate efficiency of intensive treatment. Figure 1. Figure 1. Figure 2. Figure 2. Disclosures No relevant conflicts of interest to declare.

Response to Paclitaxel, Topotecan, and Topotecan-Cyclophosphamide in Children With Untreated Disseminated Neuroblastoma Treated in an Upfront Phase II Investigational Window: A Pediatric Oncology Group Study

2004 ◽  
Vol 22 (20) ◽  
pp. 4119-4126 ◽  
Author(s):  
Cynthia S. Kretschmar ◽  
Morris Kletzel ◽  
Kevin Murray ◽  
Paul Thorner ◽  
Vijay Joshi ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Overall Survival ◽  
Bone Marrow Transplantation ◽  
Pediatric Oncology ◽  
Disease Free Survival ◽  
Oncology Group ◽  
Free Survival ◽  
Pediatric Oncology Group ◽  
Grade 3 ◽  
Disease Free

Purpose Most children older than 1 year of age with metastatic neuroblastoma (NB) die despite intensive chemotherapy and bone marrow transplantation. The Pediatric Oncology Group conducted a study of paclitaxel, topotecan, and topotecan-cyclophosphamide (topo-cyclo) in newly diagnosed children with stage IV NB. Patients and Methods There were 102 patients enrolled between September 1993 and October 1995; two of them were later shown to be ineligible. Of the remaining 100 patients, the first cohort of 33 patients received paclitaxel 350 mg/m2 intravenously (IV) over 24 hours every 14 to 21 days; the next 33 patients received topotecan 2 mg/m2/d for 5 days IV every 21 days; a third cohort of 34 patients were treated with IV cyclophosphamide 250 mg/m2 followed by topotecan 0.75 mg/m2 each day for 5 days every 21 days. Patients were re-evaluated after two courses and then treated with intensive induction therapy and bone marrow transplantation. Results Objective responses (complete response + partial response + mixed response) were documented in 67% of children who received topotecan, 76% after topo-cyclo, and 25% after paclitaxel. Four patients had grade 3 to 4 allergic reactions to paclitaxel; most patients developed grade 3 to 4 marrow suppression after topotecan or topo-cyclo. Neither disease-free survival nor overall survival differed significantly between children who received a phase II agent and those who did not. The 6-year disease-free survival and overall survival rates for all 100 children were 18% ± 5% and 26% ± 5%, respectively. Conclusion Topotecan and topo-cyclo are active in children with NB, are well tolerated, and should be evaluated further in combination regimens.

Predictable Prognostic Factor of CD56 Expression in Acute Myeloid Leukemia with t(8:21) Including Allogeneic Hematopoietic Stem Cell Transplantation.

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 3288-3288
Author(s):  
Deok-Hwan Yang ◽  
Yeung-Chul Mun ◽  
Ho-Jin Shin ◽  
Yeo-Kyeoung Kim ◽  
Je-Jung Lee ◽  
...  
Keyword(s):  
Overall Survival ◽  
Acute Myeloid Leukemia ◽  
Stem Cell ◽  
Myeloid Leukemia ◽  
Disease Free Survival ◽  
Hematopoietic Stem ◽  
Free Survival ◽  
Cd56 Expression ◽  
Disease Free ◽  
Acute Myeloid

Abstract CD56 expression in acute myeloid leukemia (AML) has been associated with extramedullary leukemia and multi-drug resistance, but the clinical and prognostic significances are not yet clearly defined. Recently, some investigators reported that AML patients with t(8;21) showed more frequent CD56 expression rate and the expression of CD56 antigen adversely affected disease-free survival (DFS). It could explain a diverse clinical outcome in AML patients with favorable cytogenetics. This study investigated CD56 expression in 37 adult de novo AML patients with t(8:21) between November 1996 and June 2005 at three institutions. Immunophenotyping was performed with flow cytometry (Coulter EPICS XL) and considered positive if at least 20% of blasts expressed. CD56 was expressed in 25 cases (67.6%). There was no statistically significant differences in age, sex, leukocyte count, the percentage of bone marrow blasts or the presence of additional cytogenetic abnormalities between the CD56+ and the CD56- group. The complete remission (CR) rate to standard dose cytarabine or N4-behenoyl-1-D-arabinofuranosylcytosine (BH-AC) and idarubicin was similar in both groups (91.7% v 88.7%; P=0.73), but the relapse rate to high-dose cytarabine or allogeneic hematopoietic stem cell transplantation (HST) was quite different (60% v 25%; P=0.08). Allogeneic HST was performed from siblings in 15 patients (40.5%) who achieved CR, 8 patients (32.0%) in CD56+ and 7 patients (58.3%) in CD56- group (P=0.16). The median durations of DFS were significantly shorter in CD56+ (median, 12.2 months) than in the CD56- group (median, not reached) (P =0.02). Also, the median durations of survival showed the same results in the CD56+ group (median, 14.9 months) compared with the CD56- group (median, not reached) (P=0.01). Within fifteen transplanted patients, the median durations of DFS in eight CD56+ patients was significantly shorter than seven CD56- patients (median, 24.4 months v not reached; P=0.02)(Fig.1 and 2).We concluded that CD56 expression was associated with reduced DFS and survival for AML patients with t(8:21) including transplanted patients. Although further larger studies are needed, we suggested that CD56 expression at diagnosis is a predictable prognostic factor in AML with t(8:21). Fig. 1 Disease-free survival (DFS) and Overall survival (OS) for patients with t(8:21) with CD56+ (n−25) and CD56− (n−12) group. Fig. 1. Disease-free survival (DFS) and Overall survival (OS) for patients with t(8:21) with CD56+ (n−25) and CD56− (n−12) group. Fig. 2 Within transplanted patients, decreases from survival (DFS) for patients with t(8:21) with and without CD56 expression. Fig. 2. Within transplanted patients, decreases from survival (DFS) for patients with t(8:21) with and without CD56 expression.

Efficacy of Reduced Induction Chemotherapy and Early Transplantation in ALL Patients

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 4487-4487
Author(s):  
Ardeshir Ghavamzadeh ◽  
Amir Hooshang Pourkhani ◽  
Kamran Alimoghaddam ◽  
Mohammadreza Ostadali Dehaghi ◽  
Amir Ali Hamidieh ◽  
...  
Keyword(s):  
Overall Survival ◽  
Causes Of Death ◽  
Disease Free Survival ◽  
P Value ◽  
Hematopoietic Stem ◽  
Free Survival ◽  
One Year ◽  
And Control ◽  
Disease Free

Abstract Abstract 4487 Eighty percent of acute lymphoblastic leukemia (ALL) patients achieve a complete remission (CR) but only 30–40% are long-term survivors. Hematopoietic stem cell transplantation (HSCT) is only curable treatment in ALL patients. However, the efficacy of induction, consolidation chemotherapy and early hematopoietic stem cell transplantation remain unclear. Therefore, at our center, patients with newly diagnosed ALL, are randomly divided into 2 arms from 2008 to 2011. Patients in the study arm received reduced induction chemotherapy (Vincristine 1 mg/m2 every week for 4 weeks plus Dexamethasone 24mg/d for 28 days) and undergone early HSCT after disease stabilized within 15–30 days without intention of achieving complete remission (CR). The control arm received conventional chemotherapy (routine induction and then consolidation) followed by HSCT. Both arms received Busulfan (4mg/kg for 4 days) plus Cyclophosphamide (60mg/kg for 2 days) as a conditioning regimen. The GVHD prophylaxis consisted of Methotrexate plus Cyclosporine. Here, we compare the efficacy of these two kinds of treatment. A total of 89 patients enrolled in the study. Seventeen patients allocated to the study arm and 72 others allocated to the control arm. The median age was 22 years (range: 16–33) in the study arm and 22 years (range: 3–49) in the control arm. All patients underwent Allogeneic HSCT with peripheral blood source. The median waiting time from diagnosis to HSCT was 708 days (range: 45–219) in the control arm. In the study arm, 13 patients (76%) were in CR1.The median follow-up time was 15.5 months (range: 1–30) in the study arm and 10.2 months (range: 1–34) in the control arm. Relapse occurred in 2 (11.7%) and 6 (8.3%) patients of the study and the control arms, respectively. Five patients (29.4%) of the study arm and 10 patients (13.5%) of the control arm were died. The causes of death were GVHD and sepsis in 4 (80%) patients and relapse in 1 (20%) patient in the study arm. The causes of death were GVHD in 6 (60%) patients and relapse in 4 (40%) patients in the control arm. One-year overall survival was 75% and 83.7% in study and control arms, respectively (Fig 1, P-value: 0.212). One-year disease-free survival was 75% and 81.1% in study and control group, respectively (Fig 2, P-value: 0.273).Figure 1.Overall Survival of ALL Patients in Study arm vs. Control armFigure 1. Overall Survival of ALL Patients in Study arm vs. Control armFigure 2.Disease-free Survival of ALL Patients in Study arm vs. Control armFigure 2. Disease-free Survival of ALL Patients in Study arm vs. Control arm Reduced induction followed by early transplantation without consolidation reveals no significant statistical different outcome compared with routine treatment. This result might be due to small size of patients and short time of follow-up. A study with more cases and long time follow-up is recommended. Disclosures: No relevant conflicts of interest to declare.

Lack Of Advantage Of Peripheral Blood Hematopoietic Stem Cell Transplantation Over Bone Marrow From HLA Matched Related Donors After Myeloablative Conditioning For Hematologic Malignancies

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 5470-5470
Author(s):  
Paulo Vidal Campregher ◽  
Nelson Hamerschlak ◽  
Vergilio Antonio Renzi Colturato ◽  
Marcos Mauad ◽  
Mair Pedro de Souza ◽  
...  
Keyword(s):  
Bone Marrow ◽  
High Risk ◽  
Peripheral Blood ◽  
Cumulative Incidence ◽  
Disease Free Survival ◽  
Hematopoietic Stem ◽  
Free Survival ◽  
High Risk Disease ◽  
Standard Risk ◽  
Disease Free

Abstract Although bone marrow (BM) and peripheral blood mobilized hematopoietic stem cells (PBSC) are widely used as graft sources in patients undergoing hematopoietic stem cell transplantation (HSCT), the graft of choice for each subset of patients remains to be determined. Several studies have, prospectively and retrospectively, addressed this question with inconsistent results. While the increased incidence of chronic graft versus host disease (cGVHD) in PBSC recipients has been unanimously found, data regarding disease free survival (DFS), overall survival (OS) and acute graft versus host disease (aGVHD) incidence have been controversial, mainly for patients with high risk disease. We retrospectively compared the clinical outcomes of 334 patients with acute leukemia and chronic myeloid malignancies receiving related BMT or PBSCT after myeloablative conditioning regimen, treated at seven transplantation centers in Brazil from 2008 to 2009. Median OS was 2.85 and 2.39 years (HR 1.19; 95% CI, 0.84 to 1.68, p=0.34), and DFS was 2.48 and 2.18 years for BM and PBSC recipients respectively (HR 1.07; 95% CI, 0.77 to 1.48, p=0.70). For patients with high risk disease, median OS was 2.1 and 1.72 years (HR 1.18; 95% CI, 0.73 to 1.91, p=0.50) and DFS was 0.46 and 0.58 years (HR 1.04; 95% CI, 0.66 to 1.64, p=0.86) for BMT and PBSCT respectively. Additionally, in agreement with previous reports, the cumulative incidence of chronic GVHD at three years was 53.7% and 79.8% (HR 1.93; 95% CI 1.38 to 2.69, p< 0.001) for BM and PBSC respectively (Figure 1). On the other hand, aGVHD incidence / severity, cumulative incidence of relapse and non relapse mortality (NRM) were not different between BM or PBSC recipients. The same being true when only patients with high risk disease were evaluated. Figure 1 Major outcomes after transplant according to bone marrow (dark line) and peripheral blood (gray line) as the graft sources. Overall survival for the whole cohort (panel A), high risk (panel B), cumulative incidences of: and standard risk (panel C) patients; disease free survival for the whole cohort (panel D), high risk (panel E) and standard risk (panel F) patients, and relapse (panel G), grades III-IV aGVHD (panel H) and cGVHD (panel I) for the whole cohort. The only outcome that was statistically different was the cumulative incidence of cGVHD – (item I) Figure 1. Major outcomes after transplant according to bone marrow (dark line) and peripheral blood (gray line) as the graft sources. Overall survival for the whole cohort (panel A), high risk (panel B), cumulative incidences of: and standard risk (panel C) patients; disease free survival for the whole cohort (panel D), high risk (panel E) and standard risk (panel F) patients, and relapse (panel G), grades III-IV aGVHD (panel H) and cGVHD (panel I) for the whole cohort. The only outcome that was statistically different was the cumulative incidence of cGVHD – (item I) In conclusion, in our cohort, there was no OS or DFS benefit for patients receiving PBSC when compared to BM recipients, even when only high risk patients were analyzed. Disclosures: No relevant conflicts of interest to declare.

Comparison of clinical outcomes between enucleation and regular pancreatectomy in patients with non-functional pancreatic neuroendocrine tumors: a retrospective multicenter and propensity score-matched study

2021 ◽  
Author(s):  
Zhen Yang ◽  
Hengjun Gao ◽  
Jun Lu ◽  
Zheyu Niu ◽  
Huaqiang Zhu ◽  
...  
Keyword(s):  
Overall Survival ◽  
Postoperative Complications ◽  
Propensity Score ◽  
Propensity Score Matching ◽  
Neuroendocrine Tumors ◽  
Disease Free Survival ◽  
Pancreatic Neuroendocrine Tumors ◽  
Free Survival ◽  
Estimated Blood Loss ◽  
Disease Free

Abstract Objective There are limited data from retrospective studies on whether therapeutic outcomes after regular pancreatectomy are superior to those after enucleation in patients with small, peripheral and well-differentiated non-functional pancreatic neuroendocrine tumors. This study aimed to compare the short- and long-term outcomes of regular pancreatectomy and enucleation in patients with non-functional pancreatic neuroendocrine tumors. Methods Between January 2007 and July 2020, 227 patients with non-functional pancreatic neuroendocrine tumors who underwent either enucleation (n = 89) or regular pancreatectomy (n = 138) were included. Perioperative complications, disease-free survival, and overall survival probabilities were compared. Propensity score matching was performed to balance the baseline differences between the two groups. Results The median follow-up period was 60.76 months in the enucleation group and 43.29 months in the regular pancreatectomy group. In total, 34 paired patients were identified after propensity score matching. The average operative duration in the enucleation group was significantly shorter than that in the regular pancreatectomy group (147.94 ± 42.39 min versus 217.94 ± 74.60 min, P &lt; 0.001), and the estimated blood loss was also significantly lesser (P &lt; 0.001). The matched patients who underwent enucleation displayed a similar overall incidence of postoperative complications (P = 0.765), and a comparable length of hospital stay (11.12 ± 3.90 days versus 9.94 ± 2.62 days, P = 0.084) compared with those who underwent regular pancreatectomy. There were no statistically significant differences between the two groups in disease-free survival and overall survival after propensity score matching. Conclusion Enucleation in patients with non-functional pancreatic neuroendocrine tumors was associated with shorter operative time, lesser intraoperative bleeding, similar overall morbidity of postoperative complications, and comparable 5-year disease-free survival and overall survival when compared with regular pancreatectomy.

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