scholarly journals Haploidentical Stem Cell Transplantation in Lymphomas—Expectations and Pitfalls

2020 ◽  
Vol 9 (11) ◽  
pp. 3589
Author(s):  
Jacopo Mariotti ◽  
Stefania Bramanti ◽  
Armando Santoro ◽  
Luca Castagna
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Therapeutic Option ◽  
Unrelated Donor ◽  
Disease Relapse ◽  
Haploidentical Stem Cell Transplantation ◽  
Gvhd Prophylaxis ◽  
Life Threatening ◽  
Related Donor

T-cell replete Haploidentical stem cell transplantation (Haplo-SCT) with Post-transplant cyclophosphamide (PT-Cy) is an emerging therapeutic option for patients with advanced relapsed or refractory lymphoma. The feasibility of this platform is supported by several retrospective studies showing a toxicity profile that is improved relative to umbilical cord blood and mismatched unrelated donor (UD) transplant and comparable to matched unrelated donor transplant. In particular, cumulative incidence of chronic graft-versus-host disease (GVHD) is reduced after Haplo-SCT relative to UD and matched related donor (MRD) transplant thanks to PT-Cy employed as GVHD prophylaxis. This achievement, together with a similar incidence of acute GVHD and disease relapse, results in a promising advantage of Haplo-SCT in terms of relapse-free/GVHD free survival. Unmet needs of the Haplo-SCT platform are represented by the persistence of a not negligible rate of non-relapse mortality, especially due to infections and disease relapse. Future efforts are warranted in order to reduce life-threatening infections and to employ Halo-SCT with PT-Cy as a platform to build new immunotherapeutic strategies.

scholarly journals Haploidentical Donors: Can Faster Transplantation Be Life-Saving for Patients with Advanced Disease?

2016 ◽  
Vol 135 (4) ◽  
pp. 211-216 ◽  
Author(s):  
Alina Tanase ◽  
Ciprian Tomuleasa ◽  
Alexandra Marculescu ◽  
Alexandru Bardas ◽  
Anca Colita ◽  
...  
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Advanced Disease ◽  
Therapeutic Option ◽  
Unrelated Donor ◽  
Haploidentical Stem Cell Transplantation ◽  
Life Saving ◽  
Donor Cells ◽  
Related Mortality

Haploidentical stem cell transplantation is a therapeutic option for patients without an HLA-matched donor. It is increasingly being used worldwide due to the application of posttransplantation cyclophosphamide and is associated with lower incidence of graft-versus-host disease and treatment-related mortality. Haploidentical donors are generally available for most patients and stem cells can be rapidly obtained. Delays in transplantation while waiting for unrelated donor cells can be potentially problematic for patients with advanced disease at risk for progression; thus, the use of haploidentical donors, especially in this setting, can be life-saving. Here we reviewed the literature on haploidentical stem cell transplantation performed with posttransplantation cyclophosphamide.

scholarly journals Haploidentical Stem Cell Transplantation: A Gateway to Infrequent Availability of HLA-Matched Related Donors

2018 ◽  
Vol 2018 ◽  
pp. 1-4 ◽  
Author(s):  
Hafiz Muhammad Aslam ◽  
Shumaila Muhammad Iqbal ◽  
Hira Shaikh ◽  
Faizan A. Faizee ◽  
Ambreen A. Merchant ◽  
...  
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Allogeneic Transplantation ◽  
Graft Versus Host ◽  
Haploidentical Transplantation ◽  
Haploidentical Stem Cell Transplantation ◽  
Engraftment Failure ◽  
Desensitization Protocol ◽  
Related Donor

Haploidentical stem cell transplantation provides a plausible alternative for the patients when a fully matched donor is unavailable. Historically, the decision of considering haploidentical transplant has remained elusive; however, with the recent advances, the consideration of haploidentical grafts as a treatment option has become more apparent for both allografting for diseases and engraftment failure. We are reporting here an anecdotal case of a successful haploidentical engraftment in a patient with the prior graft failure of an HLA-matched related donor. Since the patient was severely alloimmunized, desensitization protocol was utilized before the haploidentical transplant, and the patient after 8 months of her second allogeneic transplantation is doing great with successful engraftment, no relapse, and no graft-versus-host disease (GVHD). Numerous reports pertinent to haploidentical graft have shown favorable outcomes in the graft placement, a decline in the rate of GVHD, and an improvement in the morbidity and mortality in affected individuals. Based on the current reports, haploidentical transplantation might be more feasible and has meaningful implications in the situations where matched donors are infrequent.

Immune Reconstitution and Immune Correlates of Clinical Outcome IN Acute LEUKEMIA PATIENTS Treated with Haploidentical STEM CELL TRANSPLANTATION.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 46-46
Author(s):  
Alessandra Forcina ◽  
Maddalena Noviello ◽  
Veronica Valtolina ◽  
Attilio Bondanza ◽  
Daniela Clerici ◽  
...  
Keyword(s):  
T Cells ◽  
Stem Cell ◽  
T Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Immune Reconstitution ◽  
Patient Specific ◽  
Haploidentical Stem Cell Transplantation ◽  
Gvhd Prophylaxis ◽  
Kinetics Of

Abstract Abstract 46 The broader application of haploidentical stem cell transplantation (haplo-HCT), is limited by the delayed immune reconstitution (IR) secondary to the procedures for GvHD prophylaxis. This ultimately results in a high-rate of infectious complications and non-relapse mortality. We dynamically analyzed immunoreconstitution (IR) in patients undergoing haplo-HCT for acute leukemias enrolled in two different phase I-II clinical trials aimed at improving IR. In the first trial (TK007), 28 patients (out of 50 enrolled) received suicide-gene transduced donor T cells at day +42 after a T-cell depleted graft, in the absence of post-transplant immunosuppression. In the second trial (TrRaMM), 40 patients received an unmanipulated graft and a rapamycin-based GvHD prophylaxis. T-cell immune reconstitution was more rapid in TrRaMM than in TK007 patients, with a threshold of CD3 cells>100/μl reached at days +30 and +90, respectively. In both trials IR was mainly composed of Th1/Tc1 lymphocytes with an inverted CD4/CD8 ratio. While in TrRaMM patients we observed an early expansion of naïve and central memory T cells, producing high amounts of IL-2, in TK patients IR was mainly composed of activated effectors. Furthermore, in TrRaMM patients we detected high levels of CD4+CD25+CD127- T regulatory cells (up to 15% of circulating T lymphocytes) that persisted after rapamycin withdrawal, and was significantly superior to that observed in TK patients and in healthy controls. Interestingly, in contrast to the different kinetics of T-cell reconstitution, no differences were observed in time required to gain protective levels of CMV-specific T cells, as shown by ψIFN ELISPOT analysis. Protective frequencies of CMV-specific lymphocytes were observed 3 months after HCT in both groups, a time-point that in TrRaMM patients corresponds to the average time of rapamycin withdrawal. In both trials the number of circulating CMV-specific T cells was inversely correlated to the number and severity of subsequent CMV reactivations and days of antiviral therapy. GvHD was diagnosed in 16 TrRaMM patients (40%) and in 10 TK patients (35% of patients who received TK cells). Severity of GvHD was different in the two cohort of patients with 5 TrRaMM patients (12,5%) and only 2 TK patients (7%) with grade III-IV GvHD. Of interest, in the TrRaMM group CMV-specific immunity was significantly hampered by the immunosuppressive treatment required to treat GvHD. On the contrary, in the TK group, the administration of ganciclovir was able to activate the suicide machinery and control GvHD without impairing viral-specific T-cell immunocompetence. These results matched with the kinetics of CMV reactivations. We observed that while in TrRaMM patients 80% of viral reactivations occurred after the immunosuppressive therapy, in TK patients no significant differences could be assessed before and after therapy. IFN-ψ ELISPOT might thus be a relevant and predictive test to guide patient-specific clinical monitoring and antiviral treatment. Overall, these results show that early immune reconstitution can be promoted in haplo-HCT by different strategies associated with a wide range of alloreactive potential. The risks and benefits associated with alloreactivity should guide the therapeutic choice tuned on patient disease status and co-morbidities. Disclosures: Bordignon: Molmed Spa: Employment.

Comparative Analysis of Outcomes of Allogeneic Peripheral Blood Stem Cell Transplantation From Related and Unrelated Donors for Adults with Hematologic Malignancies

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 2302-2302
Author(s):  
Chunji Gao ◽  
Xiaohong Li ◽  
Honghua Li ◽  
Wenrong Huang ◽  
Xiaoxiong Wu ◽  
...  
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Peripheral Blood ◽  
Peripheral Blood Stem Cell ◽  
Hematologic Malignancies ◽  
Blood Stem Cell ◽  
Unrelated Donor ◽  
Gvhd Prophylaxis ◽  
Blood Stem Cell Transplantation

Abstract Abstract 2302 Although allogeneic peripheral blood stem cell transplantation from a matched related donor (RD-PBSCT) presents the best curable opportunity for many patients with hematologic malignancies, only about one third of individuals have HLA matched sibling donors. Fortunately, from unrelated donor peripheral blood stem cell transplantation has been acceptable and expanded recently. In order to evaluate the effect of allogeneic peripheral blood stem cell transplantation from unrelated donor (URD-PBSCT), we compare results of URD-PBSCT and RD-PBSCT that were done for 172 consecutive adult patients with hematologic maligancies from Jan 2002 to Dec 2008 at a single-center. Among these patients, 62 cases underwent URD-PBSCT and 110 cases underwent RD-PBSCT. Myeloablative conditioning was adopted for all patients. In graft versus host disease (GVHD) prophylaxis, 49 URD-PBSCT recipients received CSA, MTX, MMF and ATG (URD-ATG group), 13 recipients were given simulect more in base of URD-ATG group (URD-ATG+CD25 group) while RD-PBSCT recipients (RD group) received CSA, MTX and MMF. Engraftment was achieved in 98.4% of URD-PBSCT patients and 98.2% of RD-PBSCT patients (100% for patients surviving beyond 28 days after transplant). The cumulative incidence of acute GVHD (grade II-IV) was 15.4%, 36.7% and 29.1% respectively in the URD-ATG+CD25, URD-ATG and RD groups (P = 0.275). The cumulative incidence of chronic GVHD was 0%, 45.6%, 39.6% respectively and significant lower in URD-ATG+CD25 group than the other two groups (P = 0.0134). Relapse incidence was 53.8%, 12.2%, 14.5% respectively and significant higher in URD-ATG+CD25 group than the other s (P = 0.0059), while there was no different between the URD-ATG and RD groups (P = 0.610). Estimated overall survival (OS) at 5 years was 30.8%, 69.4% and 67.3% respectively and no significant difference between URD-ATG group and RD group (p=0.898). Adverse prognosis factors for relapse and OS included transplant not in remission and GVHD prophylaxis with simulect. Our results indicate PBSCT from unrelated donor may be considered comparable to those from related donor. Disclosures: No relevant conflicts of interest to declare.

Poor Performance Status, Chemorefractory Disease At Transplantation, and Umbilical Cord Blood As Donor Source Were Adverse Prognostic Factors for Overall Survival After Allogeneic Stem Cell Transplantation for Follicular Lymphoma: Retrospective Study of the Japan Society of Hematopoietic Stem Cell Transplantation (JSHCT) Lymphoma Working Group

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 3093-3093
Author(s):  
Koji Izutsu ◽  
Ritsuro Suzuki ◽  
Shinichi Kako ◽  
Rika Sakai ◽  
Takehiko Mori ◽  
...  
Keyword(s):  
Stem Cell ◽  
Prognostic Factors ◽  
Retrospective Study ◽  
Stem Cell Transplantation ◽  
Cord Blood ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Unrelated Donor ◽  
Hematopoietic Stem ◽  
Related Donor

Abstract Abstract 3093 Allogeneic hematopoietic stem cell transplantation (allo SCT) is potentially curative treatment for relapsed follicular lymphoma (FL) that remains non-curative disease even with modern immunochemotherapy. Number of potential candidate of allo SCT for FL has been increasing with the development of reduced intensity conditioning (RIC) regimens and increased donor availability due to use of hematopoietic stem cell from unrelated donor including cord blood (CB). However, short-term and long-term transplantation-related complications are still major obstacles in applying this treatment. Recently, EBMT and CIBMTR reported a prognostic score of allo SCT for FL (Ann Oncol 2011;22 :Suppl 4, iv94). However, this score awaits validation. To elucidate prognostic factors for OS after allo SCT for FL, we conducted a retrospective study using the national registry data of the Japan Society of Hematopoietic Cell Transplantation (JSHCT). In total, 472 cases of allo SCT for FL performed from 2000 to 2008 were identified. 220 (46.6%) were male and the median age at allo SCT was 50 yo (range: 27–75). Forty six (9.7%) patients were 60 yo or older. Eighty two (17.3%) patients had previous history of autologous transplantation (ASCT). Stem cell source was BM or PB from related donor in 215 (45.5%), unrelated donor BM in 180 (38.1%), and unrelated donor umbilical cord blood (CB) in 77 (16.3%). Conditioning regimen was myeloablative (MAC) in 20.7% and reduced intensity (RIC) in 79.3%, respectively. Patients undergoing MAC were younger than those with RIC (45 vs 50 yo, P<0.01). T-cell depletion with ATG or ALG was performed in 7.5%. ECOG performance status (PS) at allo SCT was ≥ 2 in 10.7%. 71.5% had chemosensitive disease at allo SCT and disease status was CR in 38.9%. With a median follow up of 47.3 mo (range: 0.9–122) among survivors, overall survival rate at 4years after allo SCT (4y OS) was 52.6%. Patients who had CB transplantation had worse OS than those transplanted from related donor or unrelated BM (30.0%, 58.5%, 54.6%, P =1.35e-6, logrank). Additionally, in univariate analysis, PS ≥2, chemoresistant disease at allo SCT, older age, male sex were adverse prognostic factors for OS, while previous history of ASCT, time from diagnosis to allo SCT (<3y vs ≥3y), disease status at allo SCT (CR vs non CR), conditioning regimen (MAC vs RIC), and year of allo SCT (2000–2 vs 2003–5 vs 2006–8) were not. 4y OS of patients undergoing MAC and RIC were 46.2% and 54.0% (P =0.28), respectively. In multivariate analysis with proportional hazard modeling, PS ≥2 (HR 3.2, P =0.00015), chemorefractory disease (HR 2.7, P =0.039), and use of CB (HR 3.8, P =0.039) were independent adverse predictors of OS. Although risk factors that are incorporated in the proposed EBMT/CIBMTR risk score other than PS were not predictive of OS in the present analysis, we applied this score to the population of the present study. In the entire population, 4y OS were 61.0% and 38.5% (P =9.5e-08) in patients with the score <3 (n=294) and ≥3 (n=77), respectively. This score was predictive of OS in patients who underwent transplantation from related donor (65.4% vs 36.7%, P =3.77e-08) and from unrelated BM (59.0% vs 41.7%, P =0.0217). In conclusion, this retrospective study of JSHCT registry data showed that allo SCT for FL is a reasonable option for patients with relapsed FL when suitable donor is available. Poor PS, chemorefractory disease, and CB as donor source were adverse prognostic factors for OS. Disclosures: No relevant conflicts of interest to declare.

Clinical Outcomes of Haploidentical Donor Compared with Unrelated and HLA-Matched Related Donor Hematopoietic Stem Cell Transplantation for Hematologic Malignancies

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 4159-4159
Author(s):  
Yi Luo ◽  
Yamin Tan ◽  
Xiaoyu Lai ◽  
Weiyan Zheng ◽  
Jimin Shi ◽  
...  
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Conditioning Regimen ◽  
Hematologic Malignancies ◽  
Unrelated Donor ◽  
Hematopoietic Stem ◽  
Haploidentical Transplantation ◽  
Related Donor ◽  
Matched Donor

Abstract Abstract 4159 Introduction: Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is a potentially curative therapeutic option for hematologic malignancies. In clinical trials, a HLA-matched donor can only be found for about 50% to 60% of patients referred for HSCT which greatly limit the application of this important procedure. Haploidentical HSCT would increase the availability of donors for nearly 100% of patients. However, haploidentical HSCT may be associated with high risks of complications, such as graft rejection, severe GVHD and infection etc. Although great progress have been achieved in haploidentical HSCT based on advanced technologies and novel drugs, no study has simultaneously compared the outcomes of haploidentical, unrelated and HLA-matched related donor HSCT. Materials and Methods: In this study, 225 patients with hematologic malignancies received allo-HSCT from diffent donor sources in our center (69 with haploidentical donors, 62 with HLA-matched related donors and 94 with unrelated donors). The clinical outcomes of haploidentical HSCT cohort, unrelated donor HSCT cohort and HLA-matched related donor HSCT cohort were compared. In HLA-matched sibling and unrelated donor transplantation cohorts, patients received a same conditioning regimen consisting of intravenous busulfan 3.2 mg/kg/d on days –7 to –4, intravenous cyclophosphamide 60 mg/kg/d on days –3 to –2, and 250 mg/m2 of Me-CCNU orally on day -1. For haploidentical HSCT, conditioning regimen consisted of Ara-C (4 g/m2/d) on day -10 and -9, Bu (9.6mg/kg) on day -8, -7 and -6, Cy (1.8 g/m2/d) on day -5 and -4, Me-CCNU (250 mg/kg) on day -3, and ATG (2.5 mg/kg/d) on day -5 to -2. GVHD prophylaxis consisted of cyclosporine A (CsA), mycophenolate mofetil (MMF), and short-term methotrexate while ATG (1.5 mg/kg/d) for three or four days, were added in HLA-matched or mismatched unrelated HSCT. Results: The patients receiving haploidentical HSCT experienced grades III-IV aGVHD more frequently than those receiving unrelated donor HSCT and related matched donor HSCT (24.4% vs 12% vs 2.6% respectively, p<0.05). However the incidence of cGVHD was comparable (17.8% in the haploidentical cohort vs 37.3% in the unrelated donor cohort vs 25.6% in the related matched donor cohort, p>0.05). The transplantation-related mortality (TRM) at d100 were 17.4%, 8.5% and 1.6% in the haploidentical, unrelated and related matched transplantation cohorts respectively (p<0.05). The 3-year relapse incidence were 10.1%, 15.9%, 17.7% in the haploidentical, unrelated and relate matched transplantation cohorts respectively (p>0.05). The 3- year overall survival (OS) was comparable in three cohorts (64.2±6.4% in the haploidentical cohort vs 67.5±5.3% in the unrelated donor cohort vs 77.5±5.8% in the related matched cohort, p>0.05). Conclusion: Although a higher incidence of aGVHD and TRM was observed in the haploidentical transplantation cohort, the incidences of cGVHD and relapse were comparable in the haploidentical, unrelated and related matched transplantation cohorts. Ultimately the patients receiving haploidentical transplantation achieved comparable OS with those receiving unrelated donor transplantation. An HLA-matched HSCT is commonly the preferred transplantation and donors from HLA-matched related siblings are usually the first choice. Haploidentical stem cell transplantation is relatively safe and efficient for patients who do not have HLA matched donors. Disclosures: No relevant conflicts of interest to declare.

Outcomes of Mismatched Related Allogeneic Stem Cell Transplantation for Chronic Lymphocytic Leukemia: A Retrospective Study on Behalf of the Chronic Malignancies Working Party of the EBMT

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 3504-3504
Author(s):  
Gwendolyn van Gorkom ◽  
Michel van Gelder ◽  
Dimitris Ziagkos ◽  
Henric-Jan Blok ◽  
Maria Teresa van Lint ◽  
...  
Keyword(s):  
Stem Cell ◽  
High Risk ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Research Funding ◽  
Lymphocytic Leukemia ◽  
Gvhd Prophylaxis ◽  
Related Donor ◽  
Support Research

Abstract Introduction: Allogeneic hematopoietic stem cell transplantation (HCT) is a treatment for CLL that can give long disease control. Even with the availability of kinase and BCL2 inhibitors, HCT is still performed in fit patients (pts) with high-risk CLL. Almost exclusively, outcomes on matched related and unrelated donor transplantations in CLL have been published. Recently, mismatched related donors are gaining interest because of the better outcome of haploidentical HCT with post-transplantation cyclophosphamide (PTCY). Methods: All pts with CLL who received a first allogeneic HCT with a mismatched related donor and whose data were available in the EBMT registry were analyzed. Median values and ranges are reported for continuous variables and percentages for categorical variables. The probabilities of overall survival (OS) and progression-free survival (PFS) were calculated using the Kaplan-Meier method and the log-rank test for univariate comparisons. Relapse/progression and nonrelapse mortality (NRM) were analyzed together in a competing risk framework. Statistical analyses were performed using SPSS and R. Results: One-hundred-seventeen pts with CLL (74% males) underwent a mismatched related donor transplantation between 1984 and 2015 (1984-1999: 10, 2000-2004: 18, 2005-2009: 23, 2010-2016: 66). Median follow-up after HCT was 8 months (range 0-187 months). Median age at transplantation was 54 years (yrs) (range 27-71 yrs). Median time from diagnosis to HCT was 67 months (range 4-207 months). Eighteen pts (17%) had previously undergone autologous stem cell transplantation (ASCT). Disease status at HCT was CR in 16% of pts, PR in 39% and SD/PD in 45%. The Karnofsky score was known for 98 pts; 96% had a score of 70% or more at the time of HCT. Fifty-eight percent of pts received reduced-intensity conditioning, 42% myeloablative conditioning. Peripheral blood stem cells were used in 68% of pts, bone marrow in 32%. The HCT was sex matched in 41% of recipient-donor pairs. The relationship of the donor to the patient was known for 34 pts; in 53% the donor was a child, in 38% a sibling and in 6% a parent. Forty pts (38%) received PTCY as GVHD prophylaxis. In the other 77 pts various methods of T-cell depletion (TCD) were used, but not all methods were specified. At least 56% of those pts had in vivo TCD. For the whole cohort of pts OS at 2 and 5 yrs was 46% and 37%, respectively. PFS at 2 and 5 yrs was 38% and 30%, respectively. The use of PTCY did not have a significant impact on OS (49% vs. 42% at 2 yrs, 44% vs. 33% at 5 yrs, p=0.35) and PFS (45% vs. 31% at 2 yrs, 40% vs. 22% at 5 yrs, p=0.15). CI of NRM in the whole group at 2 and 5 yrs were 41% and 45%, respectively. CI of relapse at 2 and 5 yrs were 21% and 25%, respectively. The CI of NRM and relapse at 2 and 5 yrs were not statistically different in pts who received PTCY compared to other types of TCD (NRM: 38% vs. 45% at 2 yrs, 43% vs. 49% at 5 yrs, p=0.45; relapse: 17% vs. 25% at 2 yrs, 17% vs. 29% at 5 yrs, p=0.33). For the whole cohort, the incidence of acute graft-versus-host disease (aGVHD) at 100 days was 34% for grade II-IV and 16% for grade III-IV with a median time of onset of 23 days (range 4-57 days). Conclusions: Mismatched related donor HCT resulted in a 5-year PFS in 30% of the pts. This result seems only slightly inferior to matched donor transplant (5 yrs PFS 37%1). NRM was higher than expected in this cohort, but comparable to other studies on haploSCT with in vivo T-cell depleted grafts. In conclusion, a mismatched related donor HCT may be considered for high-risk chemoimmunotherapy-refractory or 17p deleted/TP53 mutated CLL pts without options for kinase and BCL2 inhibitor therapy. More data are needed to assess the value of PTCY for GVHD prophylaxis in this specific context. References: 1. Schetelig J, de Wreede L, Moreno C, et al. Risk factors for adverse outcome in patients with Chronic Lymphocytic Leukemia (CLL) undergoing Allogeneic Hematopoietic Cell transplantation (alloSCT): a Retrospective EBMT Analysis. Abstract WP024, EBMT meeting 2015. Figure 1 Figure 1. Disclosures Ciceri: MolMed SpA: Consultancy. Foà:Ariad: Speakers Bureau; Pfizer: Speakers Bureau; BMS: Consultancy; Celgene: Consultancy, Speakers Bureau; Amgen: Consultancy, Speakers Bureau; Gilead: Consultancy, Speakers Bureau; Janssen-Cilag: Consultancy, Speakers Bureau; Genetech: Consultancy; Roche: Consultancy, Speakers Bureau. Hallek:Mundipharma: Consultancy, Honoraria, Other: travel support, Research Funding, Speakers Bureau; F. Hoffmann-LaRoche: Consultancy, Honoraria, Other: travel support, Research Funding, Speakers Bureau; AbbVie: Consultancy, Honoraria, Other: travel support, Research Funding, Speakers Bureau; Janssen-Cilag: Consultancy, Honoraria, Other: travel support, Research Funding, Speakers Bureau; Celgene: Consultancy, Honoraria, Other: travel support, Research Funding, Speakers Bureau; Gilead: Consultancy, Honoraria, Other: travel support, Research Funding, Speakers Bureau; Amgen: Consultancy, Honoraria, Other: travel support, Research Funding, Speakers Bureau. Schetelig:Sanofi: Honoraria. Kröger:Sanofi: Honoraria, Research Funding; Neovii: Honoraria, Research Funding; Riemser: Honoraria, Research Funding; Novartis: Honoraria, Research Funding.

scholarly journals Haploidentical Stem Cell Transplantation in Patients with Myelodysplastic Syndrome: Case Report First Experience

2021 ◽  
Vol 9 (C) ◽  
pp. 250-253
Author(s):  
Aleksandra Pivkova-Veljanovska ◽  
Irina Panovska-Stavridis ◽  
Lazar Chadievski ◽  
Sanja Trajkova ◽  
Marija Popova-Labachevska ◽  
...  
Keyword(s):  
Stem Cell ◽  
High Risk ◽  
Complete Remission ◽  
Stem Cell Transplantation ◽  
Myelodysplastic Syndrome ◽  
Cell Transplantation ◽  
High Risk Patient ◽  
Unrelated Donor ◽  
Case Presentation ◽  
Haploidentical Stem Cell Transplantation

  BACKGROUND: Allogeneic stem cell transplantation (ASCT) is a potentially curative therapeutic approach in patients with intermediate and high-risk myelodysplastic syndrome (MDS). If a family sibling or unrelated donor is not available mismatched donors are viable option for young patients with no comorbidities. The aim of this case presentation was to evaluate our first experience with haploidentical transplantation for this indication. CASE PRESENTATION: We present a case of 50 years male patient with myelodysplastic syndrome (MDS) diagnosed at University Clinic for hematology, Skopje, North Macedonia. Patient was scored in IPSS -R as high risk patient. He was referred for HLA DNA typing of family siblings and since he didn’t have identical sibling and unrelated donor, he was referred to continue treatment with haploidentical stem cell transplantation. He received Flu Bu conditioning and PTCY, cyclosporine and MMF for GVHD prophylaxis. Peripheral blood stem cells (PBSC) from his mismatched brother were infused in the amount of CD34=5.8x106/kg. He experienced prolonged engraftment, severe infective bacterial infections and CMV reactivation with clinical manifestation of CMV colitis. He was successfully treated with antiviral drug and completely resolved. His bone marrow analysis showed complete remission and chimerism evaluation revealed high donor engraftment. Patient is now +34 months post transplant in complete remission. CONCLUSION: The use of a mismatched donor increases the risk of NRM, but there is also evidence to suggest that an haploidentical donor is a valid choice, as general outcome appears to be at least similar to MUD.

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