ESHAP+/− Rituximab as Salvage Therapy for Relapsed Lymphoma Prior to Stem Cell Transplant: Single Institution Experience.

Blood ◽  
2004 ◽  
Vol 104 (11) ◽  
pp. 4601-4601 ◽  
Author(s):  
Sagun Shrestha ◽  
Christina Johnson ◽  
Sanjeev Jain ◽  
Dilip V. Patel ◽  
Bhoomi Mehrotra
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Salvage Therapy ◽  
Progressive Disease ◽  
Stem Cell Transplant ◽  
Cell Transplant ◽  
Non Hodgkins Lymphoma ◽  
Disease Free

Abstract OBJECTIVE: High dose chemotherapy with stem cell transplantation (ABMT) has become the standard treatment for relapsed aggressive non-Hodgkins and Hodgkins Lymphomas. However, there is no one salvage therapy accepted as a standard regimen prior to ABMT. ESHAP, as a combination chemotherapy regimen consisting of Etoposide, Methylprednisolone, Cytosine arabinoside, and infusional Cisplatin, has been shown to be an active regimen for the treatment of refractory Non-Hodgkins Lymphoma in previously published trials. The objective of this study was to determine the efficacy of ESHAP with and without Rituximab as a cytoreductive regimen in relapsed lymphomas prior to stem cell transplant. METHODS: This retrospective study was approved by the Institutional Review Board. Patients with relapsed Hodgkins and Non-Hodgkins lymphoma who were treated at our institution between March 1997 and July 2004, with the intent of proceeding to ABMT after obtaining a complete remission and were treated with ESHAP containing salvage regimen, were included in this study. Data collection was done by review of all pertinent medical records. RESULTS: Twenty four patients with relapsed lymphomas were identified that were treated with ESHAP +/− Rituximab during this period, with the intent to proceed with ABMT after achieving a maximal response. Histology included: large cell lymphoma (n=15), mantle cell lymphoma (n=3), and Hodgkin’s disease (n=6). Median age was 54 years and male-to-female ratio was 1.4:1. Staging of disease at the time of initial diagnosis was done by PET scan, CT scans, and bone marrow examination. Staging at time of salvage treatment for relapsed disease: stage I (n=1) stage II (n=2)), stage III (n=12), stage IV (n=9). Prior treatment consisted of CHOP, ABVD, TVC, MOPP/ABVD, CNOP, CVP, and CAV. The median disease free interval prior to salvage therapy was 12 months. Of 24 patients, 17 received ESHAP and 7 received ESHAP+ Rituximab as salvage therapy. The median time to transplant after initiation of salvage therapy was four months. Nineteen of twenty four patients (79%) proceeded to receive stem cell transplantation. Five patients did not proceed to ABMT secondary to failure to achieve a complete remission or had progressive disease. Patients treated with ESHAP + rituximab compared to ESHAP alone had no evidence of altered transplant related morbidity, as measured by time to engraftment, duration of hospitalization and the number of episodes of febrile neutropenia. No treatment related mortality occurred in all patients. Of the patients who underwent ABMT, 53% are disease free at a median follow up of 48 months. Among those who did not receive stem cell transplant, 4 died of progressive disease, and one has no evidence of disease in 12 months of follow up. CONCLUSIONS: ESHAP with and without Rituximab is an effective and a feasible salvage therapy for patients with relapsed lymphomas when used as a cytoreductive regimen prior to stem cell transplantation and does not negatively impact on transplant treatment related morbidity and mortality. Further studies are necessary to compare its efficacy with other standard salvage regimens prior to ABMT.

High (95%) Response Rates in Relapsed/Refractory Mantle Cell Lymphoma after R-HCVAD Alternating with R-Methotrexate/Cytarabine (R-M-A).

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 2446-2446 ◽  
Author(s):  
Jorge Enrique Romaguera ◽  
Luis E. Fayad ◽  
Michael Wang ◽  
Fernando Cabanillas ◽  
Fredrick Hagemeister ◽  
...  
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Stem Cell Transplant ◽  
Cell Lymphoma ◽  
Previous Treatment ◽  
Median Number ◽  
Cell Transplant ◽  
Mantle Cell

Abstract Mantle cell lymphoma (MCL) has a poor prognosis. Relapsed/refractory patients must respond to salvage chemotherapy in order to receive potentially curative stem cell transplantation (SCT). A salvage regimen with higher rate of response will offer the patient a better chance of survival. We have previously reported the results of R-HCVAD alternating with R-M-A in frontline therapy of MCL (Blood, 104:40a, 2004, (Abstract #128). The current trial looked at relapsed/refractory MCL patients treated also with R-HCVAD alternating with R-M-A. Since August 2001, the trial has accrued 24 out of a planned number of 41 patients of whom 21 are evaluable for response and survival. Median age was 63 years (range 45–78) and male:female ratio was 5:1. Three patients had received previous R-HCVAD alternating with R-M-A and three patients had failed an autologous stem cell transplant. Immediate therapy prior to the study for the 21 patients included R-HCVAD/SCT (1), CHOPw/wo rituximab (8 patients), cyclophosphamide, vincristine and rituximab (1), fludarabine (1), fludarabine, mitoxantrone, dexamethasone and rituximab (2), fludarabine and cyclophosphamide (1), radiotherapy (2), gemcitabine, mitoxantrone and dexamathasone (1) ifosfamide, carboplatin, etoposide and rituximab (1), Velcade (1), gemcitabine (1), and rituximab (1). The median number of prior regimens was one (range 1–6). Responses to the previous treatment included complete response (CR; 8 patients, 38%), partial response (PR; 6 patients, 29%), and no response or progression (7 patients, 33%). Results of the trial are as follows: Median number of cycles received = 4 (range 1–7), with an overall response rate (ORR) of 95% (43% CR/Cru; 52% PR). 5/5 patients who had progressed through the previous treatment responded (1CR, 4 PR), and 2/2 patients who had no change to the prior therapy responded (2 PR’s). We evaluated 12 cases whose response in our trial was classified as PR and found that in 4 of them it was the best response achieved but another 4 were referred to transplant while the tumor was still responding and in another case treatment was still ongoing. In 3 cases toxicity precluded continuation of therapy. Five (24%) of the patients were consolidated with non-myeloablative allogeneic stem cell transplantation. Sixteen were not transplanted for the following reasons: age (2 patients), lack of donor (5), Progressive disease (2), patient refusal (4), physician’s choice (1), waiting for match (1), and lost to follow up (1). Toxicity after 81 cycles included neutropenic fever (14%), grade 4 neutropenia (58%) and grade 4 thrombocytopenia (53%). The were no deaths due to toxicity. With a median follow-up of 21 months range 5–45 months), the median failure-free survival is 18 months as compared to a median FFS of 9 months response duration with the previous therapy, with no plateau in the curve. Patients who underwent stem cell transplant were censored at the time of transplant. The high response rates achieved R-HCVAD alternating with R-M-A makes this regimen an excellent choice for induction therapy prior to stem cell transplantation.

Allogeneic Stem Cell Transplant Versus Tandem High-Dose Melphalan for Front-Line Treatment of Deletion 13q14 Myeloma – An Interim Analysis of the German DSMM V Trial.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 51-51 ◽  
Author(s):  
Stefan Knop ◽  
Peter Liebisch ◽  
Holger Hebart ◽  
Ernst Holler ◽  
Monika Engelhardt ◽  
...  
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Interim Analysis ◽  
Stem Cell Transplant ◽  
High Dose ◽  
Cell Transplant ◽  
Allogeneic Stem Cell Transplant ◽  
High Dose Melphalan

Abstract Abstract 51 Background Allogeneic stem cell transplantation (allo SCT), a treatment modality based on transfer of immunocompetent donor lymphocytes offers curative potential to subjects with a variety of hematological cancers. In multiple myeloma (MM), high-dose melphalan followed by autologous stem cell transplantation (auto SCT) is adopted as a standard of care. However, it remains palliative since virtually all patients (pts) relapse and renders allo SCT an option of interest. Deletion of chromosome 13q14 (13q-) in MM has been shown to negatively impact prognosis. Therefore, improvement of therapy for 13q- pts is highly desirable. Patients and methods A prospective two-arm multi-center trial (DSMM V) was set up by our group to compare tandem high-dose melphalan 200 mg/m2 (HD Mel) with a reduced intensity conditioning allo-SCT after one cycle of HD Mel for 13q- MM. Eligibility criteria were 13q- on bone marrow FISH analysis; age up to 60 years; newly diagnosed MM in Salmon and Durie stages II and III; and measurable disease. Allocation to either treatment arm was by availability of an HLA-matched (one mismatch allowed) volunteer related (VRD) or unrelated donor (VUD). Initially, all pts received four cycles of anthracycline/dexamethasone-based induction followed by chemomobilization of peripheral blood stem cells (PBSCT) and one cycle of HD Mel. Allogeneic SCT was performed after preparation with fludarabine (30 mg/m2 for 3 consecutive days) and melphalan 140 mg/m2. ATG was administered for VUD transplants. Results 199 pts with a median age of 53 (range, 30 – 60) years were enrolled between October 2002 and March 2007 and included in this interim analysis. Sixty-seven percent had stage III disease. Allo SCT was performed in 126 of 199 pts (63%), 76 of whom (60%) received VUD allografts. The remaining 73 subjects uniformely received tandem HD Mel. Pts following allo SCT were more likely to achieve CR (59%) when compared to tandem HD Mel (32%; p=.003) within one year after end of therapy. Similarly, overall response rate was significantly higher with allo SCT (91% versus 86%; p=.003). Of note, depth of response to allo SCT was not associated with presence of acute graft-versus-host disease (GVHD): 62% CR with grades II to IV GVHD vs 58% CR with grades 0 and I (p=.75). Treatment-related mortality (TRM) at 2 years from allo SCT was 16/126 (12.7%). At a median follow up of 25 months for tandem HD Mel and 34 months for allo SCT, projected 3-year overall survival is 72% (auto) and 60% (auto/allo SCT; p=0.22), respectively. Conclusions This is the largest trial on first-line allogeneic stem cell transplant in MM so far. Our interim results show a higher CR rate in FISH 13q- subjects undergoing allo SCT when compared to tandem HD Mel. Despite a majority of allografts in our study being delivered from unrelated donors, TRM was comparable to trials confined to sibling transplants. At a relatively short follow-up, there is not yet a difference between both arms regarding OS, albeit longer follow-up may be important as previously described. This as well as analysis of the impact of donor type and chronic GVHD on outcome will be presented at the meeting. Disclosures: No relevant conflicts of interest to declare.

Finalization of autologous stem cell transplant in complex and multirelapsed follicular lymphoma

2020 ◽  
Vol 106 (6) ◽  
pp. NP5-NP8
Author(s):  
Matteo Carella ◽  
Vittorio Stefoni ◽  
Cinzia Pellegrini ◽  
Lisa Argnani ◽  
Michele Cavo ◽  
...  
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Follicular Lymphoma ◽  
Cell Transplantation ◽  
Stem Cell Transplant ◽  
Young Patients ◽  
High Dose ◽  
Cell Transplant ◽  
Aggressive Approach ◽  
Frequent Relapses

Background: Follicular lymphoma (FL) is characterized by frequent relapses and need for multitude lines of therapy, which includes different immunochemotherapy regimens, novel monoclonal antibodies, novel drugs, and autologous or allogenic stem cell transplant. Early use of autologous stem cell transplantation (ASCT) improves prognosis in patients with FL who may be candidates for an aggressive approach. Case presentation: We report the case of a 49-year-old woman with thrombophilia with relapsed/refractory grade 3A FL, heavily pretreated, who achieved third complete remission after high-dose chemotherapy and ASCT, despite experiencing life-threatening adverse events during her treatment history. Conclusions: Stem cell transplantation has emerged as the standard of care for young patients with FL but may be effective also in complex and multirelapsed clinical cases.

scholarly journals Autologous Hematopoietic Stem Cell Transplantation for Refractory Lupus Nephritis

2019 ◽  
Vol 14 (5) ◽  
pp. 719-727 ◽  
Author(s):  
Xianghua Huang ◽  
Wencui Chen ◽  
Guisheng Ren ◽  
Liang Zhao ◽  
Jinzhou Guo ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Lupus Nephritis ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Disease Free Survival ◽  
Cell Transplant ◽  
Hematopoietic Stem

Background and objectivesOur study evaluated the efficiency and safety of autologous hematopoietic stem cell transplantation treatment for patients with refractory lupus nephritis.Design, setting, participants, & measurementsFrom July 2011 to January 2015, a total of 22 patients with refractory lupus nephritis were enrolled in this study. Peripheral blood stem cells were mobilized with cyclophosphamide and granulocyte colony stimulating factor and reinfused after treatment with cyclophosphamide and antithymocyte globulin. The primary end point was the rate of remission, and secondary end points included the survival and relapse rates, changes in proteinuria, kidney function, and serology immunologic test. All complications were recorded for safety assessment.ResultsTwenty-two patients were enrolled and underwent stem cell mobilization. There were nine men and 13 women, with a median lupus nephritis duration of 46 (33–71) months. The mean number of CD34+ cells was (7.3±3.8)×106/kg. All patients had successful engraftment, and the median times of granulocyte and platelet engraftment were 8 (7–9) and 9 (6–10) days, respectively. The major complications of stem cell transplantation were fever and gastrointestinal tract symptoms. The treatment-related mortality was 5% (one of 22). After a median follow-up of 72 (60–80) months, 18 (82%) patients achieved completed remission, one (5%) patient achieved partial remission, and one patient had no response and received peritoneal dialysis at 12 months after transplantation. The 5-year overall survival and disease-free survival rates were 91% and 53%, respectively. Six patients experienced relapse during the follow-up, and the relapse rate was 27%.ConclusionsAutologous hematopoietic stem cell transplant could be used as a treatment option for refractory lupus nephritis, because it was relatively safe and associated with good outcomes.

Incidence and Risk Factors for Thromboembolism in Patients with Hematologic Malignancies Undergoing Allogeneic and Autologous Stem Cell Transplantation.

Blood ◽  
2004 ◽  
Vol 104 (11) ◽  
pp. 1141-1141
Author(s):  
Nicole M. Kuderer ◽  
Alok A. Khorana ◽  
Jonathan W. Friedberg ◽  
Eva Culakova ◽  
Gordon L. Phillips ◽  
...  
Keyword(s):  
Risk Factors ◽  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Stem Cell Transplant ◽  
Multivariate Logistic Regression Analysis ◽  
Hematologic Malignancies ◽  
Cell Transplant ◽  
Multivariate Logistic Regression ◽  
Transplant Patients

Abstract Background: Thromboembolism (TE) is a common complication of hospitalized non-transplant cancer patients. To date, the extent and impact of TE in stem cell transplant patients is unknown, in part because it is considered a low risk group given the high prevalence of thrombocytopenia. The purpose of this study is to evaluate the incidence of and risk factors for TE in cancer patients undergoing stem cell transplantation. Methods: We conducted a retrospective analysis of all discharge summaries from the 115 US academic health centers reporting to the University HealthSystem Consortium from 1995 to 2002. We identified a total of 7,087 patients with hematologic malignancies undergoing allogeneic and autologous stem cell transplantation. The incidence of and risk factors for venous and arterial TE was analyzed in univariate and multivariate logistic regression analysis with adjusted odds ratios as estimates of relative risk. Results: TE was reported in 389 (5.5%) transplant patients with 4.8% patients developing venous and 0.7% arterial TE. The incidence of TE was greater in allogeneic (6.8%) compared to autologous (4.8%) transplant patients (p<0.0001). Among those receiving allogeneic transplantations, BMT patients experienced a higher rate of TE than PBSC patients (7.5% vs 5.6%; p<0.05). In multivariate logistic regression analysis, the following clinical variables were significantly associated with TE in stem call transplant patients with hematologic malignancies: gram negative sepsis (OR=1.76; 1.02–3.02; p=0.04), gram positive sepsis (OR=1.75; 1.25–2.45; p=0.001), line infections (OR=1.48; 1.11–1.97; p=0.008), central venous catheters (OR=1.74; 1.39–2.17; P<0.0001), pulmonary disease (OR=1.87; 1.47–2.37; p<0.0001), liver disease (OR=1.31; 1.01–1.70; p=0.04) and length of stay >30 days (OR=1.66; 1.30–2.13; p<0.0001). Hodgkin’s disease (OR= 0.59; 0.37–0.94; p=0.026) was associated with a lower risk of TE. In multivariate analysis, the type of transplant failed to remain an independent risk factor for TE after controlling for other transplant complications. Conclusions: This is the first substantive report on the incidence of thromboembolism in stem cell transplant patients. We found that thromboembolic events are a frequent complication in patients with hematologic malignancies undergoing stem cell transplantation. The incidence of TE is high among most subgroups studied. Prospective studies are needed to evaluate the efficacy and safety of thromboprophylaxis in this high-risk population.

Front-Line Consolidation with Autologous Stem Cell Transplantation in Patients with Nodal Aggressive Peripheral T-Cell Lymphoma (PTCL). A Prospective Study of the GEL-TAMO.

Blood ◽  
2006 ◽  
Vol 108 (11) ◽  
pp. 2463-2463
Author(s):  
Jose Rodriguez ◽  
Eulogio Conde ◽  
Antonio Gutierrez ◽  
Reyes Arranz ◽  
Angel Leon ◽  
...  
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Autologous Stem Cell Transplantation ◽  
Salvage Therapy ◽  
Front Line ◽  
Free Survival ◽  
Line Therapy ◽  
Gallium Scan

Abstract Background: Nodal aggressive PTCL have a poor prognosis with conventional chemotherapy regimens for aggressive non-Hodgkin lymphomas. Therefore innovative approaches are needed. Among them, strategies including consolidation with high-dose chemotherapy and autologous stem cell transplantation in front line therapy may improve the outcome of these diseases. Aim: To report the experience of a national cooperative group in 26 patients with aggressive nodal PTCL who underwent treatment including ASCT as consolidation of front line therapy. Patients and Methods: Twenty-six patients Gallium scan positive with nodal PTCL entered into the study: 11 cases of PTCL-unspecified (42%), 8 ALCL (31%) and 7 AIL (27%). Patients received three courses of MegaCHOP and subsequently were evaluated by CT scan and Gallium scan. Those patients Gallium scan negative received another course of MegaCHOP and then the transplant. Those patients Gallium scan positive after 3 courses of MegaCHOP received 2 courses of the IFE regimen (Ifosfamide 3.3g/m2 days 1–3 and Etoposide 300 mg/m2 days 1–3) and if at least achieved a partial response received the transplant otherwise they are out of the study. Median age was 44 years, 96% of the patients presented Ann Arbor stage III or IV; 54% presented B symptoms; 31% bone marrow involvement and 72% of the patients presented 2–3 factors of the a-IPI. Results: Thirteen patients (50%) achieved a CR after 3 courses of MegaCHOP and 12 patients received IFE as salvage therapy. Twenty patients out of the 26 initial patients were able to proceed to the transplant. After the transplant 85% of patients achieved a CR. The 6 patients who did not received the transplant was due to progression of the disease in 4 cases, lethal toxicity in one case and refusal in another case. With a median follow up of 35 months for alive patients, the overall survival (OS) and progression-free survival (PFS) at 3 years were 73% and 53%, respectively. OS, PFS and disease-free survival for the 20 patients who underwent the ASCT consolidation was 84%, 53% and 63%, respectively. Conclusion: Early salvage therapy for patients not in CR after 3 courses of chemotherapy and ASCT consolidation for chemosensitive patients may improve the outcome of patients with nodal aggressive PTCL. Larger series and longer follow up are needed to confirm this promising approach.

Norovirus Infections in Patients with Hematological Diseases and After Stem Cell Transplantation; Epidemiological and Clinical Aspects.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 2661-2661
Author(s):  
Per T. Ljungman ◽  
Per Bernell ◽  
Richard Lerner ◽  
Jonas Mattsson ◽  
Maria Rotzén Östlund ◽  
...  
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Median Duration ◽  
Stem Cell Transplant ◽  
Positive Sample ◽  
Clinical Aspects ◽  
Cell Transplant ◽  
Hematological Diseases ◽  
Virus Excretion

Abstract Abstract 2661 Poster Board II-637 Norovirus infections have become a major practical clinical problem during the last few years causing outbreaks in many different situations including in hospitals infecting both patients and staff. These infections have also been associated with prolonged virus excretion in renal transplant recipients and a risk of mortality in the elderly. Little is known about the clinical impact of norovirus infections on patients who are severely immunosuppressed. The aim of this study was to analyze the impact of norovirus infections in patients with hematological diseases and after hematopoietic stem cell transplantation (HSCT). The laboratory records from the Clinical Microbiological Laboratory at Karolinska University Hospital were examined in order to identify patients with proven norovirus infection hospitalized on the haematology or allogeneic stem cell transplant wards from 2006 to 2009. The diagnostic methodology was based on an accredited protocol including a reverse transcription real- time PCR procedure with the use of oligonucleotide primers specific for detection of norovirus genotype 1 or 2 in separate wells. After identification of cases, the patient charts were reviewed to assess outcome, possible norovirus associated clinical complications, and delay of antitumor therapy. The duration of virus excretion was defined as the time from the first to the last positive sample. 65 patients were identified. 19 patients had NHL, 14 AML, 8 multiple myeloma, 8 non-malignant hematological disorders, 5 ALL, 5 CLL, 4 MDS, and one patient had CML. 24 patients had undergone HSCT; 22 allogeneic and 2 autologous. The median age was 63.1 (1.1–84.2). The cases occurred in two major and 3 minor clusters over the 3 year period with some additional sporadic cases occurring between the clusters. One of the haematology wards had to be closed for admission twice and one ward once since also several cases occurred among the staff. 17 of the detected viruses were typed to genogroup 2, 2 to genogroup 1, and 46 were not typed. 29 patients had only one positive sample of which 11 had a negative follow-up sample. The median duration of viral detection in the entire cohort was 2 days (1–216 days). Among the patients with more than one positive sample, the median duration was 15 days (2–216 days). 25/65 (38%) patients were PCR positive more than one week, 18 (28%) for more than two weeks, and 9 (14%) for more than four weeks. The majority of patients had minor and quickly resolved gastrointestinal symptoms. Five patients died in close temporal association with the norovirus infection (within a week). Three patients had fluid balance and electrolyte abnormalities and in of these a pre-existing renal failure worsened and the patient required dialysis. One patient died from pneumonia and one patient died from multiple causes with an end-stage malignancy. Seven patients (11%) had planned cytotoxic chemotherapy postponed; one of these patients had a delay in a planned allogeneic HSCT. We conclude that norovirus infection is a significant clinical complication to management of patients with hematological malignancies and stem cell transplant patients. Fatal outcome is possible primarily in patients with severe underlying conditions. Delay in planned chemotherapy was common and in addition the required closing of the ward presumably delaying chemotherapy for other non-infected patients. Disclosures: No relevant conflicts of interest to declare.

5-Azacytidine Before or After Stem Cell Transplantation in Acute Myeloid Leukaemia (AML) and Myelodysplastic Syndromes (MDS)

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 4267-4267
Author(s):  
Ana Garrido ◽  
Miguel Ortín ◽  
Rodrigo Martino ◽  
Josep Nomdedeu ◽  
Ana Aventin ◽  
...  
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Disease Progression ◽  
Cell Transplantation ◽  
Conflicts Of Interest ◽  
Conditioning Regimen ◽  
Complete Response ◽  
Cell Transplant ◽  
Safe Procedure

Abstract Abstract 4267 The acceptable toxic profile of 5-aza-citidine (5-aza) allows its use in fragile or elderly patients in whom intensive chemotherapy should be avoided. Whether it is possible to take advantage of this low toxicity in patients awaiting for donor search and/or stem cell transplant (SCT) and in those experiencing leukemia recurrences after the procedure remains unknown. We analysed the clinical results of using 5-aza in these two settings to define the feasibility, safety and results of this approach. Patients and methods: From 2007 to 2011, 15 patients (11 males, 4 females) received 5-aza as last treatment prior to an allogeneic SCT (n=13) or as rescue after an early post-transplant relapse (n=2) at our centre. Diagnosis was MDS in 3 cases (median age 62; range 58–63) and AML in 12 cases (median age 58; range 37–67). Patients with MDS received a median of 6 courses of 5-aza (range 3–8) as the only treatment from diagnosis, except for one patient who had received panobinostat prior to 5-aza. Amongst patients with AML, 12 patients received 5-aza either as treatment for AML (2/12) or after remission (8/12) because of the high relapse risk while awaiting for a suitable donor to be found. Two patients with AML received 5-aza as treatment for early post-SCT relapse. AML patients treated with 5-aza before SCT received a median of 5 courses (range 1–19), whilst patients receiving treatment for relapse received 1 and 3 courses, respectively. Ten patients received a nonmyeloablative conditioning regimen, 1 received a conventional conditioning regimen, 2 patients are still in the process of donor search and the other 2 patients received 5-aza after an autologous stem cell transplantation relapse. RESULTS: All MDS patients engrafted and are in complete remission (CR) after a median of 696 days of follow-up (range 377–1227). One of those patients died because of aGvHD. Nine of 12 AML patients receiving 5-aza prior to SCT are alive after a median 373 days follow-up (133–995). One patient showing refractoriness to 3 different lines of treatment died from disease progression after 211 days. All patients receiving 5-aza as treatment for early relapse are dead, 41 and 401 days after starting treatment. Most interestingly, AML patients receiving 5-aza as maintenance of an already-achieved CR while awaiting transplantation did not experience disease progression despite the median time they remained on this treatment was prolonged (9 months). Graft-versus-host disease ≥ grade II was seen in 3 patients. No graft failures were seen and all patients who received an allogeneic stem cell transplantation remain in complete response. CONCLUSION: The use of 5-aza for maintaining or achieving a response in patients with AML who are awaiting SCT is a safe procedure and adds flexibility to schedule the treatment without the need to administer potentially toxic therapy. The use of 5-aza before transplant did not appear to interfere either with engraftment, incidence of GvHD or short-term relapse after transplant. Disclosures: No relevant conflicts of interest to declare.

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