EBMT NEWSLETTER | January 2018 | Volume 60 - Issue 1

Important dates

Summary of four EBMT recent studies published in peer-reviewed journals

Post-Transplantation Cyclophosphamide-Based Haploidentical Transplantation as Alternative to Matched Sibling or Unrelated Donor Transplantation for Hodgkin Lymphoma: A Registry Study of the Lymphoma Working Party of the European Society for Blood and Marrow Transplantation.
Martínez C, Gayoso J, Canals C, Finel H, Peggs K, Dominietto A, Castagna L, Afanasyev B, Robinson S, Blaise D, Corradini P, Itälä-Remes M, Bermúdez A, Forcade E, Russo D, Potter M, McQuaker G, Yakoub-Agha I, Scheid C, Bloor A, Montoto S, Dreger P, Sureda A; Lymphoma Working Party of the European Group for Blood and Marrow Transplantation.
J Clin Oncol. 2017 Oct 20;35(30):3425-3432. doi: 10.1200/JCO.2017.72.6869. Epub 2017 Aug 28.
PMID: 28846465
In this registry study by the Lymphoma working Party (LWP) outcome of patients with Hodgkin lymphoma who received post-transplantation cyclophosphamide-based haploidentical (HAPLO) allogeneic hematopoietic cell transplantation was compared with the outcome of patients who received conventional HLA-matched sibling donor (SIB) and HLA-matched unrelated donor (MUD) in 709 adult patients. No differences were observed between groups in the incidence of acute graft-versus-host disease (GVHD). HAPLO was associated with a lower risk of chronic GVHD (26%) compared with MUD (41%; P = .04). Cumulative incidence of nonrelapse mortality at 1 year was 17%, 13%, and 21% in HAPLO, SIB, and MUD, respectively, and corresponding 2-year cumulative incidence of relapse or progression was 39%, 49%, and 32%, respectively. On multivariable analysis, relative to SIB, nonrelapse mortality was similar in HAPLO ( P = .26) and higher in MUD ( P = .003), and risk of relapse was lower in both HAPLO ( P = .047) and MUD ( P < .001). Two-year overall survival and progression-free survival were 67% and 43% for HAPLO, 71% and 38% for SIB, and 62% and 45% for MUD, respectively. There were no significant differences in overall survival or progression-free survival between HAPLO and SIB or MUD. The rate of the composite end point of extensive chronic GVHD and relapse-free survival was significantly better for HAPLO (40%) compared with SIB (28%; P = .049) and similar to MUD (38%; P = .59).
The authors conclude that Post-transplantation cyclophosphamide-based HAPLO transplantation results in similar survival outcomes compared with SIB and MUD, which confirms its suitability when no conventional donor is available.
Haploidentical transplant in patients with myelodysplastic syndrome.
Robin M, Porcher R, Ciceri F, van Lint MT, Santarone S, Ehninger G, Blaise D, Güllbas Z, Gonzáles Muñiz S, Michallet M, Velardi A, Koster L, Maertens J, Sierra J, Selleslag D, Radujkovic A, Díez-Martin JL, Kanz L, Arroyo CH, Niederwieser D, Huang H, McDonald A, de Witte T, Koc Y, Kröger N. Blood Adv. 2017 Sep 27;1(22):1876-1883

Haploidentical stem cell transplantation with post transplant cyclophophamide as GvHD prophylaxis has become a reasonable option for patients with AML or NHL who are lacking a suitable donor. Data of haploidentical SCT in MDS patients were reported only very few. Here the CMWP reported on  228 patients transplanted from a mismatched HLA-related donor between 2007 and 2014. The median age at transplant was 56 years. Eighty-four (37%) patients had MDS transformed into acute myeloid leukemia at the time of transplant. Ex vivo T-cell depletion was used in 34 patients. One hundred ninety-four patients received a T-cell replete transplant and 102 patients received posttransplant cyclophosphamide (PT-CY) as graft-versus-host disease (GVHD) prophylaxis. The cumulative incidences of acute and chronic GVHD in PT-CY vs other patients were 25% vs 37% and 37% vs 24%, respectively. The cumulative incidence of nonrelapse mortality was 55% in patients who did not receive PT-CY (no PT-CY) and 41% in patients who did receive PT-CY. Three-year overall survival was 28% in no PT-CY patients and 38% in PT-CY patients. In multivariable analysis, the main risk factors were the intensity of the conditioning regimen and the use of PT-CY. The authors concluded that the outcomes of MDS patients who received an haploidentical transplant are close to the results other transplantations from HLA-mismatched donors with approximately one-third of patients alive and free of disease 3 years after transplant, and the use of PT-CY may improve their outcomes.

Fertility preservation issues in pediatric hematopoietic stem cell transplantation: practical approaches from the consensus of the Pediatric Diseases Working Party of the EBMT and the International BFM Study Group.
Balduzzi A, Dalle JH, Jahnukainen K, von Wolff M, Lucchini G, Ifversen M, Macklon KT, Poirot C, Diesch T, Jarisch A, Bresters D, Yaniv I, Gibson B, Willasch AM, Fadini R, Ferrari L, Lawitschka A, Ahler A, Sänger N, Corbacioglu S, Ansari M, Moffat R, Dalissier A, Beohou E, Sedlacek P, Lankester A, De Heredia Rubio CD, Vettenranta K, Wachowiak J, Yesilipek A, Trigoso E, Klingebiel T, Peters C, Bader P.
Bone Marrow Transplant. 2017 Oct;52(10):1406-1415. doi: 10.1038/bmt.2017.147. Epub 2017 Jul 24.
PMID: 28737775

Infertility is a major complication of stem cell transplantation. Especially for pediatric and young adults fertility preservation is an unmet clinical need. The Pediatric Disease Working Party (PWP) addressed this issue by providing a consensus  recommendation on fertility preservation. They recommend that patients and families should be informed of possible gender- and age-specific cryopreservation strategies that should be tailored according to the underlying disease, clinical condition and previous exposure to chemotherapy. Semen collection should be routinely offered to all postpubertal boys at the diagnosis of any disease requiring therapy that could potentially impair fertility. Testicular tissue collection might be offered to postpubertal boys; nevertheless, its use has been unsuccessful to date. Oocyte collection after hormonal hyperstimulation should be offered to postpubertal girls facing gonadotoxic therapies that could be delayed for the 2 weeks required for the procedure. Ovarian tissue collection could be offered to pre-/post-pubertal girls. Pregnancies have been reported after postpubertal ovarian tissue reimplantation; however, to date, no pregnancy has been reported after the reimplantation of prepubertal ovarian tissue or in vitro maturation of pre-/post-pubertal ovarian tissue. Health authorities should prioritize fertility preservation projects in pediatric transplantation to improve patient care and quality of life.

Transplant results in adults with Fanconi anaemia.
Bierings M, Bonfim C, Peffault De Latour R, Aljurf M, Mehta PA, Knol C, Boulad F, Tbakhi A, Esquirol A, McQuaker G, Sucak GA, Othman TB, Halkes CJM, Carpenter B, Niederwieser D, Zecca M, Kröger N, Michallet M, Risitano AM, Ehninger G, Porcher R, Dufour C; EBMT SAA WP.
Br J Haematol. 2018 Jan;180(1):100-109. doi: 10.1111/bjh.15006. Epub 2017 Nov 2.
PMID: 29094350
Hematopoietic stem cell transplantation for Fanconi Anemia (FA)  is mainly reported for pediatric patients . Data about stem cell transplantation in adults are reported only very few. The SAA Working Party collected 199 adult patients with FA transplanted between 1991 and 2014. Patients underwent transplantation at a median age of 23 years. Time between diagnosis and transplant was shortest (median 2 years) in those patients who had a human leucocyte antigen identical sibling donor. Fifty four percent of patients had bone marrow (BM) failure at transplantation and 46% had clonal disease (34% myelodysplasia, 12% acute leukaemia). BM was the main stem cell source, the conditioning regimen included cyclophosphamide in 96% of cases and fludarabine in 64%. Engraftment occurred in 82% (95% confidence interval [CI] 76-87%), acute graft-versus-host disease (GvHD) grade II-IV in 22% (95% CI 16-28%) and the incidence of chronic GvHD at 96 months was 26% (95% CI 20-33). Non-relapse mortality at 96 months was 56% with an overall survival of 34%, which improved with more recent transplants. Median follow-up was 58 months. Patients transplanted after 2000 had improved survival (84% at 36 months), using BM from an identical sibling and fludarabine in the conditioning regimen. Factors associated with improved outcome in multivariate analysis were use of fludarabine and an identical sibling or matched non-sibling donor. Main causes of death were infection (37%), GvHD (24%) and organ failure (12%). The presence of clonal disease at transplant did not significant impact on survival. Secondary malignancies were reported in 15 of 131 evaluable patients.


Evolution, trends, outcomes, and economics of hematopoietic stem cell transplantation in severe autoimmune diseases.
Snowden JA, Badoglio M, Labopin M, Giebel S, McGrath E, Marjanovic Z, Burman J, Moore J, Rovira M, Wulffraat NM, Kazmi M, Greco R, Snarski E, Kozak T, Kirgizov K, Alexander T, Bader P, Saccardi R, Farge D.
Blood Adv. 2017 Dec 20;1(27):2742-2755. doi: 10.1182/bloodadvances.2017010041. eCollection 2017 Dec 26.
PMID: 29296926
Association of aplastic anaemia and lymphoma: a report from the severe aplastic anaemia working party of the European Society of Blood and Bone Marrow Transplantation.
Rovó A, Kulasekararaj A, Medinger M, Chevallier P, Ribera JM, Peffault de Latour R, Knol C, Iacobelli S, Kanfer E, Bruno B, Maury S, Quarello P, Koh MBC, Schouten H, Blau IW, Tichelli A, Hill A, Risitano A, Passweg J, Marsh J, Dreger P, Dufour C.
Br J Haematol. 2017 Dec 19. doi: 10.1111/bjh.15074. [Epub ahead of print] No abstract available.
PMID: 29265360
Melphalan 140mg/m2 or 200mg/m2 for autologous transplantation in myeloma: results from the Collaboration to Collect Autologous Transplant Outcomes in Lymphoma and Myeloma (CALM) study. A report by the EBMT Chronic Malignancies Working Party.
Auner HW, Iacobelli S, Sbianchi G, Knol-Bout C, Blaise D, Russell NH, Apperley JF, Pohlreich D, Browne P, Kobbe G, Isaksson C, Lenhoff S, Scheid C, Touzeau C, Jantunen E, Anagnostopoulos A, Yakoub-Agha I, Tanase A, Schaap N, Wiktor-Jedrzejczak W, Krejci M, Schönland SO, Morris C, Garderet L, Kröger N.
Haematologica. 2017 Dec 7. pii: haematol.2017.181339. doi: 10.3324/haematol.2017.181339. [Epub ahead of print]
PMID: 29217776
Distinct factors determine the kinetics of disease relapse in adults transplanted for acute myeloid leukaemia.
Craddock C, Versluis J, Labopin M, Socie G, Huynh A, Deconinck E, Volin L, Milpied N, Bourhis JH, Rambaldi A, Chevallier P, Blaise D, Manz M, Vellenga E, Vekemans MC, Maertens J, Passweg J, Vyas P, Schmid C, Löwenberg B, Ossenkoppele G, Mohty M, Cornelissen JJ, Nagler A; Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation and HOVON-SAKK.
J Intern Med. 2017 Dec 7. doi: 10.1111/joim.12720. [Epub ahead of print]
PMID: 29214689
CAR-T cells: the narrow path between hope and bankruptcy?
Chabannon C, Kuball J, Mcgrath E, Bader P, Dufour C, Lankester A, Basak GW, Montoto S, Nagler A, Snowden JA, Styczynski J, Duarte RF, Kröger N, Mohty M.
Bone Marrow Transplant. 2017 Dec;52(12):1588-1589. doi: 10.1038/bmt.2017.241. No abstract available.
PMID: 29209061
Donor age determines outcome in acute leukemia patients over 40 undergoing haploidentical hematopoietic cell transplantation.
Canaani J, Savani BN, Labopin M, Huang XJ, Ciceri F, Arcese W, Koc Y, Tischer J, Blaise D, Gülbas Z, Van Lint MT, Bruno B, Mohty M, Nagler A.
Am J Hematol. 2018 Feb;93(2):246-253. doi: 10.1002/ajh.24963. Epub 2017 Dec 6.
PMID: 29114918
The survey on cellular and tissue-engineered therapies in Europe and neighboring Eurasian countries in 2014 and 2015.
Ireland H, Gay MHP, Baldomero H, De Angelis B, Baharvand H, Lowdell MW, Passweg J, Martin I; International Society for Cellular Therapy (ISCT); Tissue Engineering and Regenerative Medicine International Society—Europe (TERMIS-EU); International Cartilage Repair Society (ICRS); International Federation for Adipose Therapeutics (IFAT); European Group for Blood and Marrow Transplantation.
Cytotherapy. 2018 Jan;20(1):1-20. doi: 10.1016/j.jcyt.2017.08.009. Epub 2017 Oct 4.
PMID: 28988692
Occurrence of graft-versus-host disease increases mortality after umbilical cord blood transplantation for acute myeloid leukaemia: a report from Eurocord and the ALWP of the EBMT.
Baron F, Ruggeri A, Beohou E, Labopin M, Mohty M, Sanz J, Vigouroux S, Furst S, Bosi A, Chevallier P, Cornelissen JJ, Michallet M, Sierra J, Karakasis D, Savani BN, Gluckman E, Nagler A.
J Intern Med. 2017 Oct 4. doi: 10.1111/joim.12696. [Epub ahead of print]
PMID: 28977716
Relatively favorable outcome after allogeneic stem cell transplantation for BCR-ABL1-positive AML: A survey from the acute leukemia working party of the European Society for blood and marrow transplantation (EBMT).
Lazarevic VL, Labopin M, Depei W, Yakoub-Agha I, Huynh A, Ljungman P, Schaap N, Cornelissen JJ, Maillard N, Pioltelli P, Gedde-Dahl T, Lenhoff S, Houhou M, Esteve J, Mohty M, Nagler A.
Am J Hematol. 2018 Jan;93(1):31-39. doi: 10.1002/ajh.24928. Epub 2017 Oct 31.
PMID: 28971504
High dose chemotherapy and autologous stem cell transplantation in nodular lymphocyte-predominant Hodgkin lymphoma: A retrospective study by the European society for blood and marrow transplantation-lymphoma working party.
Akhtar S, Montoto S, Boumendil A, Finel H, Masszi T, Jindra P, Nemet D, Fuhrmann S, Beguin Y, Castagna L, Ferrara F, Capria S, Malladi R, Moraleda JM, Bloor A, Ghesquières H, Meissner J, Sureda A, Dreger P.
Am J Hematol. 2018 Jan;93(1):40-46. doi: 10.1002/ajh.24927. Epub 2017 Nov 9.
Unrelated matched versus autologous transplantation in adult patients with good and intermediate risk acute myelogenous leukemia in first molecular remission.
Gorin NC, Labopin M, Pabst T, Remenyi P, Wu D, Huynh A, Volin L, Cahn JY, Yakoub-Agha I, Mercier M, Houhou M, Mohty M, Nagler A; Acute Leukemia Working Party of the EBMT.
Am J Hematol. 2017 Dec;92(12):1318-1323. doi: 10.1002/ajh.24904. Epub 2017 Sep 28.
PMID: 28960419
Prognostic Scoring Systems in Allogeneic Hematopoietic Stem Cell Transplantation: Where Do We Stand?
Potdar R, Varadi G, Fein J, Labopin M, Nagler A, Shouval R.
Biol Blood Marrow Transplant. 2017 Nov;23(11):1839-1846. doi: 10.1016/j.bbmt.2017.07.028. Epub 2017 Aug 7. Review.
PMID: 28797781
An Integrative Scoring System for Survival Prediction Following Umbilical Cord Blood Transplantation in Acute Leukemia.
Shouval R, Ruggeri A, Labopin M, Mohty M, Sanz G, Michel G, Kuball J, Chevallier P, Al-Seraihy A, Milpied NJ, de Heredia CD, Arcese W, Blaise D, Rocha V, Fein J, Unger R, Baron F, Bader P, Gluckman E, Nagler A.
Clin Cancer Res. 2017 Nov 1;23(21):6478-6486. doi: 10.1158/1078-0432.CCR-17-0489. Epub 2017 Jul 28.
PMID: 28754820
Thiotepa-based conditioning versus total body irradiation as myeloablative conditioning prior to allogeneic stem cell transplantation for acute lymphoblastic leukemia: A matched-pair analysis from the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation.
Eder S, Canaani J, Beohou E, Labopin M, Sanz J, Arcese W, Or R, Finke J, Cortelezzi A, Beelen D, Passweg J, Socié G, Gurman G, Aljurf M, Stelljes M, Giebel S, Mohty M, Nagler A.
Am J Hematol. 2017 Oct;92(10):997-1003. doi: 10.1002/ajh.24823. Epub 2017 Jul 19.
PMID: 28614903
Incidence of Second Primary Malignancies after Autologous Transplantion for Multiple Myeloma in the Era of Novel Agents.
Sahebi F, Iacobelli S, Sbianchi G, Koster L, Blaise D, Reményi P, Russell NH, Ljungman P, Kobbe G, Apperley J, Trneny M, Krejci M, Wiktor-Jedrzejczak W, Sanchez JF, Schaap N, Isaksson C, Lenhoff S, Browne P, Scheid C, Wilson KMO, Yakoub-Agha I, González Muñiz S, Schönland S, Morris C, Garderet L, Kröger N.
Biol Blood Marrow Transplant. 2018 Jan 12. pii: S1083-8791(18)30021-1. doi: 10.1016/j.bbmt.2018.01.006. [Epub ahead of print
Long-term outcome analysis of reduced-intensity allogeneic stem cell transplantation in patients with mantle cell lymphoma: a retrospective study from the EBMT Lymphoma Working Party.
Robinson SP, Boumendil A, Finel H, Peggs KS, Chevallier P, Sierra J, Finke J, Poiré X, Maillard N, Milpied N, Yakoub-Agha I, Koh M, Kröger N, Nagler A, Koc Y, Dietrich S, Montoto S, Dreger P.
Bone Marrow Transplant. 2018 Jan 15. doi: 10.1038/s41409-017-0067-3. [Epub ahead of print]
Outcomes of haploidentical stem cell transplantation for chronic lymphocytic leukemia: a retrospective study on behalf of the chronic malignancies working party of the EBMT.
van Gorkom G, van Gelder M, Eikema DJ, Blok HJ, van Lint MT, Koc Y, Ciceri F, Beelen D, Chevallier P, Selleslag D, Blaise D, Foá R, Corradini P, Castagna L, Moreno C, Solano C, Müller LP, Tischer J, Hilgendorf I, Hallek M, Bittenbring J, Theobald M, Schetelig J, Kröger N; CLL subcommittee; Chronic Malignancies Working Party of the EBMT.
Bone Marrow Transplant. 2017 Dec 18. doi: 10.1038/s41409-017-0023-2. [Epub ahead of print]
Allogeneic Stem Cell Transplantation for Myelodysplastic Syndrome Patients with a 5q Deletion.
Garderet L, Ziagkos D, van Biezen A, Iacobelli S, Finke J, Maertens J, Volin L, Ljungman P, Chevallier P, Passweg J, Schaap N, Beelen D, Nagler A, Blaise D, Poiré X, Yakoub-Agha I, Lenhoff S, Craddock C, Schots R, Rambaldi A, Sanz J, Jindra P, Mufti GJ, Robin M, Kröger N.
Biol Blood Marrow Transplant. 2017 Nov 28. pii: S1083-8791(17)30863-7. doi: 10.1016/j.bbmt.2017.11.017. [Epub ahead of print]
Outcome after relapse of myelodysplastic syndrome and secondary acute myeloid leukemia after allogeneic stem cell transplantation: a retrospective registry analysis on 698 patients by the Chronic Malignancies Working Party of European Society of Blood and Marrow Transplantation.
Schmid C, de Wreede LC, van Biezen A, Finke J, Ehninger G, Ganser A, Volin L, Niederwieser D, Beelen D, Alessandrino P, Kanz L, Schleuning M, Passweg J, Veelken H, Maertens J, Cornelissen JJ, Blaise D, Gramatzki M, Milpied N, Yakub-Agha I, Mufti G, Rovira M, Arnold R, De Witte T, Robin M, Kröger N.
Haematologica. 2017 Nov 3. pii: haematol.2017.168716. doi: 10.3324/haematol.2017.168716. [Epub ahead of print]

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Bone Marrow Transplantation publishes high quality, peer reviewed original research and reviews that address all aspects of basic biology and clinical use of haemopoietic cell transplantation.
The journal also covers all aspects of the research and treatment of transplant-related complications and consequences including quality of life and psychological issues. Basic research studies on topics of relevance are also covered.

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Read the top articles from 2017…
Use of haploidentical stem cell transplantation continues to increase: the 2015 European Society for Blood and Marrow Transplant activity survey report
J R Passweg, H Baldomero et al
PD-1 checkpoint blockade in patients with relapsed AML after allogeneic stem cell transplantation
J C Albring, S Inselmann et al
Impact of drug development on the use of stem cell transplantation: a report by the European Society for Blood and Marrow Transplantation (EBMT)
J R Passweg, H Baldomero et al
Antibiotic-mediated modification of the intestinal microbiome in allogeneic hematopoietic stem cell transplantation
J Whangbo, J Ritz & A Bhatt
Next-generation sequencing indicates false-positive MRD results and better predicts prognosis after SCT in patients with childhood ALL
M Kotrova, V H J van der Velden et al
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