scholarly journals Hematopoietic Stem Cell Transplantation in Children with Refractory Langerhans Cell Histiocytosis

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 4657-4657
Author(s):  
Kazuko Kudo ◽  
Miho Maeda ◽  
Nobuhiro Suzuki ◽  
Hirokazu Kanegane ◽  
Shouichi Ohga ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Median Time ◽  
Langerhans Cell Histiocytosis ◽  
Japanese Society ◽  
Disease Status ◽  
Hematopoietic Stem ◽  
Active Disease

Abstract Introduction Hematopoietic stem cell transplantation (HSCT) is one of the promising treatment strategies for children with refractory Langerhans cell histiocytosis (LCH), because of its immunomodulatory effects.Efficacy and indication of HSCT has been still undetermined. We analyzed the outcomes of HSCT in children with refractory LCH registered in the Transplant Registry Unified Management Program (TRUMP) conducted by the Japanese Society for Hematopoietic Cell Transplantation. Patients and methods Between 1996 and 2014, 30 patients <15 years old with refractory LCH who underwent an allogeneic HSCT were registered in the TRUMP database. Data collected as of March 2017 were analyzed. The histiocyte committee of the Japanese Society of Pediatrc Hematology and Oncology (JSPHO) sent the questionnaires to the institutions where HSCT was done for LCH, and clinical data of 25 patients were collected. Definition of response to initial therapy and disease state at HSCT was described previously. Failures were defined as relapse after HSCT, secondary malignancy, and death from any causes. Results Patients' characteristics The male/female ratio was 18/12. Ages of onset of LCH and at BMT were median 9 months (range 3-23 months) and median 1 years (range 0-11 years), respectively. At diagnosis of LCH, 19 patients were positive for risk organ involvement, 6 were negative. Eleven patients underwent HSCT using myeloablative conditioning (MAC) regimen, whereas 19 patients reduced intensity conditioning (RIC) regimen. Eight patients received total body irradiation (TBI) > 8Gy (10-12Gy) regimen, 3 received full dose busulfan (BU: 16mg/kg po or 17.6mg/kg iv) regimen. Seven received fludarabine (FLU: 125-180mg/m2) + melphalan (MEL: 70-180mg/m2) without TBI and 9 received FLU(120-180mg/m2)+MEL (140-180mg/m2) with low dose irradiation (2-5.1Gy) and the remaining 3 received other RIC regimens. Cyclosporine was used in 13 patients and tacrolimus was used in 16 patients for graft versus host disease (GVHD) prophylaxis. Donor sources were related donor in 9 patients and unrelated in 21 patients (cord blood 19 and bone marrow 2). Four related donors were father or mother and 3 of them were haplo-identical. In these patients, induction therapy at onset achieved a good (5/25), partial (8/25), no (4/25) and progressive (8/25) diseases. In regard to disease status at HSCT, recipients with no active diseae (NAD) were 4/25, active disease-regression (AD-r) 2/25, active disease-stable (AD-s) 4/25 and progressive (AD-p) 15/25. Eight patients received 2-chlorodeoxyadenosine (2-CdA), including 4 patients who received the combination of 2-CdA and high dose Ara-C before HSCT as salvage therapy. At HSCT, 15/23 (65%) patients were in primary induction failure and 8/23 (35%) experienced first or additional relapse, respectively. Transplantation outcomes Neutrophil recovery was observed in 24 patients and the median time to engraftment was 21 days. Platelets engraftment was observed in 17 patients and the median time to engraftment was 52 days. Acute GVHD of grade II - IV, chronic GVHD (cGVHD) were observed in 6 and 4 patients, respectively. Extensive cGVHD was observed in 3 patients who received MAC regimens (p= 0.079). Relapse after HSCT were observed 1 patient in RIC regimens and 2 patients in MAC (P=0.613). With follow-up of median 433 days (range 9-5307days) after HSCT, 17/30 (57%) patients are alive and 13 died. Death occurred within 3 months after HSCT in 8/13. The overall survival (OS) was not different between RIC and MAC (56.8% vs 63.6%, p=0.843). In regard to the correlation of disease status at HSCT and outcome, 6 patients with NAD/ AD-r had better outcome than 19 with AD-s/ AD-p (5-year OS 100% vs 52.1%, p=0.035). The 5-year FFS of the 16 patients who received FLU+ MEL based regimen were 68.8%, and were marginally better than those of the 14 patients who were conditioned with other regimens (68.8% (95%CI: 46.0-91.5) vs 42.9% (95%CI: 16.9-68.8), P=0.209). Discussion In conclusions, of 30 HSCT-recipients for refractory LCH, 17/30 (57%) are alive while post-transplant death occurred in 13/30 (43%). Our study showed that in regard to the correlation of disease status at HSCT and outcome, 6 patients with NAD/ AD-r had better outcome than 19 with AD-s/ AD-p (5-year OS 100% vs 52.1%, p=0.035). Novel bridging measures, such as targeted inhibition of the MAPK pathway, are required to stabilize the disease activity before HSCT. Disclosures No relevant conflicts of interest to declare.

Faster Registration on International Donor Registries and Shorter Time to Allogeneic Hematopoietic Stem Cell Transplantation After Having Found a Donor Confers Better Outcome In Acute Leukemia Patients

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 2371-2371
Author(s):  
Mauricette Michallet ◽  
Mohamad Sobh ◽  
Stephane Morisset ◽  
Xavier Thomas ◽  
Youcef Chelghoum ◽  
...  
Keyword(s):  
Stem Cell ◽  
Molecular Markers ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Acute Leukemia ◽  
Cell Transplantation ◽  
Median Time ◽  
Disease Status ◽  
No Donor ◽  
Hematopoietic Stem

Abstract Abstract 2371 Background: Allogeneic hematopoietic stem cell transplantation (allo-HSCT) appears to offer a potential advantage in terms of event free survival (EFS) and overall survival (OS) in patients with acute leukemia (AL) with bad prognostic factors. The main issue is to find a donor; usually, a patient has 30% chance to find an HLA identical sibling donor (SD) and approximately 40% chance to find a suitable unrelated donor (UD) from international registries. Unfortunately, 40% of registered patients relapse or die before finding a donor. Aim: Our objective was to evaluate the outcome (OS & EFS) in AL patients, whether they had an HLA identical SD, an UD or no donor (ND) after registration on France Greffe de Moelle (FGM) registry, either transplanted later or not. Our secondary objective was to evaluate the impact of interval between, diagnosis and allo-HSCT, donor finding and allo-HSCT, interval registration-allo-HSCT, on OS and EFS. Methods: We have analyzed 251 AL patients diagnosed between January 2000 and December 2008, for whom a search for a donor was initiated for a required allo-HSCT. There were 117 (47%) males and 134 females with a median age at diagnosis of 40 years [16-66], 177 (71%) were AML (prognosis according to cytogenetics & molecular markers: 59 were good, 97 poor and 21 not done) 75 were ALL (prognosis according to cytogenetics & molecular markers: 33 were good, 16 poor and 26 not done). Seventy six (30%) patients had an available SD and received allo-HSCT within a median time of 3.5 months (0.5 – 43) (59 AML & 17 ALL), and 38 (15%) with SD but were not transplanted due to early relapse and/or death. For patients with no available SD, a registration on FGM registry was done. One hundred thirty seven patients were registered after a median interval of 2.3 months (0.4-135) from diagnosis, 33 (13%) patients (24 AML & 9 ALL) did not find any donor, have not been transplanted and they received the standard of care. One hundred and four (41%) patients (62 AML & 42ALL) found an UD or cord blood cell (CBC) unit after a median registration time of 1.6 months (0.3 – 26): 86 with UD of which only 60 have been transplanted within a median time of 2.3 months (0.4 – 14), 18 with CBU of which only 17 were transplanted. Among transplanted patients, 113 (74%) were in complete response (CR) at transplantation, and 40 were in less than CR. Fifty (33%) received peripheral blood (PBSC) (23 UD & 27 SD), 86 (57%) received bone marrow (BM) (37 UD & 49 SD) and 17 (10%) CBC units. For conditioning, 56 (37%) were reduced intensity (29 SD & 27 UD) and 96 standard (47SD & 50 UD). For HLA, there were 45 HLA 10/10, (27BM & 18 PBSC), 14 HLA 9/10 (9BM 5PBSC) 1 HLA 8/10 (BM) and for CBC 14 HLA 4/6 and 3 HLA 5/6. Results: After a median follow-up of 25 months (0.2- 234), the median OS was 78 months (51 – 133) for transplanted patients with SD (3years OS: 68%), it was 33 months (27 – 47) for transplanted patients with UD (3years OS: 44%), 21 months (15 – 37) for not transplanted patients with available SD or UD (3years OS: 34%) and finally it was 31 months (23-221) for patients with ND (3years OS: 45%). The median EFS for the same groups was 38 months (23 – 133), 24 months (17 – 36), 15 months (11-24) and 23 months (14 – 48) respectively. The only factors negatively affecting OS in multivariate analysis (studying age, sex, AL type, cytogenetics, donor or no donor, transplanted or not, UD or SD) were age and allo-transplanted from UD. When adjusting only on transplanted patients, taking into account in addition to previous factors, the time between diagnosis-registration, time between registration-allo-HSCT, disease status at allo-HSCT, stem cell source, conditioning; three significant factors affected OS: disease status (<CR) HR= 2.8 [1.5-5.3] p<0.001; long interval between diagnosis-registration HR= 2 [1.2-3.6] p=0.001 and conditioning (standard) HR=0.27 [0.1-0.8] p=0.02. (3years OS for SD allo-HSCT, UD allo-HSCT late and early registration: 59%, 29% and 47% respectively). Conclusion: The results of our first analysis seemed surprising regarding our knowledge of outcome in allo-HSCT from SD and UD, which led us to investigate on unusual interfering factors. We have explored the relation between the interval diagnosis-registration for a donor search and the interval registration-allo-HSCT that appeared as major factors affecting survival in UD allo-HSCT settings. Disclosures: No relevant conflicts of interest to declare.

scholarly journals Bendamustine, Carfilzomib, and Melphalan (BCM) Conditioning Prior to Autologous Hematopoietic Stem Cell Transplantation for Multiple Myeloma: A Feasibility Study

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 4635-4635
Author(s):  
A. Samer Al-Homsi ◽  
Marlee Muilenburg ◽  
Kelli Cole ◽  
Muneer H. Abidi ◽  
Stephanie F. Williams
Keyword(s):  
Multiple Myeloma ◽  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Partial Response ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Median Time ◽  
Conditioning Regimen ◽  
Disease Status ◽  
Hematopoietic Stem

Abstract High-dose melphalan (M) followed by autologous hematopoietic stem cell transplantation (AHSCT) remains the standard treatment for multiple myeloma in eligible patients, even in the era of novel agents. However, the majority of patients ultimately relapse and succumb to their disease. Several studies have explored integrating novel agents into the conditioning regimen prior to AHSCT in order to improve complete remission rates and ultimately overall survival. We aimed to assess the feasibility of adding bendamustine (B) and carfilzomib (C) to melphalan (BCM) and performed a phase I dose escalation study. Thirteen patients were enrolled between June 2014 and June 2016. All patients received C at a fixed dose (20 mg/m2) on days (d) -29, -28, -22, -21, -15, -14. The conditioning regimen and doses administered for each cohort were as described in the table below. Due to excessive toxicity, the study was amended after the first 6 patients. Per oversight of a data safety monitoring board, the dose of M was reduced to 140 mg/m2 and C dose on d +6 was omitted. Median age was 58 years (39-68). There were 8 males and 5 females. Performance status was ≥ 80% in all patients. Per the International Staging System (ISS), 3 patients had stage I disease, 5 had stage II, 4 had stage III, and 1 had unknown staging. Three patients had high-risk cytogenetics: 2 with t(4;14) and 1 with deletion 17p. Three patients had received prior AHSCT. Disease status prior to study treatment was stable disease (SD) (n=2), partial response (PR) (n=8), or very good partial response (VGPR) (n=3). Median CD34+ cell dose was 3.24x106/kg (2.23-6.92x106). Median follow-up was 14.2 months (1-24.5). Median time to neutrophil engraftment was 12 d (11-15). One patient died before achieving platelet engraftment. For the remaining patients, median time to platelet engraftment was 16 d (12-20). Non-hematologic toxicities included grade 3 acute mucositis (n=1), lower GI complications (n=6), electrolyte disturbances (n=6), transaminase elevation (n=1) renal insufficiency (n=1), pulmonary edema (n=1), prolonged QTc (n=1), atrial fibrillation (n=1), and elevated troponin (n=1) and grade 4 acute sepsis (n=2), resulting in 1 death in cohort 2 on d +44. Seven patients went on to receive maintenance therapy: 3 with bortezomib, 3 with lenalidomide, and 1 with lenalidomide, dexamethasone, and C. Post-transplant disease status was assessed per protocol by SPEP, SPIF, and serum free light chains and light chain ratio. Nine patients were evaluable on d +100. One patient had SD, 6 had VGPR, and 2 had complete response (CR). Six out of 7 (86%) evaluable patients on d +365 remain disease progression-free. In summary, BCM conditioning prior to AHSCT at the doses described in cohort 3b seems feasible with manageable toxicities. Five additional patients are being enrolled at the same dose level. Table Table. Disclosures No relevant conflicts of interest to declare.

scholarly journals Induction Treatment and Feasibility of Allogeneic Hematopoietic Stem Cell Transplantation in Young Patients (<65 years) with High-Risk Cytogenetic and Secondary Acute Myeloid Leukemia (AML): A Single Center Experience in Argentina with CLAG-M and 7+3

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 5132-5132
Author(s):  
Maria Lucia Fuente ◽  
Maria Del Rosario Custidiano ◽  
Santiago Cranco ◽  
Laura Korin ◽  
Paola Ochoa ◽  
...  
Keyword(s):  
Stem Cell ◽  
High Risk ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Median Time ◽  
Induction Treatment ◽  
Hematopoietic Stem ◽  
Free Survival

BACKGROUND Patients with adverse cytogenetic or secondary AML (s-AML) have significantly worse outcomes and lower survival rates. In this high risk subgroup of patients, early consolidation with allogeneic hematopoietic stem cell transplantation (HSCT) in first complete remission (CR1) can improve results, especially in those who achieve negative measurable residual disease (MRD-). More effective treatments than standard 7+3 are needed. CLAG-M is a salvage regimen that has demonstrated high response rates with good tolerance, and seems to be promising in the upfront setting. AIMS To estimate CR and MRD- rates, overall survival (OS) and event free survival (EFS) in transplant eligible patients with high risk AML treated in our center.To compare CR rate and transplant feasibility in CR1 with 7+3 vs. CLAG-M as induction treatment in s-AML. PATIENTS AND METHODS We analyzed adult patients (18-65 years old) with high risk AML (defined by adverse cytogenetic according to ELN2017 or s-AML) who were treated in our institution between 2010 and 2018. All patients were transplant eligible and had an available donor. Clinical information was collected from medical records. We evaluated CR1 and MRD- rates, EFS and OS. We also compared CR rates and HSCT feasibility in s-AML after treatment induction with CLAG-M and 7+3. The survival analysis was estimated with Kaplan-Meier method and the comparison between variables was performed through log-rank test. RESULTS Twenty-one patients were included (13 s-AML and 8 with adverse cytogenetic). The median age at diagnosis was 54 years (21-64); 13 female/8 male. Out of 21 patients, 14 received 7+3 induction and 7 CLAG-M. The median follow-up time was 11 months (0.9-90.8), median EFS and OS for the whole group was 1.05 and 13.5 months, respectively. Two-year OS was 35%. CR1 was achieved in sixteen patients (76%), 10 of them MRD-. The median time to CR1 was 33 days, the median OS of these patients was 26.7 months (figure 1). Eleven patients (52%) were refractory to first induction, 10/14 in the 7+3 subgroup, and only 1/7 patients treated with CLAG-M. Six of them converted to CR after reinduction (5 with CLAG-M). Fourteen (67%) underwent HSCT in CR1. The median time to HSCT consolidation was 106 days. The median relapse free survival in transplanted patients has not been reached (Table 1). Considering only s-AML, 6 patients received 7+3 and 7 CLAG-M. Median age in 7+3 subgroup was 41 vs. 57 years in CLAG-M. The median OS was 13.5 months. In the 7+3 cohort, only 1 achieved CR (16%); the other five received reinduction with CLAG-M, and 4 converted to CR1. The median time to CR1, EFS and OS were 82 days, 1 month and 26 months respectively. In contrast, 4 of the 7 patients (57%) that received CLAG-M achieved CR1, but only 1 of the 3 that were refractory could convert to CR. The median time to CR1 in patients treated with CLAG-M was 27 days, median EFS 7.5 months and median OS has not been reached (Figure 2). There were no statistically significant differences between the two treatment groups. Eight patients (62%) could be bridged to HSCT, 4 of each subgroup (Table 2). CONCLUSIONS Our results in this real life small cohort of high risk AML were similar to historical controls. In the s-AML subgroup, differences between 7+3 and CLAG-M were not statistically significant probably due to the low number of patients analyzed. However, patients who received CLAG-M required less cycles of treatment to achieved CR1, allowing HSCT rapidly in this selected population. Since most of the refractory patients to 7+3 responded to reinduction with CLAG-M, both groups had similar transplant rates. According to our experience CLAG-M might be an attractive treatment option with high CR rates and acceptable safety profile. In this high risk AML population, two thirds of the patients were effectively "bridged" to HSCT with a 2-year OS rate of 35%. Disclosures No relevant conflicts of interest to declare.

scholarly journals Tandem autologous hematopoietic stem cell transplantation for treatment of adult T-cell lymphoblastic lymphoma: a multiple center prospective study in China

Haematologica ◽  
2019 ◽  
Vol 106 (1) ◽  
pp. 163-172 ◽  
Author(s):  
Yao Liu ◽  
Jun Rao ◽  
Jiali Li ◽  
Qin Wen ◽  
Sanbin Wang ◽  
...  
Keyword(s):  
Stem Cell ◽  
T Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Disease Status ◽  
Lymphoblastic Lymphoma ◽  
Hematopoietic Stem ◽  
Chemotherapy Group

T-cell lymphoblastic lymphoma (T-LBL) is a highly aggressive form of lymphoma with poor clinical outcomes and lacks of a standard treatment regimen. In this study, we assessed the safety and efficacy of tandem autologous hematopoietic stem cell transplantation (auto-HSCT) strategy for adult T-LBL and evaluated prognostic factors affecting survival. 181 Newly-diagnosed adult T-LBL patients were enrolled, 89 patients were treated with chemotherapy alone, 46 patients were allocated to single auto-HSCT group, 46 patients were treated with tandem auto-HSCT. The median follow-up time was 37 months, the 3-year progression/relapse rate of the tandem auto-HSCT group was significantly lower than that of the single auto-HSCT group and chemotherapy group (26.5% vs 53.1% and 54.8%). The 3-year PFS and OS rate of the tandem auto-HSCT group (73.5% and 76.3%) were significantly higher than those of the single auto-HSCT group (46.9% and 58.3%) and the chemotherapy group (45.1% and 57.1%). In the tandem auto-HSCT group, age and disease status after the first transplantation impacted the OS and PFS. Multivariate analysis identified that disease status after the first transplantation was the only independent prognostic factor for patients treated with tandem-HSCT. In addition, diagnostic models of the initial CD8+CD28+/CD8+CD28- T cell ratio in predicting the disease status were found to be significant. Taken together, tandem auto-HSCT can be considered an optimal strategy for adult T-LBL patients (ChiCTR-ONN-16008480).

scholarly journals Autologous hematopoietic stem cell transplantation for efficient treatment of multisystem, high-risk, BRAF V600E-negative Langerhans cell histiocytosis

2019 ◽  
Vol 47 (9) ◽  
pp. 4522-4529
Author(s):  
Yaozhu Pan ◽  
Rui Xi ◽  
Cunbang Wang ◽  
Lei Fang ◽  
Jiaofeng Bai ◽  
...  
Keyword(s):  
Stem Cell ◽  
High Risk ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Lymph Nodes ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Langerhans Cell Histiocytosis ◽  
Braf V600e ◽  
Hematopoietic Stem

Langerhans cell histiocytosis (LCH) is a disorder caused by clonal proliferation of CD1a+/CD207+ cells and characterized by varying degrees of organ involvement. Treatment of LCH is risk adapted; patients with multisystem disease and risk-organ involvement require more intensive therapy. Optimal therapies for multisystem, high-risk LCH remain uncertain. Recently, targeted therapy using inhibitors of mutated BRAF (the gene encoding serine/threonine-protein kinase B-Raf) has proven very effective in patients with multisystem refractory LCH. Herein, we report a case of LCH with involvement of the bones, liver, and lymph nodes. Using next-generation sequencing of the patient’s pathological sample, we identified a mutation in MAP2K1 in exon 3 (c.362G>C, p.Cys121Ser) and no mutation in BRAF; thus, high-risk, multisystem LCH with MAP2K1 mutation and wild-type BRAF was diagnosed. After four chemotherapy treatments (COEP regimen), the patient received autologous hematopoietic stem cell transplantation (auto-HSCT). Complete remission was confirmed by follow-up positron emission tomography–computed tomography, which showed no lesions in liver, lymph nodes, or bones compared with the pretreatment period. To date, the patient has sustained good health for 24 months. In conclusion, auto-HSCT may be an effective treatment option for high-risk, multisystem BRAF V600E-negative LCH.

Bortezomib Therapy for Multiple Myeloma in Relapse after Reduced Intensity Conditioning Allogeneic Hematopoietic Stem Cell Transplantation (RICT).

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 5399-5399
Author(s):  
Sophie Ducastelle ◽  
Daniela Revesz ◽  
Jacques Troncy ◽  
Samira Mahmoudi ◽  
Xavier Thomas ◽  
...  
Keyword(s):  
Multiple Myeloma ◽  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Median Time ◽  
New Drugs ◽  
Hematological Toxicity ◽  
Hematopoietic Stem

Abstract Multiple myeloma (MM) remains incurable and specially of poor prognosis in case of refractory or relapsed disease. However, recent therapeutic advances including the use of new drugs are promising. Although, Bortezomib, new proteasome inhibitor, has proven its efficacy in patients with refractory or relapsed MM, it has not been sufficiently evaluated in the setting of allogeneic hematopoietic stem cell transplantation. Here, we report four cases of MM patients, very heavily pre-treated and all previously transplanted, who received bortezomib associated to dexamethasone for relapse after allogeneic RICT (median time from diagnosis: 68 months, median time from RICT: 41 months). We studied the feasibility, efficacy and safety of bortezomib: two patients achieved a complete remission, one patient a very good partial response, and one had no response. The tolerance was excellent with only one patient who developed a reversible grade 3 hematological toxicity. No GVHD recrudescence was observed. All patients relapsed after completion of bortezomib treatment. This questions about the potential benefit of maintenance therapy with bortezomib. Given the feasibility and the high efficacy of bortezomib combined with dexamethasone in relapsed MM after RICT, our results argue in favour of an immune-modulatory activity of bortezomib. New strategies combining RICT and bortezomib are warranted to increase and maintain an adequate response. Combination RICT and bortezomib regimen are anticipated to further extend survival in relapsed MM.

Haploidentical Hematopoietic Stem Cell Transplantation of Sex-Matched Donor-Recipient Pair Has Survival Advantage: A Single-Center Study of 440 Cases

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 228-228 ◽  
Author(s):  
Tong Wu ◽  
Yan-Li Zhao ◽  
Jing-Bo Wang ◽  
Xing-Yu Cao ◽  
Yu-Ming Yin ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematological Malignancies ◽  
Disease Status ◽  
Hematopoietic Stem ◽  
Male Donor ◽  
Female Donor ◽  
Matched Donor

Abstract Abstract 228 Haploidentical hematopoietic stem cell transplantation (haplo-HSCT) is an important alternative option for patients with hematological malignancies who need urgent transplant but without HLA matched either sibling or unrelated donor (Dao-Pei Lu et al., Blood 2006; 107:3065). Each patient usually has several haploidentical family members who could be selected as a donor. To determine the principal of donor selection among all available related haploidentical candidates, the clinical outcomes of a large series of haplo-HSCT in our hospital are analyzed. From April 2002 to April 2010, consecutive 440 patients with hematological malignancies who underwent haplo-HSCT were included. The median age of patients was 23 (3-59) years old. The diagnosis included AML (39.8%), ALL (35.9%), MDS (3.6%), CML (16.1%), and others (4.6%). Transplants at CR1 or CML-CP1, ≥CR2 or CML-CP2/AP, and advanced disease (refractory/relapsed acute leukemia or CML-BC) were 33.4%, 40.9% and 25.7%. HLA mismatched at 1, 2, 3 loci was 13.2 %, 27.5%, 59.3%, respectively. Transplants in sex-matched donor-recipient pair, female donor to male recipient, and male donor to female recipient were 55.1%, 33.0% and 13.9%. Major clinical characteristics among these three arms were similar. All patients received unmanipulated combined marrow and peripheral blood stem cells for transplant after BUCy2/CyTBI plus ATG conditioning. Fludarabine was substituted for cyclophosphamide in some patients due to impaired organ function. Prophylaxis and treatment of GVHD were reported previously. Steady hematopoietic reconstitution was seen in 98.6% of recipients. The cumulative incidences of grade II to IV acute GVHD and chronic GVHD were 32.6%, 61.3%, respectively. 100-day mortality was 10.5%. With the median follow-up of 32 (3-99) months, 2-year and 5-year overall survival (OS) rates for patients who were in different disease status were 57.9% and 52.9%. Univariate and multivariate analysis all showed that disease status before transplant, CD34+ cell dosage infused and sex-matched or not between donor and recipient but not HLA disparity and age were pivotal impact factors on survival. Two-year OS of transplants in sex-matched donor-recipient pair, female donor to male recipient, male donor to female recipient were 65.5%, 55.3%, 37.6, respectively (p=0.000). No significant differences were found on OS of transplants among haploidentical donors from sibling or parent or offspring or other relatives. In conclusion, our clinical results from a large series of transplants demonstrate that haplo-HSCT in sex-matched donor-recipient pair has survival advantage. Therefore, in haplo-HSCT, sex-matched donor should be the first choice, if not available, then female donor to male recipient could be the second option. Disclosures: No relevant conflicts of interest to declare.

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