157 results on '"G. Helbig"'
Search Results
2. Interferon α induces a good molecular response in a patient with chronic eosinophilic leukemia (CEL) carrying the JAK2V617F point mutation
- Author
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G. Helbig, B. Stella-Holowiecka, M. Majewski, M. Lewandowska, and J. Holowiecki
- Subjects
Diseases of the blood and blood-forming organs ,RC633-647.5 - Abstract
The JAK2 V617F point mutation is very rare in hypereosinophilic syndome and/or chronic eosinophilic leukemia. Here we report on a patient with chronic eosinophilic leukemia and detectable JAK2 mutant clone, who achieved a good molecular response to interferon α-2a after 4 months of treatment. The molecular response correlated with only moderate haematological improvement
- Published
- 2007
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3. PB1916 MIRNAS AND ANGIOGENIC FACTORS IN PLASMA BEFORE AND AFTER TKI TREATMENT IN CML PATIENTS
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K. Lewandowski, Karolina Łuczkowska, Zofia Litwińska, M. Gniot, Ewa Pius-Sadowska, G. Helbig, Anna Sobuś, Bogusław Machaliński, Dorota Rogińska, and Edyta Paczkowska
- Subjects
business.industry ,microRNA ,Cancer research ,Medicine ,Hematology ,business - Published
- 2019
4. Epstein-Barr Virus (EBV)-Induced Hemophagocytic Lymphohistiocytosis in the Course of Metamphetamine Abuse
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G, Helbig, primary and E, Rzenno, additional
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- 2015
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5. Postępowanie u chorego ze wznową ostrej białaczki promielocytowej
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G. Helbig
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Oncology ,Hematology - Published
- 2015
6. A laboratory comparison of the efficacy of battery-operated, non-rechargeable power toothbrushes
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G M, Driesen, P R, Warren, U, Bielfeldt, and G, Helbig
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Toothbrushing ,Time Factors ,Rotation ,Statistics as Topic ,Dental Plaque ,Gingiva ,Equipment Design ,Robotics ,Tooth, Artificial ,Models, Dental ,Statistics, Nonparametric ,Electric Power Supplies ,Image Processing, Computer-Assisted ,Humans ,Stress, Mechanical - Abstract
To evaluate the cleaning efficacy of three battery-operated, non-rechargeable, oscillating/rotating power toothbrushes, using a robot system to simulate normal clinical toothbrush use.The study compared the cleaning efficacy of the new Braun Oral-B D4/EB4 with the Actibrush and the Dr. Best Powerclean in two independent experiments. Plaque substitute was applied to the artificial teeth of typodonts, which were cleaned by the robot system for a total of 2 minutes at a brushing force of 1.95 N. The remaining plaque substitute on buccal + lingual/palatal and occlusal surfaces, as well as gingival margin and interproximal sites, was measured using a computerized vision system.The new D4/EB4 was found to remove more plaque substitute than the Actibrush at all sites, and for all surfaces, lingual surfaces and occlusal surfaces the difference in favor of the D4/EB4 was statistically significant (P0.05). In comparison with the Powerclean toothbrush, the D4/EB4 was significantly more effective at all sites (P0.001). These results indicate that not all battery-operated oscillating/rotating power toothbrushes have equal efficacy with respect to plaque removal and that, in this series of laboratory experiments, the Braun Oral-B D4/EB4 was more effective than the Actibrush and the Dr. Best Powerclean.
- Published
- 2002
7. The proportion of CD3− CD4+ T-cell population remained unaffected after corticosteroids treatment for lymphocytic variant hypereosinophilic syndrome (L-HES)
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J. Dziaczkowska-Suszek, G. Helbig, Ryszard Wichary, K. Wozniczka, M. Rodzaj, M. Razny, and Slawomira Kyrcz-Krzemien
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education.field_of_study ,biology ,Cd4 t cell ,medicine.diagnostic_test ,Hypereosinophilic syndrome ,business.industry ,CD3 ,Immunology ,Population ,General Medicine ,medicine.disease ,Flow cytometry ,Prednisone ,medicine ,biology.protein ,education ,business ,medicine.drug - Published
- 2010
8. Das Bundesministerium für Bildung und Forschung
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G. Helbig
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General Chemical Engineering ,General Chemistry ,Industrial and Manufacturing Engineering - Published
- 2004
9. EFFECTS OF OXYGEN ON A FIXED VOLUME HEMORRHAGE MODEL IN RODENTS
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G. Helbig, J. Davis, H. Ho, and R. A. Gunther
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chemistry ,Volume (thermodynamics) ,Emergency Medicine ,chemistry.chemical_element ,Critical Care and Intensive Care Medicine ,Oxygen ,Biomedical engineering - Published
- 2001
10. Doppler sonographic examination of reactive hyperemia in the diagnosis of peripheral vascular disease
- Author
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W. Stork, G. Helbig, and K. H. Vogelberg
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Male ,medicine.medical_specialty ,Pathology ,Arterial Occlusive Diseases ,Hyperemia ,Internal medicine ,Drug Discovery ,Occlusion ,medicine ,Humans ,Ultrasonics ,Reactive hyperemia ,Genetics (clinical) ,Ultrasonography ,Leg ,Vascular disease ,business.industry ,General Medicine ,Blood flow ,Middle Aged ,medicine.disease ,Peripheral ,Intensity (physics) ,Stenosis ,Blood pressure ,Cardiology ,Molecular Medicine ,Female ,business - Abstract
Fifty-four patients with angiographically confirmed peripheral vascular disease (PVD) were examined in order to find out whether the occlusive form of this disease can be better diagnosed by measuring the reappearance time and mean velocity of the blood flow during reactive hyperemia than by determining the peripheral systolic blood pressure, using Doppler ultrasound for both measurements. It was shown that the Doppler pressure was only reliable for a screening diagnosis of PVD. However, using the reappearance time of reactive hyperemia, it was possible to distinguish specific localization types of sclerosis; while reactive hyperemia already reached its half maximum in controls in 4.6 s this occurred in the stenosis type of PVD after 6.9 s, in the upper leg occlusion type after 21.6 s, in the lower leg occlusion type after 46.6 s, and in the multilevel disease after 70.1 s. The delay in the half-maximum reappearance time was significantly different, not only in comparison with controls (P
- Published
- 1988
11. Deutsche Grammatik
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G. Helbig and J. Buscha
- Published
- 1981
12. [Improvement of the solubility of problem drugs. 18. Iomeglamic acid-PVA effervescent powder]
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F, Fahr, H, Kala, G, Fries, and G, Helbig
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Solubility ,Iodobenzenes ,Chemistry, Pharmaceutical ,Polyvinyl Alcohol ,Powders - Published
- 1988
13. Sonographic examination of gastric motility in diabetics with autonomic neuropathy
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K H, Vogelberg, W, Rathmann, and G, Helbig
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Blood Glucose ,Male ,Eating ,Diabetes Mellitus, Type 1 ,Autonomic Nervous System Diseases ,Diabetic Neuropathies ,Humans ,Female ,Fasting ,Middle Aged ,Gastrointestinal Motility ,Ultrasonography - Abstract
The frequency, intensity and velocity of antral contractions were measured by ultrasonography in 32 patients with insulin-dependent diabetes mellitus and in 12 controls before and up to 60 min after a test breakfast. The examination showed that motility was lower in the diabetics with autonomic neuropathy than in those without and in the non-diabetic controls. The frequency of contractions was determined in the 3 groups as follows: 3.6 +/- 2.0 vs. 4.8 +/- 1.7 vs. 4.8 +/- 1.6 contractions per 2 min (p less than 0.0025); the intensity of contractions was 30.9 +/- 8.2 vs. 41.4 +/- 5.2 vs. 57.5 +/- 8.8 delta % of antral area (p less than 0.025, resp. p less than 0.0005); the velocity of contractions was 4.8 +/- 1.5 vs. 8.2 +/- 1.2 vs. 9.95 + 2.8 delta % of antral area/sec (p less than 0.0125 resp. p less than 0.005). There was a significant difference in the intensity and velocity of contractions between the patients without autonomic neuropathy and the non-diabetic controls (p less than 0.0025 resp. p less than 0.025). 10 min after the test breakfast the motility indices reached a maximum and then decreased continuously towards the end of the test period (p less than 0.01). 20 min after the test meal this decrease was significantly faster in the patients with autonomic neuropathy than in those without and in controls. In all the diabetics the velocity was positively correlated to the coefficient of the variation from beat to beat of the heart, and also positively correlated to the increase of blood glucose concentrations (p less than 0.05).(ABSTRACT TRUNCATED AT 250 WORDS)
- Published
- 1987
14. Vereinfachte gasvolumetrische Gehaltsbestimmung von Diazodinitrophenol
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G. Helbig and F. Rungeâ€Eschen
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General Chemical Engineering ,General Chemistry - Abstract
Diazodinitrophenol wird durch Reduktion der Diazonium-Gruppe zu molekularem Stickstoff in einer Sandmeyer-Reaktion mit CuCl oder durch Umsetzung mit TiCl3 quantitativ bestimmt. Da diese Reaktionen selektiv verlaufen, storen nitrogruppenhaltige Beimengungen nicht. Zur vereinfachten routinemasigen Durchfuhrung wird ein modifizierter Schulze-Tiemann-Apparat verwendet.
- Published
- 1982
15. Prognostic factors impacting post-transplant outcomes in adult T-cell acute lymphoblastic leukemia: a registry-based study by the EBMT acute leukemia working party.
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El Cheikh J, Ngoya M, Galimard JE, Reményi P, Kulagin A, Aljurf M, Mousavi A, Wu D, Ozcelik T, Salmenniemi U, Castilla-Llorente C, Socie G, Helbig G, Schroeder T, Sakellari I, Rambaldi A, Burt R, Busca A, Balsat M, Stelljes M, Brissot E, Giebel S, Peric Z, Nagler A, Bazarbachi A, Ciceri F, and Mohty M
- Subjects
- Humans, Adult, Male, Female, Middle Aged, Adolescent, Young Adult, Aged, Prognosis, Precursor T-Cell Lymphoblastic Leukemia-Lymphoma therapy, Precursor T-Cell Lymphoblastic Leukemia-Lymphoma mortality, Transplantation Conditioning methods, Survival Rate, Registries, Hematopoietic Stem Cell Transplantation methods
- Abstract
T-cell acute lymphoblastic leukemia (T-ALL) predominantly affects individuals in late childhood and young adulthood. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is a curative modality particularly in the setting of poor risk genetics and/or persistent minimal residual disease. Limited studies have directly explored the impact of patient- and transplant-related factors on post-transplant outcomes in T-ALL. Using a large dataset from the European Society for Blood and Marrow Transplantation registry, we identified 1907 adult T-ALL patients (70% male) who underwent their first allo-HSCT in first complete remission (CR1) from matched sibling donors (MSD; 45%), unrelated donors (UD; 43%) or haploidentical donors (12%) between 2010 and 2021. The median age at transplant was 33.4 years (18.1-75). The median follow up was 2.9 years. Most patients underwent total body irradiation (TBI)-based myeloablative conditioning (69%). The 2-year overall survival (OS) was 69.4%, and leukemia -free survival (LFS) was 62.1%. In multivariate analysis, advanced age at transplant negatively affected LFS (for each 10-year increment, HR = 1.11, p = 0.004), GVHD-free, relapse-free survival (GRFS) (HR = 1.06, p = 0.04), OS (HR = 1.12, p = 0.002), and non-relapse mortality (NRM) (HR = 1.23, p < 0.001). More recent years of allo-HSCT were associated with improved GFRS (For each 3-year increment, HR = 0.89, p < 0.001), OS (HR = 0.9, p = 0.02), and decreased NRM (HR = 0.82, p = 0.008). TBI improved LFS. (HR = 0.79, p = 0.02), GRFS (HR = 0.83, p = 0.04), and relapse incidence (RI) (HR = 0.65, p < 0.001). Female-to-male transplant negatively affected GRFS (HR = 1.21, p = 0.02) and OS (HR = 1.23, p = 0.048). In vivo T-cell depletion significantly improved GFRS (HR = 0.74, p < 0.001). This large study identified prognostic factors, such as age at transplant conditioning regimen, in influencing post-transplant in adult T-ALL patients undergoing allo-HSCT. Importantly, a significant improvement over time was noted. These findings hold great promise for new adapted treatment strategies and can serve as a benchmark for future studies in that setting., (© 2024. The Author(s), under exclusive licence to Springer Nature Limited.)
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- 2024
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16. Autologous stem cell transplantation in T-cell/histiocyte-rich large B-cell lymphoma: EBMT Lymphoma Working Party study.
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Renders S, Ngoya M, Finel H, Rubio MT, Townsend WM, Schroers R, Novak U, Schaap N, Aljurf M, Helbig G, Collin M, Kobbe G, Anne H, Pérez-Simón JAA, Bloor A, Ghesquieres H, Sureda A, Schmitz N, Glass B, and Dreger P
- Abstract
Although broadly employed, consolidative autologous hematopoietic stem cell transplantation (autoHCT) for relapsed/refractory (r/r) T-cell/histiocyte-rich large B-cell lymphoma (THRLBCL) has never been specifically investigated. Here we have analyzed outcomes of autoHCT for THRLBCL compared to diffuse large cell B-cell lymphoma not otherwise specified (DLBCL). Eligible for this retrospective registry study were adult patients with r/r THRLBCL and DLBCL, respectively, who underwent a first autoHCT in a salvage-sensitive disease status as assessed by PET-CT between 2016 and 2021 and were registered with the European Society for Blood and Marrow Transplantation (EBMT) database. Primary endpoint was progression-free survival (PFS) 2 years after transplantation. Two-hundred-one patients with THRLBCL and 5,543 with DLBCL were included. There were no significant differences in terms of disease status at HCT, pretreatment lines, and interval from diagnosis to transplant between the cohorts, but patients with THRBCL were significantly younger, contained a higher proportion of men, and had a better performance status. Compared to DLBCL, THRLBCL was associated with significantly better 2-year PFS (78% vs. 59%; p<0.001) and overall survival (OS; 81% vs. 74%; p=0.02) because of a significantly lower 2-year relapse incidence (RI; 16% vs. 35%; p<0.001). On multivariate analysis, favorable relapse risk (hazard ratio (HR) 0.46, 95%CI 0.31-0.7) and PFS (HR 0.58, 95%CI 0.41-0.82) of patients with THRLBCL remained significant, while OS benefits (HR 0.78, 95%CI 0.54-1.12) did not. These results were validated in a propensity-score matched analysis. These data prove autoHCT as an effective treatment option for salvage-sensitive r/r THRLBCL., (Copyright © 2024 American Society of Hematology.)
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- 2024
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17. Donor's age influences outcome in haploidentical hematopoietic stem cell transplantation with post-transplant cyclophosphamide - a single center experience.
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Zielińska P, Wieczorkiewicz-Kabut A, Białas K, Koclęga A, Gruenpeter K, Kopińska A, Woźniczka K, Noster I, Gromek T, Czyż J, Grosicki S, Wierzbowska A, Krzanowski J, Butrym A, and Helbig G
- Subjects
- Humans, Male, Female, Middle Aged, Adult, Age Factors, Aged, Graft vs Host Disease etiology, Graft vs Host Disease prevention & control, Tissue Donors, Transplantation, Haploidentical, Adolescent, Transplantation Conditioning methods, Young Adult, Retrospective Studies, Hematologic Neoplasms therapy, Hematologic Neoplasms mortality, Immunosuppressive Agents therapeutic use, Treatment Outcome, Survival Rate, Cyclophosphamide therapeutic use, Cyclophosphamide administration & dosage, Hematopoietic Stem Cell Transplantation methods
- Abstract
Haploidentical stem cell transplantation (haplo-SCT) using post-transplantation cyclophosphamide (post-Cy) is considered a reasonable therapeutic option for patients who lack matched donor or who urgently need transplant procedure due to high risk disease. We analyzed the results of haplo-SCT performed in years 2018-2023. Eighty one patients (46 males) at median age of 52 years underwent haplo-SCT using peripheral blood as a stem cell source in most cases. Indications included hematological malignancies (acute leukemias in 88% of cases). In 25 cases (31%) transplantation was performed in relapsed/refractory disease. Majority of patients (61%) presented with very high and high disease risk index (DRI). Conditioning regimens were as follows: nonmyeloablative - 46 cases (57%), myeloablative - in 18 (22%) and reduced intensity - 17(20%). 90% of patients engrafted. All patients received unified immunosuppressive treatment (post-Cy/TAC/MMF). Median follow-up time was 12 months The cumulative incidence of acute and chronic GVHD was 37.5% and 37.6%, respectively. Estimated 2-year overall survival (OS) was 43.1% and donor's age was the only factor influencing survival. The 2-year progression-free survival (PFS) was 42.5%, whereas relapse incidence (RI) - 35%. The cumulative incidence of non-relapse mortality (NRM) was 44% and was mostly due to infections. Haplo-SCT is a feasible treatment option for hematological patients. Younger donor improves post-transplant survival. Strategies to reduce infection-related mortality and relapse rate remain a challenge., (© 2024. The Author(s).)
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- 2024
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18. Stem Cell Mobilization Performed with Different Doses of Cytarabine in Plasma Cell Myeloma Patients Relapsing after Previous Autologous Hematopoietic Cell Transplantation-A Multicenter Report by the Polish Myeloma Study Group.
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Drozd-Sokołowska J, Waszczuk-Gajda A, Topczewska M, Maciejewska M, Dutka M, Zaucha JM, Szmigielska-Kapłon A, Nowicki M, Olszewska-Szopa M, Szeremet A, Czyż A, Kozioł M, Hus M, Mańko J, Hus I, Romejko-Jarosińska J, Kopińska A, Helbig G, Mądry K, Boguradzki P, Król M, Snarski E, Hayden PJ, Jamroziak K, Dwilewicz-Trojaczek J, and Basak GW
- Abstract
Salvage autologous hematopoietic cell transplantation (auto-HCT) may be used to treat relapse of plasma cell myeloma occurring after previous auto-HCT. When an insufficient number of hematopoietic stem cells have been stored from the initial harvest, remobilization is necessary. Here, we aimed to analyze the efficacy and safety of different doses of cytarabine (total 800 vs. 1600 vs. 2400 mg/m
2 ) for remobilization. Sixty-five patients, 55% male, with a median age at remobilization 63 years, were included. Remobilization was performed with cytarabine_800 in 7, cytarabine_1600 in 36, and cytarabine_2400 in 22 patients. Plerixafor rescue was used in 25% of patients receiving cytarabine_1600 and 27% of those receiving cytarabine_2400. Patients administered cytarabine_800 were not rescued with plerixafor. Remobilization was successful in 80% of patients (57% cytarabine_800; 86% cytarabine_1600; 77% cytarabine_2400; p = 0.199). The yield of collected CD34+ cells did not differ between the different cytarabine doses ( p = 0.495). Patients receiving cytarabine_2400 were at the highest risk of developing severe cytopenias, requiring blood product support, or having blood-stream infections. One patient died of septic shock after cytarabine_2400. In summary, remobilization with cytarabine is feasible in most patients. All doses of cytarabine allow for successful remobilization. Cytarabine_2400 is associated with higher toxicity; therefore, lower doses (800 or 1600 mg/m2 ) seem to be preferable.- Published
- 2024
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19. Post-transplant cyclophosphamide, calcineurin inhibitor, and mycophenolate mofetil compared to anti-thymocyte globulin, calcineurin inhibitor, and methotrexate combinations as graft-versus-host disease prophylaxis post allogeneic stem cell transplantation from sibling and unrelated donors in patients with acute myeloid leukemia: a study on behalf of the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation.
- Author
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Nagler A, Labopin M, Swoboda R, Schroeder T, Hamladji RM, Griskevicius L, Salmenniemi U, Rambaldi A, Mielke S, Kulagin A, Passweg J, Luft T, Gedde-Dahl T, Forcade E, Helbig G, Stelljes M, Castilla-Llorente C, Spyridonidis A, Brissot E, Ciceri F, and Mohty M
- Subjects
- Humans, Male, Female, Middle Aged, Adult, Adolescent, Aged, Hematopoietic Stem Cell Transplantation methods, Hematopoietic Stem Cell Transplantation adverse effects, Young Adult, Allografts, Transplantation, Homologous methods, Immunosuppressive Agents therapeutic use, Graft vs Host Disease prevention & control, Graft vs Host Disease etiology, Antilymphocyte Serum therapeutic use, Mycophenolic Acid therapeutic use, Leukemia, Myeloid, Acute therapy, Cyclophosphamide therapeutic use, Calcineurin Inhibitors therapeutic use, Unrelated Donors, Methotrexate therapeutic use, Siblings
- Abstract
Post-transplant cyclophosphamide plus calcineurin inhibitor (CNI)(tacrolimus or cyclosporine A) plus mycophenolate mofetil (PTCy/TAC or CSA/MMF) and anti-thymocyte globulin plus CNI (tacrolimus or cyclosporine A) plus methotrexate (ATG/TAC or CSA/MTX) are common graft-versus-host disease (GVHD) prophylaxis regimens. We compared the two regimens in patients with acute myeloid leukemia (AML) undergoing allogeneic transplantation from matched siblings or unrelated donors. 402 received PTCy/TAC or CSA/MMF and 5648 received ATG/TAC or CSA/MTX. Patients in the PTCy-based group were younger (48.7 vs. 51.5 years, p = 0.024) and there was a higher frequency of patient cytomegalovirus seropositivity and female donor to male patient combination in this group (77.8% vs. 71.8%, p = 0.009 and 18.4% vs. 14.4%, p = 0.029, respectively). More patients in the PTCy-based group received reduced-intensity conditioning (51.5% vs. 41%, p < 0.0001). No differences were observed in the incidence of acute GVHD grade II-IV and III-IV (21.2% vs. 20.4%, p = 0.92 and 8.1% vs. 6%, p = 0.1) or 2-year total and extensive chronic GVHD (33.7% vs. 30%, p = 0.09 and 10.7% vs. 11.2%, p = 0.81) between the groups. In the multivariate analysis, all transplant outcomes did not differ between the groups. PTCy/CNI/MMF and ATG/CNI/MTX are alternative regimens for GVHD prophylaxis in AML patients., (© 2024. The Author(s), under exclusive licence to Springer Nature Limited.)
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- 2024
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20. Impact of comorbidities and body mass index on the outcomes of allogeneic hematopoietic cell transplantation in myelofibrosis: A study on behalf of the Chronic Malignancies Working Party of EBMT.
- Author
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Polverelli N, Bonneville EF, de Wreede LC, Koster L, Kröger NM, Schroeder T, Peffault de Latour R, Passweg J, Sockel K, Broers AEC, Clark A, Dreger P, Blaise D, Yakoub-Agha I, Petersen SL, Finke J, Chevallier P, Helbig G, Rabitsch W, Sammassimo S, Arcaini L, Russo D, Drozd-Sokolowska J, Raj K, Robin M, Battipaglia G, Czerw T, Hernández-Boluda JC, and McLornan DP
- Subjects
- Humans, Body Mass Index, Retrospective Studies, Transplantation Conditioning, Primary Myelofibrosis therapy, Neoplasms, Hematopoietic Stem Cell Transplantation
- Abstract
Investigating the evaluation of eligibility for transplant in myelofibrosis (MF): The role of HCT-CI and BMI. HCT-CI emerges as a key prognostic factor, while BMI shows limited impact. This study expands insights for better clinical decision-making in MF allo-HCT., (© 2024 Wiley Periodicals LLC.)
- Published
- 2024
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21. The Impact of Cryopreservation on Hematopoietic Stem Cell Engraftment and Post-transplant Outcome During the COVID-19 Pandemic.
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Strzelec A, Gawlik-Rzemieniewska N, Klima A, Panek K, and Helbig G
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- Humans, Male, Female, Adult, Middle Aged, Retrospective Studies, Graft Survival, Transplantation, Homologous, Treatment Outcome, Aged, Pandemics, Graft vs Host Disease etiology, Graft vs Host Disease epidemiology, Young Adult, Adolescent, Cryopreservation methods, COVID-19 epidemiology, Hematopoietic Stem Cell Transplantation methods, Hematopoietic Stem Cells cytology, SARS-CoV-2
- Abstract
Background/aim: The COVID-19 pandemic has had a significant impact on the current management of allotransplanted patients in whom fresh hematopoietic stem cells (HSCs) were replaced by cryopreserved ones. The aim of the study was to determine the efficacy and safety of cryopreserved HSCs when compared with the fresh ones., Patients and Methods: A retrospective analysis of 254 allogeneic stem cell transplantations (HSCT) procedures performed between 2020-2021 included the following donors: matched related (MRD; n=68), matched unrelated (MUD; n=148) and haploidentical (HID; n=38). 50% of patients (non-cryo group) received fresh grafts, whereas the remaining patients (cryo group) were transplanted with cryopreserved cells., Results: No differences in terms of median days to neutrophil [MRD/MUD/HID cryo- and non-cryo groups: 17 vs. 16 (p=0.27), 19 vs. 18 (p=0.83), 22 vs. 22 (p=0.83) days, respectively] and platelet [MRD/MUD/HID cryo- and non-cryo groups: 14 vs. 14 (p=0.25), 17 vs. 17 (p=0.33), 21 vs. 19 (p=0.36) days, respectively] engraftments were demonstrated. Among MUD graft recipients, platelet engraftment rates were 81% in the cryo- and 96% in the non-cryo group (p=0.01). OS rates were comparable at 1 year after HSCT between MRD/MUD/HID cryo- and non-cryo groups: 53% vs. 60% (p=0.54), 60% vs. 66% (p=0.5), 50% vs. 41% (p=0.56), respectively., Conclusion: During the COVID-19 pandemic, cryopreserved HSCs did not have a negative impact on median engraftment time and OS when compared to fresh HSCs. In the MUD group, platelet engraftment rate was lower in cryopreserved HSC recipients., (Copyright © 2024, International Institute of Anticancer Research (Dr. George J. Delinasios), All rights reserved.)
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- 2024
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22. Splenic irradiation for myelofibrosis prior to hematopoietic cell transplantation: A global collaborative analysis.
- Author
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Gagelmann N, Hobbs GS, Campodonico E, Helbig G, Novak P, Schroeder T, Schneider A, Rautenberg C, Reinhardt HC, Bosques L, Heuser M, Panagiota V, Thol F, Gurnari C, Maciejewski JP, Ciceri F, Rathje K, Robin M, Pagliuca S, Rubio MT, Rocha V, Funke V, Hamerschlak N, Salit R, Scott BL, Duarte F, Mitrus I, Czerw T, Greco R, and Kröger N
- Subjects
- Humans, Spleen, Splenomegaly etiology, Splenomegaly radiotherapy, Recurrence, Transplantation Conditioning methods, Primary Myelofibrosis radiotherapy, Primary Myelofibrosis complications, Hematopoietic Stem Cell Transplantation methods, Thrombocytopenia complications, Graft vs Host Disease etiology
- Abstract
Splenomegaly is the clinical hallmark of myelofibrosis. Splenomegaly at the time of allogeneic hematopoietic cell transplantation (HCT) is associated with graft failure and poor graft function. Strategies to reduce spleen size before HCT especially after failure to Janus kinase (JAK) inhibition represent unmet clinical needs in the field. Here, we leveraged a global collaboration to investigate the safety and efficacy of splenic irradiation as part of the HCT platform for patients with myelofibrosis. We included 59 patients, receiving irradiation within a median of 2 weeks (range, 0.9-12 weeks) before HCT. Overall, the median spleen size prior to irradiation was 23 cm (range, 14-35). Splenic irradiation resulted in a significant and rapid spleen size reduction in 97% of patients (57/59), with a median decrease of 5.0 cm (95% confidence interval, 4.1-6.3 cm). The most frequent adverse event was thrombocytopenia, with no correlation between irradiation dose and hematological toxicities. The 3-year overall survival was 62% (95% CI, 48%-76%) and 1-year non-relapse mortality was 26% (95% CI, 14%-38%). Independent predictors for survival were severe thrombocytopenia and anemia before irradiation, transplant-specific risk score, higher-intensity conditioning, and present portal vein thrombosis. When using a propensity score matching adjusted for common confounders, splenic irradiation was associated with significantly reduced relapse (p = .01), showing a 3-year incidence of 12% for splenic irradiation versus 29% for patients with immediate HCT and 38% for patients receiving splenectomy. In conclusion, splenic irradiation immediately before HCT is a reasonable approach in patients experiencing JAK inhibition failure and is associated with a low incidence of relapse., (© 2024 The Authors. American Journal of Hematology published by Wiley Periodicals LLC.)
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- 2024
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23. The Depth of the Molecular Response in Patients with Chronic Myeloid Leukemia Correlates with Changes in Humoral Immunity.
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Janowski M, Łuczkowska K, Gniot M, Lewandowski K, Safranow K, Helbig G, Machaliński B, and Paczkowska E
- Abstract
Background and Objectives : The effective treatment of chronic myeloid leukemia leads to the restoration of proper immune system function. We aimed to investigate fluctuations in circulating cytokines, angiogenic factors and complement components in patients with CML during the first year of treatment with TKI and correlate them with the degree of achieved molecular response. Material and Methods : We recruited 31 patients with newly diagnosed CML. Peripheral blood and bone marrow samples were obtained, and concentrations of serum proteins were measured using an immunology multiplex assay. Results : The study cohort was divided into two groups of optimal or non-optimal in accordance with the European Leukemia Net (ELN) guidelines. We found significantly higher concentrations of C1q, C4 and C5a in serum after 3 months of TKI treatment in patients who achieved optimal responses in the 6 months after diagnosis. The most alterations were observed during 12 months of therapy. Patients in the optimal response group were characterized by higher serum concentrations of TGF-β, EGF, VEGF, Angiopoietin 1, IFN-γ and IL-8. Conclusions : The later plasma concentrations of complement components were significantly increased in patients with optimal responses. The changes after 12 months of treatment were particularly significant. Similar changes in bone marrow samples were observed.
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- 2024
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24. Fatal outcome of a rare acute myeloid leukemia with t(8;16)(p11.2;p13.3) and KAT6A::CREBBP gene fusion in a young man.
- Author
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Spałek A, Bartkowska-Chrobok A, Kulińska-Kozak M, Szymczak B, Dziaczkowska-Suszek J, and Helbig G
- Abstract
Competing Interests: Conflicts of interest The authors declare no conflicts of interest. This research did not receive any specific grant from funding agencies in the public, commercial, or not-for-profit sectors.
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- 2024
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25. Allogeneic haematopoietic cell transplantation for therapy-related myeloid neoplasms arising following treatment for lymphoma: a retrospective study on behalf of the Chronic Malignancies Working Party of the EBMT.
- Author
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Nabergoj M, Eikema DJ, Koster L, Platzbecker U, Sockel K, Finke J, Kröger N, Forcade E, Nagler A, Eder M, Tischer J, Broers AEC, Kuball J, Wilson KMO, Hunault-Berger M, Collin M, Russo D, Corral LL, Helbig G, Mussetti A, Scheid C, Gurnari C, Raj K, Drozd-Sokolowska J, Yakoub-Agha I, Robin M, and McLornan DP
- Subjects
- Humans, Middle Aged, Retrospective Studies, Recurrence, Transplantation Conditioning, Leukemia, Myeloid, Acute therapy, Lymphoma etiology, Lymphoma therapy, Neoplasms, Second Primary etiology, Hematopoietic Stem Cell Transplantation adverse effects, Graft vs Host Disease etiology
- Abstract
Therapy-related myeloid neoplasms (t-MN), either myelodysplastic neoplasms (t-MDS) or acute myeloid leukemias (t-AML), have a poor prognosis and allogeneic haematopoietic cell transplantation (allo-HCT) represents the only curative option. In this multicenter, registry-based study, we analyzed outcomes of 378 patients undergoing first allo-HCT between 2006-2017 for t-MN arising secondary to lymphoma treatment. Median age was 58 years at allo-HCT; 222 (59%) had a diagnosis of t-MDS and 156 (41%) of t-AML, respectively. At the time of allo-HCT, 46% of t-MN cases were reported as in complete remission (CR) and 15% of lymphomas were recorded as not in remission. A reduced intensity conditioning regimen was used in 70% of cases. For the entire cohort, 5-year OS, and t-MN PFS, relapse incidence and NRM were 32%, 28%, 35% and 37%, respectively. In multivariable analysis, undergoing allo-HCT with t-MN not in CR and older age were associated with significantly worse OS, PFS and NRM. At 5 years post allo-HCT, the relapse incidence of lymphoma was low at 3%, while the rate of secondary malignancies was 8%. This analysis shows the curative potential of allo-HCT for patients with t-MN arising secondary to lymphoma treatment in approximately a third of patients., (© 2024. The Author(s), under exclusive licence to Springer Nature Limited.)
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- 2024
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26. Real-Life Data on the Efficacy and Safety of Letermovir for Primary Prophylaxis of Cytomegalovirus in Allogeneic Hematopoietic Stem Cell Recipients: A Single-Center Analysis
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Włodarczyk M, Wieczorkiewicz-Kabut A, Białas K, Koclęga A, Noster I, Zielińska P, and Helbig G
- Subjects
- Adult, Humans, Young Adult, Middle Aged, Aged, Cytomegalovirus, Antiviral Agents adverse effects, Retrospective Studies, Transplantation, Homologous adverse effects, Cytomegalovirus Infections drug therapy, Cytomegalovirus Infections etiology, Cytomegalovirus Infections prevention & control, Hematopoietic Stem Cell Transplantation adverse effects, Acetates, Quinazolines
- Abstract
Objective: Cytomegalovirus (CMV) reactivation is a life-threatening complication after allogeneic hematopoietic stem cell transplantation (HSCT). Introduction of letermovir (LMV) seems to improve post-transplant outcomes, but delayed-onset CMV reactivation still remains a challenge. In this study, we report on our first experience with LMV prophylaxis in 93 CMV-seropositive adult patients receiving HSCT in our center., Materials and Methods: We retrospectively analyzed the data of 93 adult CMV-seropositive recipients receiving LMV as CMV prophylaxis after HSCT for hematological malignancies between 2019 and 2023. The starting LMV dose was 480 mg daily, reduced to 240 mg daily for those receiving cyclosporin A co-administration. CMV DNA in the blood was measured by real-time polymerase chain reaction weekly for the first 2 months after transplantation, then every other week until the end of immunosuppressive treatment. LMV was continued to day +100 or to CMV reactivation., Results: The median recipient age at the time of transplant was 51 (range: 20-71) years. All patients received grafts from peripheral blood, mostly for acute myeloid leukemia (60%). The median time from transplantation to LMV initiation was 3 (range: 0-24) days. While 55% of patients were transplanted from matched related donors, 32% had unrelated donors and 13% underwent haploidentical HSCT. Four patients (4%) had CMV “blips” while on LMV, but the drug was continued and repeated assays were negative. Only 2 patients (2%) experienced CMV reactivation while on LMV, on days 48 and 34 after HSCT, respectively. Seven patients (7%) developed late-onset CMV reactivation after a median of 124 days after HSCT (range: 118-152 days) and they were successfully treated with ganciclovir. CMV disease was not observed. Grade III-IV acute graft-versus-host disease occurred in 6 patients (6%) during LMV treatment. LMV treatment was free of side effects., Conclusion: LMV prophylaxis was effective in preventing CMV reactivation with a favorable safety profile. CMV reactivation occurred mostly after LMV discontinuation; thus, extending the duration of prophylaxis beyond 100 days could be beneficial., Competing Interests: Conflict of Interest: No conflict of interest was declared by the authors., (©Copyright 2024 by Turkish Society of Hematology Turkish Journal of Hematology, Published by Galenos Publishing House.)
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- 2024
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27. Are we ready for personalized CAR-T therapy?
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Strzelec A and Helbig G
- Subjects
- Humans, T-Lymphocytes, Immunotherapy, Adoptive adverse effects, Receptors, Chimeric Antigen genetics
- Abstract
The future of chimeric antigen receptor T (CAR-T) therapy remains unclear. New studies are constantly being published confirming the efficacy and favorable safety profile of its innovative enhancements. Currently approved CAR-T drugs are manufactured exclusively for a specific patient from the recipient's own cells. This does not close the door to further modifications with subsequent personalization and better adaptation to the individual needs. Bringing such a drug to market would involve raising the already high costs, so it is necessary to lower the existing ones. On the other hand, so-called universal CAR-T are also getting closer to the patient's bed, but its implementation may struggle with multiple challenges, including development of graft-versus-host disease (GvHD) and alloimmunity. However, that off-the-shelf therapy could prove useful as a quick solution for patients in very poor condition or excluded from current therapy due to manufacturing limitations. The introduction of currently tested solutions may undoubtedly change the current paradigm of treatment., (© 2023 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd.)
- Published
- 2024
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28. Prognostic Impact of Copy Number Alterations' Profile and AID/RAG Signatures in Acute Lymphoblastic Leukemia (ALL) with BCR::ABL and without Recurrent Genetic Aberrations (NEG ALL) Treated with Intensive Chemotherapy.
- Author
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Libura M, Karabin K, Tyrna P, Czyż A, Makuch-Łasica H, Jaźwiec B, Paluszewska M, Piątkowska-Jakubas B, Zawada M, Gniot M, Trubicka J, Szymańska M, Borg K, Więsik M, Czekalska S, Florek I, Król M, Paszkowska-Kowalewska M, Gil L, Kapelko-Słowik K, Patkowska E, Tomaszewska A, Mądry K, Machowicz R, Czerw T, Piekarska A, Dutka M, Kopińska A, Helbig G, Gromek T, Lewandowski K, Zacharczuk M, Pastwińska A, Wróbel T, Haus O, Basak G, Hołowiecki J, Juszczyński P, Lech-Marańda E, Giebel S, and Jędrzejczak WW
- Abstract
Adult acute lymphoblastic leukemia (ALL) is associated with poor outcomes. ALL is initiated by primary aberrations, but secondary genetic lesions are necessary for overt ALL. In this study, we reassessed the value of primary and secondary aberrations in intensively treated ALL patients in relation to mutator enzyme expression. RT-PCR, genomic PCR, and sequencing were applied to evaluate primary aberrations, while qPCR was used to measure the expression of RAG and AID mutator enzymes in 166 adult ALL patients. Secondary copy number alterations (CNA) were studied in 94 cases by MLPA assay. Primary aberrations alone stratified 30% of the patients (27% high-risk, 3% low-risk cases). The remaining 70% intermediate-risk patients included BCR::ABL1
pos subgroup and ALL lacking identified genetic markers (NEG ALL). We identified three CNA profiles: high-risk bad-CNA (CNAhigh / IKZF1pos ), low-risk good-CNA (all other CNAs), and intermediate-risk CNAneg . Furthermore, based on RAG/AID expression, we report possible mechanisms underlying the CNA profiles associated with poor outcome: AID stratified outcome in CNAneg , which accompanied most likely a particular profile of single nucleotide variations, while RAG in CNApos increased the odds for CNAhigh / IKZF1pos development. Finally, we integrated primary genetic aberrations with CNA to propose a revised risk stratification code, which allowed us to stratify 75% of BCR::ABL1pos and NEG patients.- Published
- 2023
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29. Effect of transplanted cells with CD184 and CD26 expressions and reconstitution of CD3+ lymphocyte population on long-term survival after autologous hematopoietic stem cell transplantation for multiple myeloma.
- Author
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Kopinska A, Krawczyk-Kulis M, Wieczorkiewicz-Kabut A, Koclega A, Jagoda K, Dziaczkowska-Suszek J, and Helbig G
- Subjects
- Humans, Prospective Studies, Dipeptidyl Peptidase 4 analysis, Lymphocytes, Transplantation, Autologous, Lymphocyte Subsets, Multiple Myeloma therapy, Hematopoietic Stem Cell Transplantation adverse effects
- Abstract
Autologous hematopoietic stem cell transplantation (auto-SCT) is the recommended treatment for responding patients with multiple myeloma (MM). However, we do not know the risk factors influencing long-term survival without progression after auto-SCT. Therefore, this prospective study aimed to investigate the influence of transplanted cells with cluster of differentiation (CD)184+ expression, CD26+ lymphocytes and monocytes, and reconstitution of CD3+ lymphocytes on overall survival (OS) and progression-free survival (PFS) after auto-SCT in MM. Forty-eight patients with MM underwent auto-SCT at our center from 2011 to 2013. The numbers of CD184+ cells, CD26+ lymphocytes, and CD26+ monocytes were measured in the harvested material. In addition, the number of lymphocyte subpopulations (CD3+ lymphocytes, helpers, suppressors, natural killer (NK), cytotoxic NK, and B lymphocytes) was measured in peripheral blood during regeneration after auto-SCT. Flow cytometry was performed in both cases. The median OS was 92 months. Our analysis revealed a statistically significant effect of the number of transplanted CD184+ cells on OS and a statistically significant correlation between PFS and the number of transplanted CD184+ cells and reconstitution of CD3+ lymphocytes. In conclusion, our study showed that the increasing numbers of transplanted CD184+ cells, CD26+ lymphocytes, and CD26+ monocytes augmented the risk of death., Competing Interests: Conflict of Interest Disclosure The authors do not have any conflicts of interest to declare in relation to this work., (Copyright © 2023. Published by Elsevier Inc.)
- Published
- 2023
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30. Impact of pre-transplantation depression and anxiety on the outcome of allogeneic hematopoietic stem cell transplantation: a study from the Transplant Complications Working Party of the EBMT.
- Author
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Gjærde LK, Peczynski C, Polge E, Kröger N, de Latour RP, Finke J, Holler E, Blaise D, Helbig G, Salmenniemi U, Potter V, Bunjes D, Erzsebet L, Penack O, Schoemans H, Koenecke C, Basak GW, and Perić Z
- Subjects
- Humans, Transplantation, Homologous, Anxiety etiology, Retrospective Studies, Transplantation Conditioning, Depression, Hematopoietic Stem Cell Transplantation
- Published
- 2023
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31. Adoptive Immunotherapy via Donor Lymphocyte Infusions following Allogeneic Hematopoietic Stem Cell Transplantation for Myelofibrosis: A Real-World, Retrospective Multicenter Study.
- Author
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Rampotas A, Sockel K, Panitsas F, Theuser C, Bornhauser M, Hernani R, Hernandez-Boluda JC, Esquirol A, Avenoso D, Tsirigotis P, Robin M, Czerw T, Helbig G, Roddie C, Lambert J, and McLornan DP
- Subjects
- Humans, Middle Aged, Retrospective Studies, Immunotherapy, Adoptive adverse effects, Neoplasm Recurrence, Local complications, Lymphocytes, Recurrence, Primary Myelofibrosis therapy, Primary Myelofibrosis complications, Hematopoietic Stem Cell Transplantation adverse effects, Graft vs Host Disease
- Abstract
Allogeneic hematopoietic cell transplantation (allo-HCT) remains the sole curative option for myelofibrosis (MF). Relapse remains a significant problem, however, occurring in up to 20% to 30% of cases. Donor lymphocyte infusion (DLI) represents a potentially effective strategy for relapse prevention and management, but the optimal timing based on measurable residual disease/chimerism analyses and the choice of regimen remain undetermined. We performed a retrospective real-world analysis of a multicenter cohort of MF allo-HCT recipients from 8 European transplantation centers who received DLI between 2005 and 2022. Response was assessed using International Working Group-Myeloproliferative Neoplasms Research and Treatment-defined response criteria, and survival endpoints were estimated using the Kaplan-Meier estimator and log-rank test. The study included 28 patients with a median age of 58 years and a Karnofsky Performance Status of >80. The majority of patients had Dynamic International Prognostic Scoring System-plus intermediate-2 or high-risk disease at the time of allo-HCT. In vivo T cell depletion was used in 20 patients (71.2%), with 19 of the 20 receiving antithymocyte globulin. The indication for DLI was either "preemptive" (n = 15), due to a decrease in recipient chimerism (n = 13) or molecular relapse (n = 2), or "therapeutic" (n = 13) for clinician-defined hematologic/clinical relapse. No patient received DLI prophylactically. The median time of DLI administration was 23.4 months post allo-HCT. Of the 16 patients receiving multiple DLIs, 12 were part of a planned escalating dose regimen. The median follow-up from the time of first DLI was 55.4 months. The responses to DLI were complete response in 9 patients, partial response in 1 patient, and clinical improvement in 6 patients. Chimerism levels improved in 16 patients, and stable disease was reported in 5 patients. No response or progression was reported in 7 patients. DLI-induced acute graft-versus-host disease (aGVHD) was reported in 11 patients (39%), with grade III-IV aGVHD in 7 (25%). The median overall survival from the time of first DLI was 62.6 months, and the cumulative incidence of relapse/progression after first DLI was 30.8% at 6 months. This study highlights that good response rates can be achieved with DLI even after frank relapse in some patients in a cohort in which other treatment options are very limited. More prospective studies are warranted to identify the optimal DLI regimen and timing to improve patient outcomes., (Copyright © 2023 The American Society for Transplantation and Cellular Therapy. Published by Elsevier Inc. All rights reserved.)
- Published
- 2023
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32. Allogeneic hematopoietic stem cell transplantation for acquired severe aplastic anemia: a summary of a 20-year experience.
- Author
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Zielińska P, Noster I, Wieczorkiewicz-Kabut A, Białas K, Koclęga A, and Helbig G
- Subjects
- Male, Humans, Adult, Retrospective Studies, Quality of Life, Treatment Outcome, Immunosuppressive Agents, Anemia, Aplastic therapy, Anemia, Aplastic complications, Hematopoietic Stem Cell Transplantation adverse effects, Graft vs Host Disease etiology
- Abstract
Introduction: Severe aplastic anemia (SAA) is a rare but potentially fatal disorder characterized by hypocellular bone marrow and resulting in pancytopenia. It can be cured with allogeneic hematopoietic stem cell transplantation (allo‑HSCT), especially in young individuals., Objectives: The main objective of the study was to assess the safety of the procedure and to identify the factors influencing long‑term post‑transplant outcome., Patients and Methods: Using our institutional database, we performed a retrospective analysis of the patients with SAA allotransplanted in the years 2001-2021., Results: Seventy patients (49 men) at a median age of 25 years at transplantation underwent allo‑HSCT. Thirty-eight patients received immunosuppressive treatment (IST) before transplantation. Twenty-one patients received grafts from human leukocyte antigen-matched sibling, 44 from unrelated donors, and 5 from haploidentical related donors. Peripheral blood remained the source of stem cells in the majority of patients. Primary graft failure was observed in 2 cases. The incidence of acute graft‑versus‑host disease (GVHD) was 44%, whereas chronic GVHD was observed only in 4 patients. Median follow‑up was 3 years (interquartile range, 0.45-11.5). Post‑transplant outcome was comparable between patients with upfront allo‑HSCT and those who relapsed after IST. In the univariable analysis, only a higher Eastern Cooperative Oncology Group (ECOG) score at transplantation and infections in the post‑transplant period were found to be associated with unfavorable outcome. Fifty‑three patients were alive at last contact. Most transplanted patients died due to infectious complications. A 2‑year overall survival was 73%., Conclusions: The results of allo‑HSCT in SAA are satisfactory and offer long‑term survival and good quality of life. Higher ECOG score and the presence of infections are associated with poor post‑transplant outcome.
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- 2023
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33. Allogeneic hematopoietic stem cell transplantation remains a feasible approach for elderly with acute myeloid leukemia: a 10-year experience.
- Author
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Duda K, Wieczorkiewicz-Kabut A, Koclęga A, Zielińska P, Woźniczka K, Krzemień H, Armatys A, and Helbig G
- Subjects
- Male, Humans, Aged, Unrelated Donors, Chronic Disease, Transplantation Conditioning methods, Retrospective Studies, Leukemia, Myeloid, Acute, Hematopoietic Stem Cell Transplantation adverse effects, Graft vs Host Disease etiology, Cytomegalovirus Infections complications
- Abstract
The incidence of AML increases with age. The implementation of reduced intensity conditioning and progress in supportive care enabled to perform allo-HSCT in elderly patients. The main objective of the study was to assess the safety and efficacy of allotransplantation in elderly AML.Forty nine patients (33 males) at median age of 68 years were identified. Data on patients' and transplant's related variables were retrieved from our local transplant registry. Most patients (65%) were transplanted from 10/10-HLA or 9/10-HLA matched unrelated donor, seven patients (14%) received stem cells from matched related donor and ten patients (20%) from haploidentical donor. All patients received reduced-intensity conditioning (RIC). Peripheral blood was a source of stem cells in all patients except one (98%). Acute GVHD developed in 22 patients (44%) with 5 individuals presenting grade III-IV. CMV reactivation was demonstrated in 19 patients (39%) till day + 100. In total, 22 patients (45%) have died. The main causes of death included infectious complications (n = 9), relapse with subsequent chemotherapy resistance (n = 7), steroid-resistant GvHD (n = 4) and other causes (n = 2). Twenty-seven patients (55%) were alive at the last contact, presented full donor chimerism and remained in the complete remission. The probability of OS and relapse-free survival (RFS) were 57% and 81% at 2 years, respectively. Older donor age showed negative impact on relapse. CMV reactivation, the severity of acute graft versus host disease and older donor age negatively influenced survival. Allo-HSCT remains a safe, feasible and effective procedure for elderly AML patients., (© 2023. The Author(s).)
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- 2023
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34. Double Malignancy and Double Transplant-A Bumpy Road to Success.
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Razik M, Rozwadowska P, Koclęga A, and Helbig G
- Subjects
- Humans, Adult, Adolescent, Female, Transplantation, Homologous adverse effects, Immunosuppressive Agents therapeutic use, Hematopoietic Stem Cell Transplantation adverse effects, Graft vs Host Disease drug therapy, Graft vs Host Disease etiology, Neoplasms complications
- Abstract
The occurrence of secondary neoplasms in adult patients treated with chemotherapy in childhood is not uncommon. Prior chemotherapy is found to be an independent risk factor for the development of secondary malignancies, which are usually associated with a worse prognosis. The presented case is a 35-year-old female patient who was diagnosed with Ewing sarcoma in her late adolescence. The tumor was successfully treated with chemotherapy, but 3 years later she was diagnosed with T-cell lymphoblastic lymphoma. The patient received allogeneic hematopoietic stem cell transplantation (allo-HSCT) from human leukocyte antigen (HLA) matched related donor. The procedure was complicated by grade 2 acute graft-versus-host disease (GvHD) which resolved after implementation of immunosuppressive treatment. However, a year later, the patient developed extensive chronic GvHD (cGvHD) and required reintroduction of immunosuppressants. Prolonged immunosuppressive treatment with tacrolimus led to irreversible kidney failure. After a 2-year period of regular peritoneal dialysis, she was found to be eligible for a kidney transplant from a deceased donor. Now, 15 years after stem cell transplantation and 8 years after kidney transplantation, the patient remains in good condition overall, presenting with symptoms of limited cGvHD. The case described here presents a unique clinical scenario of a female patient who was successfully treated for her double malignancy. Moreover, she underwent effective double transplantations and was eventually found to be cured despite accompanying complications.
- Published
- 2023
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35. Allogeneic hematopoietic cell transplant for hairy cell leukemia: EBMT experience.
- Author
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Chihara D, Gras L, Zinger N, Kröger N, Mayer J, Passweg J, De Latour RP, Byrne J, Krüger W, Bohn JP, Platzbecker U, Blau IW, Bonifazi F, Helbig G, McDonald A, Mistrik M, Mohty M, Ram R, Sanz J, Llamas CV, Kreitman RJ, Hayden PJ, McLornan D, Tournilhac O, Van Gelder M, and Yakoub-Agha I
- Subjects
- Humans, Treatment Outcome, Transplantation, Homologous, Retrospective Studies, Leukemia, Hairy Cell therapy, Hematopoietic Stem Cell Transplantation
- Published
- 2023
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36. Fludarabine or cyclophosphamide in combination with total body irradiation as myeloablative conditioning prior to allogeneic hematopoietic cell transplantation for acute lymphoblastic leukemia: an analysis by the Acute Leukemia Working Party of the EBMT.
- Author
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Giebel S, Labopin M, Socié G, Aljurf M, Salmenniemi U, Labussière-Wallet H, Srour M, Kröger N, Zahrani MA, Lioure B, Reményi P, Arat M, Bourhis JH, Helbig G, Tbakhi A, Forcade E, Huynh A, Brissot E, Spirydonidis A, Savani BN, Peric Z, Nagler A, and Mohty M
- Subjects
- Adult, Humans, Whole-Body Irradiation adverse effects, Retrospective Studies, Transplantation, Homologous adverse effects, Cyclophosphamide therapeutic use, Acute Disease, Recurrence, Transplantation Conditioning adverse effects, Precursor Cell Lymphoblastic Leukemia-Lymphoma therapy, Precursor Cell Lymphoblastic Leukemia-Lymphoma complications, Hematopoietic Stem Cell Transplantation adverse effects, Leukemia, Myeloid, Acute complications, Graft vs Host Disease etiology
- Abstract
In this registry-based study we retrospectively compared outcomes of allogeneic hematopoietic cell transplantation (allo-HCT) for adult patients with acute lymphoblastic leukemia (ALL) following conditioning with total body irradiation (TBI) combined with either cyclophosphamide (Cy) or fludarabine (Flu). TBI 12 Gy + Cy was used in 2105 cases while TBI 12 Gy + Flu was administered to 150 patients in first or second complete remission. In a multivariate model adjusted for other prognostic factors, TBI/Cy conditioning was associated with a reduced risk of relapse (HR = 0.69, p = 0.049) and increased risk of grade 2-4 acute graft-versus-host disease (GVHD, HR = 1.57, p = 0.03) without significant effect on other transplantation outcomes. In a matched-pair analysis the use of TBI/Cy as compared to TBI/Flu was associated with a significantly reduced rate of relapse (18% vs. 30% at 2 years, p = 0.015) without significant effect on non-relapse mortality, GVHD and survival. We conclude that the use of myeloablative TBI/Cy as conditioning prior to allo-HCT for adult patients with ALL in complete remission is associated with lower risk of relapse rate compared to TBI/Flu and therefore should probably be considered a preferable regimen., (© 2023. The Author(s), under exclusive licence to Springer Nature Limited.)
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- 2023
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37. Ruxolitinib is highly effective in steroid-resistant graft-versus-host disease: real-world data from a single center.
- Author
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Spałek A, Wieczorkiewicz-Kabut A, Zielińska P, Kopińska A, and Helbig G
- Subjects
- Humans, Nitriles, Pyrimidines therapeutic use, Retrospective Studies, Steroids, Graft vs Host Disease drug therapy
- Published
- 2023
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38. Hemophagocytic lymphohistiocytosis in a 26-year-old male.
- Author
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Pilśniak A, Jarosińska A, Janoska-Gawrońska A, Helbig G, and Holecki M
- Abstract
Competing Interests: The authors declare no conflict of interest.
- Published
- 2023
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39. Analysis of Predictive Factors for Early Response to Ruxolitinib in 320 Patients with Myelofibrosis From the Polish Adult Leukemia Group (PALG) Registry.
- Author
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Góra-Tybor J, Gołos A, Mikulski D, Helbig G, Sacha T, Lewandowski K, Niesiobędzka-Krężel J, Bieniaszewska M, Wysogląd H, Grzybowska-Izydorczyk O, Seferyńska I, Sobas M, Czyżewska M, Michalska A, Sawicki W, Mazur M, Hus M, Bodzenta E, Olszewska-Szopa M, Włodarczyk M, Patkowska E, Świstek W, and Jamroziak K
- Subjects
- Humans, Adult, Retrospective Studies, Poland, Registries, Primary Myelofibrosis diagnosis, Primary Myelofibrosis drug therapy, Leukemia
- Abstract
Introduction: Ruxolitinib is widely used in myelofibrosis (MF). However, some patients do not optimally respond and require more efficacious treatment. Our analysis aimed to establish predictors of ruxolitinib response., Patients and Methods: We designed a multicenter, retrospective analysis of the efficacy of ruxolitinib treatment in patients with MF in 15 Polish hematology centers. As responses to ruxolitinib occur within the first 6 months, we used this point to evaluate the efficacy of treatment. Symptoms response was defined as ≥50% reduction of the MF constitutional symptoms assessed by Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS). Spleen response was defined as ≥50% reduction of the difference between the spleen's baseline length and the upper limit norm measured by ultrasonography., Results: 320 MF patients were enrolled. At 6 months of therapy, the spleen response was detected in 140 (50%) patients, and symptoms response in 241 patients (76%). Multivariable analysis identified leukocytosis <25 G/L (OR 2.06, 95%CI: 1.12-3.88, P = .0200), and reticulin fibrosis MF 1 (OR 2.22, 95%CI: 1.11-4.46, P = .0249) contributed to better spleen response. The time interval between MF diagnosis and ruxolitinib administration shorter than 3 months, and platelets ≥150 G/L (OR 1.69, 95% CI 1.01-2.83, P = .0466) influenced symptoms response., Conclusion: Establishing predictive factors for ruxolitinib response is particularly important given the potential for new therapies in MF. In patients with a low likelihood of responding to ruxolitinib, using other JAK inhibitors or adding a drug with a different mechanism of action to ruxolitinib may be of clinical benefit., (Copyright © 2022 Elsevier Inc. All rights reserved.)
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- 2023
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40. Allogeneic Hematopoietic Stem Cell Transplantation for Relapsed/Refractory Acute Myeloid Leukemia: A Single-Centre Experience.
- Author
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Kopińska A, Węglarz P, Koclęga A, Wieczorkiewicz-Kabut A, Woźniczka K, Armatys A, Spałek A, Grygoruk-Wiśniowska I, Grosicki S, Butrym A, Czyż J, Obara A, Gromek T, and Helbig G
- Subjects
- Humans, Transplantation Conditioning methods, Disease Progression, Retrospective Studies, Hematopoietic Stem Cell Transplantation adverse effects, Hematopoietic Stem Cell Transplantation methods, Leukemia, Myeloid, Acute, Cytomegalovirus Infections complications, Graft vs Host Disease etiology
- Abstract
Introduction: Patients with relapsed/refractory acute myeloid leukemia (r/r AML) are characterized as having a poor prognosis. The only viable option of treatment for these patients is allogenic stem cell transplantation (allo-HSCT). Therefore, we have attempted to analyse factors related to both the disease itself and the transplantation procedure that could have an influence on the improvement of outcomes in this group of patients., Patients and Methods: Sixty-four patients with r/r AML underwent allo-HSCT at our center in 2012 to 2021. Fifty-two had active disease at the beginning of theallo-HSCT procedure, with amedian number of blasts in bone marrow (BM) of 18, and 12 had therapeutic aplasia after the last reinduction (blasts < 5% in BM)., Results: The probability of overall survival (OS) at 2 years was 25%. The median follow-up for survivors was 21.5 months. Progression-free survival (PFS) estimates were above 46%. The main cause of death was disease progression (49%). A statistically significant effect on premature death was reported for the diagnosis of secondary AML (sAML) and cytomelovirus (CMV) reactivation post allo-HSCT. On the other hand, chronic graft versus host disease (cGVHD) decreased the risk of disease progression. sAML and CMV reactivation were found to have opposite effects., (Copyright © 2022. Published by Elsevier Inc.)
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- 2023
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41. Allogeneic hematopoietic cell transplantation in patients with chronic phase chronic myeloid leukemia in the era of third generation tyrosine kinase inhibitors: A retrospective study by the chronic malignancies working party of the EBMT.
- Author
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Chalandon Y, Sbianchi G, Gras L, Koster L, Apperley J, Byrne J, Salmenniemi U, Sengeloev H, Aljurf M, Helbig G, Kinsella F, Choi G, Reményi P, Snowden JA, Robin M, Lenhoff S, Mielke S, Passweg J, Broers AEC, Kröger N, Yegin ZA, Tan SM, Hayden PJ, McLornan DP, and Yakoub-Agha I
- Subjects
- Humans, Imatinib Mesylate therapeutic use, Protein Kinase Inhibitors therapeutic use, Retrospective Studies, Tyrosine Kinase Inhibitors, Leukemia, Myelogenous, Chronic, BCR-ABL Positive drug therapy, Leukemia, Myeloid, Chronic-Phase drug therapy, Hematopoietic Stem Cell Transplantation
- Abstract
Following the introduction of tyrosine kinase inhibitors (TKI), the number of patients undergoing allogeneic hematopoietic cell transplantation (allo-HCT) for chronic phase (CP) chronic myeloid leukemia (CML) has dramatically decreased. Imatinib was the first TKI introduced to the clinical arena, predominantly utilized in the first line setting. In cases of insufficient response, resistance, or intolerance, CML patients can subsequently be treated with either a second or third generation TKI. Between 2006 and 2016, we analyzed the impact of the use of 1, 2, or 3 TKI prior to allo-HCT for CP CML in 904 patients. A total of 323-, 371-, and 210 patients had 1, 2, or 3 TKI prior to transplant, respectively; imatinib (n = 778), dasatinib (n = 508), nilotinib (n = 353), bosutinib (n = 12), and ponatinib (n = 44). The majority had imatinib as first TKI (n = 747, 96%). Transplants were performed in CP1, n = 549, CP2, n = 306, and CP3, n = 49. With a median follow-up of 52 months, 5-year OS for the entire population was 64.4% (95% CI 60.9-67.9%), PFS 50% (95% CI 46.3-53.7%), RI 28.7% (95% CI 25.4-32.0%), and NRM 21.3% (95% CI 18.3-24.2%). No difference in OS, PFS, RI, or NRM was evident related to the number of TKI prior to allo-HCT or to the type of TKI (p = ns). Significant factors influencing OS and PFS were > CP1 versus CP1 and Karnofsky performance (KPS) score > 80 versus ≤80, highlighting CP1 patients undergoing allo-HCT have improved survival compared to >CP1 and the importance of careful allo-HCT candidate selection., (© 2022 The Authors. American Journal of Hematology published by Wiley Periodicals LLC.)
- Published
- 2023
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42. Real-world experience with ruxolitinib for steroid-refractory acute graft-versus-host disease: a single center experience.
- Author
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Spałek A, Wieczorkiewicz-Kabut A, Koclęga A, Woźniczka K, Węglarz P, Boral K, Kata D, Zielińska P, and Helbig G
- Subjects
- Humans, Pyrimidines therapeutic use, Pyrazoles adverse effects, Nitriles therapeutic use, Steroids therapeutic use, Acute Disease, Retrospective Studies, Graft vs Host Disease drug therapy, Graft vs Host Disease etiology, Hematopoietic Stem Cell Transplantation adverse effects
- Abstract
Steroid-refractory acute graft-versus-host disease (SR-aGVHD) remains a major cause of morbidity and mortality after allogeneic hematopoietic stem cell transplantation (allo-HSCT). Ruxolitinib (RUX), an oral JAK1 and JAK2 inhibitor, has recently been approved for patients with SR-aGVHD. The aim of this study was to evaluate RUX efficacy and toxicity in a real-world setting. Eighteen patients received RUX at 5 mg or 10 mg twice a day after a median 3 lines of prior unsuccessful immunosuppressive therapy. Median time on RUX therapy was 28 days (range 7-129). Five patients (28%) responded to RUX, including 4 complete responses and 1 partial response. Response to RUX was irrespective of aGVHD grade and the number of involved organs. One-year overall survival (OS) was 60% for RUX-responders versus 31% for non-responders (p = ns). Treatment duration greater than 29.5 days was found to have a positive impact on OS (p < 0.007). Major adverse events during RUX treatment were grade 3-4 thrombocytopenia (61% of patients) and cytomegalovirus reactivation (50%). After median follow-up of 55 days (range 29-706), 14 patients (78%) died, mainly due to further progression of GVHD. RUX may represent a valuable therapeutic option for some patients with advanced SR-aGVHD, but more studies are warranted., (© 2022. The Author(s).)
- Published
- 2022
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43. Spectacular and Prompt Response to Extracorporeal Photopheresis for Refractory Cutaneous Chronic Graft-Versus-Host Disease after Allogeneic Hematopoietic Stem Cell Transplantation: A Case Report.
- Author
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Spałek A, Grygoruk-Wiśniowska I, Gruenpeter K, Panz-Klapuch M, and Helbig G
- Subjects
- Humans, Steroids therapeutic use, Chronic Disease, Bronchiolitis Obliterans Syndrome, Photopheresis adverse effects, Graft vs Host Disease drug therapy, Graft vs Host Disease etiology, Hematopoietic Stem Cell Transplantation adverse effects, Skin Diseases therapy, Skin Diseases complications
- Abstract
Chronic graft-versus-host disease (cGVHD) is a serious complication after allogenic hematopoietic stem cell transplantation (allo-HSCT), negatively affecting the morbidity and mortality of recipients. Skin involvement is the most common cGVHD manifestation with a wide range of pleomorphic features, from scleroderma to ulcerations and microangiopathic changes. Despite the access to many immunosuppressive drugs, therapy for cGVHD is challenging. Systemic steroids are recommended as the first-line treatment; but, in steroid-resistant patients, extracorporeal photopheresis (ECP) remains one of the subsequent therapeutic options. Here, we present a case report of a 31-year patient suffering from advanced steroid-refractory skin and oral mucosa cGVHD who was spectacularly treated with ECP. It was the first time we observed such "overnight" resolution of the graft-versus-host disease syndrome. The present report proves the important role of ECP in the treatment of steroid-resistant cGVHD, especially when other immunosuppressive therapies have failed.
- Published
- 2022
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44. Molecular Changes in Chronic Myeloid Leukemia During Tyrosine Kinase Inhibitors Treatment. Focus on Immunological Pathways.
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Janowski M, Ulańczyk Z, Łuczkowska K, Sobuś A, Rogińska D, Pius-Sadowska E, Gniot M, Kozłowski K, Lewandowski K, Helbig G, Machaliński B, and Paczkowska E
- Abstract
Introduction: The aim of our research was to investigate changes in the molecular background of the immune response in the chronic phase (CP) of chronic myeloid leukaemia (CML) during treatment with tyrosine kinase inhibitors (TKIs)., Methods: Global gene and miRNA expression profiles were assessed using genome-wide RNA and miRNA microarray technology in bone marrow mononuclear cells. Fifty-one patients were recruited, and bone marrow samples were taken at diagnosis before treatment with TKIs and after 3, 6, and 12 months of treatment with TKIs. The largest number of upregulated genes was observed when the 0-month group (time of diagnosis) was compared to the 3-month group; 1774 genes were significantly upregulated, and 390 genes were significantly downregulated., Discussion: Upregulated biological processes according to gene ontology (GO) classification involved basic cellular processes such as cell division, cell cycle, cell-cell adhesion, protein transport, mitotic nuclear division, apoptosis, and DNA replication. Differentially expressed miRNAs were annotated using GO classification to several immunity-related processes, including the T cell receptor signalling pathway, T cell costimulation, immune response, and inflammatory response. TKI therapy exerts a significant impact on cellular cycle processes and T-cell activation, which was proven at the molecular level., Competing Interests: Dr Krzysztof Kozłowski reports grants from Medical Research Agency—Poland, during the conduct of the study. The authors declare no other conflicts of interest in this work., (© 2022 Janowski et al.)
- Published
- 2022
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45. Room for Improvement: A 20-Year Single Center Experience with Allogeneic Stem Cell Transplantation for Myelodysplastic Syndromes.
- Author
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Duda K, Wieczorkiewicz-Kabut A, Spałek A, Koclęga A, Kopińska AJ, Woźniczka K, and Helbig G
- Abstract
Allogeneic stem cell transplantation (allo-SCT) remains the only curative therapeutic approach for patients with myelodysplastic syndromes (MDS). The aim of the study was to assess the efficacy/safety of allo-SCT as well as to identify factors influencing post-transplant survival. One hundred and two MDS patients (median age: 48 years; 57 males) who underwent allo-SCT were retrospectively evaluated. Twenty seven patients were transplanted from HLA-matched sibling and 75 patients received grafts from unrelated donors. Peripheral blood was a source of stem cell for 79 patients. Reduced intensity conditioning was used in 64 subjects. Acute and chronic graft versus host disease (GvHD) developed in 61 and 19 of patients, respectively. In total, 61 patients have died. The causes of deaths included infectious complications (n = 30), steroid-resistant GvHD (n = 17), MDS relapse (n = 9) and transformation to AML (n = 5). Non-relapse mortality and cumulative incidence of relapse at 2 years were 49.8% and 9%, respectively. 41 patients are alive at last contact and present full donor chimerism. 38 patients remain in complete hematological remission (CHR), 3 patients had CHR with incomplete platelet recovery. Median follow-up from diagnosis of MDS and transplantation are 27.1 months and 7 months respectively. Overall survival and relapse-free survival were 41% at 2 years. Increased serum ferritin level > 1000 ng/ml, presence of acute GvHD, grades III-IV acute GvHD and high hematopoietic cell transplantation-comorbidity index were found to negatively influenced survival. Allo-SCT for MDS is feasible procedure with a proportion of patients to be cured., Competing Interests: Conflict of interestsAll authors declared that they have no conflict of interest., (© The Author(s) 2021.)
- Published
- 2022
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46. Bing-Neel Syndrome, a Rare Presentation of Waldenström Macroglobulinemia-A Multicenter Report by the Polish Lymphoma Research Group.
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Drozd-Sokołowska J, Waszczuk-Gajda A, Witkowska M, Sienkiewicz E, Kopińska A, Kołkowska-Leśniak A, Barankiewicz J, Długosz-Danecka M, Smolewski P, Helbig G, Lech-Marańda E, Jurczak W, Biecek P, Giebel S, Wiktor-Jędrzejczak W, and Basak G
- Abstract
Bing-Neel syndrome (BNS) is a rare presentation of Waldenström macroglobulinemia (WM). BNS is a consequence of the central nervous system (CNS) involvement by lymphoplasmacytic lymphoma (LPL) and, rarely, the peripheral nervous system. The data on BNS are extremely scarce. Therefore, we performed a multicenter retrospective analysis of BNS patients diagnosed and treated in centers aligned with the Polish Lymphoma Research Group. The analysis covers the years 2014-2021. Eleven patients were included, 55% females and the median age at BNS diagnosis was 61 years. The median time from WM to BNS was 3.5 years; 27% of patients did have a diagnosis of WM and BNS made simultaneously or within 30 days from each other. Isolated parenchymal involvement was the least frequent (20%). Patients were treated with different regimens, mostly able to cross the blood-brain barrier, including 18% treated with ibrutinib first line. The cumulative objective response to treatment was 73%. With the median follow-up of 20 months (95% CI, 2-32), the 36-month estimates were: overall survival (OS) 47%, progression-free survival (PFS) 33%, and cumulative incidence of BNS-associated death 41%. The performance status according to ECOG was significant for PFS (HR = 7.79) and the hemoglobin concentration below 11 g/dL was correlated with PFS. To conclude, BNS is a very rare manifestation of WM. It is associated with a poor outcome with most patients succumbing to BNS.
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- 2022
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47. Allogeneic hematopoietic stem cell transplantation for relapsed B‑cell acute lymphoblastic leukemia after failure of autologous hematopoietic stem cell transplantation: a retrospective single-center analysis.
- Author
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Panz-Klapuch M, Spałek A, Duda K, Kopińska A, Wieczorkiewicz-Kabut A, and Helbig G
- Subjects
- Humans, Retrospective Studies, Transplantation, Autologous, Hematopoietic Stem Cell Transplantation, Precursor Cell Lymphoblastic Leukemia-Lymphoma therapy
- Published
- 2022
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48. Outcomes and toxicity of allogeneic hematopoietic cell transplantation in chronic myeloid leukemia patients previously treated with second-generation tyrosine kinase inhibitors: a prospective non-interventional study from the Chronic Malignancy Working Party of the EBMT.
- Author
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Masouridi-Levrat S, Olavarria E, Iacobelli S, Aljurf M, Morozova E, Niittyvuopio R, Sengeloev H, Reményi P, Helbig G, Browne P, Ganser A, Nagler A, Snowden JA, Robin M, Passweg J, Van Gorkom G, Wallet HL, Hoek J, Blok HJ, De Witte T, Kroeger N, Hayden P, Chalandon Y, and Agha IY
- Subjects
- Dasatinib adverse effects, Humans, Imatinib Mesylate adverse effects, Middle Aged, Prospective Studies, Protein Kinase Inhibitors adverse effects, Graft vs Host Disease etiology, Hematopoietic Stem Cell Transplantation adverse effects, Hematopoietic Stem Cell Transplantation methods, Leukemia, Myelogenous, Chronic, BCR-ABL Positive drug therapy
- Abstract
Allogeneic hematopoietic cell transplantation (allo-HCT) remains a treatment option for patients with chronic myeloid leukemia (CML) who fail to respond to tyrosine kinase inhibitors (TKIs). While imatinib seems to have no adverse impact on outcomes after transplant, little is known on the effects of prior use of second-generation TKI (2GTKI). We present the results of a prospective non-interventional study performed by the EBMT on 383 consecutive CML patients previously treated with dasatinib or nilotinib undergoing allo-HCT from 2009 to 2013. The median age was 45 years (18-68). Disease status at transplant was CP1 in 139 patients (38%), AP or >CP1 in 163 (45%), and BC in 59 (16%). The choice of 2GTKI was: 40% dasatinib, 17% nilotinib, and 43% a sequential treatment of dasatinib and nilotinib with or without bosutinib/ponatinib. With a median follow-up of 37 months (1-77), 8% of patients developed either primary or secondary graft failure, 34% acute and 60% chronic GvHD. There were no differences in post-transplant complications between the three different 2GTKI subgroups. Non-relapse mortality was 18% and 24% at 12 months and at 5 years, respectively. Relapse incidence was 36%, overall survival 56% and relapse-free survival 40% at 5 years. No differences in post-transplant outcomes were found between the three different 2GTKI subgroups. This prospective study demonstrates the feasibility of allo-HCT in patients previously treated with 2GTKI with a post-transplant complications rate comparable to that of TKI-naive or imatinib-treated patients., (© 2021. The Author(s).)
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- 2022
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49. Influence of pretransplant inflammatory bowel disease on the outcome of allogeneic hematopoietic stem cell transplantation: a matched-pair analysis study from the Transplant Complications Working Party (TCWP) of the EBMT.
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Peric Z, Peczynski C, Polge E, Kröger N, Sengeloev H, Radujkovic A, Helbig G, Russell N, Bunjes D, Socié G, Potter V, Beelen D, Crawley C, Bloor A, Finke J, Schoemans H, Penack O, Snowden JA, Koenecke C, and Basak GW
- Subjects
- Humans, Matched-Pair Analysis, Retrospective Studies, Transplantation Conditioning, Treatment Outcome, Hematopoietic Stem Cell Transplantation, Inflammatory Bowel Diseases therapy
- Published
- 2021
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50. Impact of prior JAK-inhibitor therapy with ruxolitinib on outcome after allogeneic hematopoietic stem cell transplantation for myelofibrosis: a study of the CMWP of EBMT.
- Author
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Kröger N, Sbianchi G, Sirait T, Wolschke C, Beelen D, Passweg J, Robin M, Vrhovac R, Helbig G, Sockel K, Conneally E, Rubio MT, Beguin Y, Finke J, Bernasconi P, Morozova E, Clausen J, von dem Borne P, Schaap N, Schroyens W, Patriarca F, Di Renzo N, Yeğin ZA, Hayden P, McLornan D, and Yakoub-Agha I
- Subjects
- Adult, Aged, Female, Graft vs Host Disease pathology, Humans, Male, Middle Aged, Primary Myelofibrosis pathology, Primary Myelofibrosis therapy, Recurrence, Retrospective Studies, Survival Rate, Treatment Outcome, Graft vs Host Disease immunology, Hematopoietic Stem Cell Transplantation methods, Janus Kinase Inhibitors therapeutic use, Nitriles therapeutic use, Primary Myelofibrosis drug therapy, Pyrazoles therapeutic use, Pyrimidines therapeutic use
- Abstract
JAK1/2 inhibitor ruxolitinib (RUX) is approved in patients with myelofibrosis but the impact of pretreatment with RUX on outcome after allogeneic hematopoietic stem cell transplantation (HSCT) remains to be determined. We evaluated the impact of RUX on outcome in 551 myelofibrosis patients who received HSCT without (n = 274) or with (n = 277) RUX pretreatment. The overall leukocyte engraftment on day 45 was 92% and significantly higher in RUX responsive patients than those who had no or lost response to RUX (94% vs. 85%, p = 0.05). The 1-year non-relapse mortality was 22% without significant difference between the arms. In a multivariate analysis (MVA) RUX pretreated patients with ongoing spleen response at transplant had a significantly lower risk of relapse (8.1% vs. 19.1%; p = 0.04)] and better 2-year event-free survival (68.9% vs. 53.7%; p = 0.02) in comparison to patients without RUX pretreatment. For overall survival the only significant factors were age > 58 years (p = 0.03) and HLA mismatch donor (p = 0.001). RUX prior to HSCT did not negatively impact outcome after transplantation and patients with ongoing spleen response at time of transplantation had best outcome., (© 2021. The Author(s).)
- Published
- 2021
- Full Text
- View/download PDF
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