Questions List

The technology worked perfect even at teh end of the world like Chile Thank YouPosted onNovember 15, 2020 5:55 pm

Thank you. The speakers and the chats very interesting and entertaining. Posted onNovember 15, 2020 5:52 pm

Thanks and was a great session...AustinPosted onNovember 15, 2020 5:38 pm

Fabian-Did your patient with AML with SBDS mutations have any of the clinical features of SDS eg short stature, pancreatic insufficiency in retrospect? Do you know the TP53 mutation status at presentation with AML? Alan WarrenPosted onNovember 15, 2020 5:35 pm

Question for Lucy Godley: Would you comment on the management of patients who are in remission at the time of their germline variant predisposing to hematologic malignacy is uncovered? E.g., do they warrant pre-emptive transplant? Posted onNovember 15, 2020 5:15 pm

1) In your molecular tumour profiling group, what is your take on confirming the inherited nature of the variant (for eg DDX41) on skin biopsy vs sample after induction (in remission)? Totally understand skin is ideal!! 2) BM vs Blood for monitoring asymptomatic carriers of germline predisposing genes, for acquired mutations or cytogenetic clones. Great Talk as usual... AustinPosted onNovember 15, 2020 5:12 pm

@Sten Erik- Nice talk. Did all the 15/16 patients have the same gene mutations detected in bulk BM prior to allograft? Did presence of such mutations in either bulk or HSPCs prior to allograft increase the risk of relapse post HSCT? Thanks AustinPosted onNovember 15, 2020 4:56 pm

Question from Lucio Luzzatto to Matthew Porteus. The 30% Hb S 'correction' that you quoted is based - I believe - on HSCT mixed chimerism data, whereby autologous ((SS) cells compete with donor cells in hematopoietic reconstitution. With your gene editing approach the corrected cells are autologous: therefore do you think that, by not suffering competition, they may be more effective in repopulating the marrow? . Posted onNovember 15, 2020 4:48 pm

We hope soPosted onNovember 15, 2020 4:36 pm

We are ready to share in this trialPosted onNovember 15, 2020 4:34 pm

The question re innate immunity was from LucioPosted onNovember 15, 2020 4:04 pm

Complement-related innate immunity is important in the first years of life. This is a further reason to avoid targeting C3 in pregnant women in order the baby to safeguardPosted onNovember 15, 2020 4:01 pm

Given the autoimmune phenomena (e.g. glomerulonephritis, SLE) associated with congenital C3 deficiency, is there any cause for concern with the novel C3 inhibitor compounds? especially in the context of SAA-associated PNH? Thank you very much for the fascinating discussion (Giovanni Del Borrello, Gaslini institute, Genoa)Posted onNovember 15, 2020 3:50 pm

30. y.o male with PNH who received eculizumab and then matched sib transplant, with subsequent graft loss and complete autologous recovery. Now completely asymptomatic with normal WBC/ Hb except for thrombocytopenia around 40 K and a PNH clone around 10%. How would you follow and would you advise prophylactic anticoagulation or any other interventions ? K. Nadiminti, USAPosted onNovember 15, 2020 3:44 pm

What about minor PNH clones without hemolysis? Would you still recommend primary prophylaxis with anti-complement therapy? Posted onNovember 15, 2020 3:42 pm

To Antonio and Regis: What do with pregnancy in context of the non Ecu-treatments? Patient on Ravulizumab => back to Ecu? What to do with more proximal complement-inhibitors in unplanned pregnancys? Expected risks? With best regards Britta HöchsmannPosted onNovember 15, 2020 3:40 pm

Comment. from Carlo Dufour Great talks everybody! Another excellent session!Posted onNovember 15, 2020 3:36 pm

To the full panel: How do you handle infectious prophyilaxis in PNH patients on anticomplement therapy (PNH clone > 30%) with moderate aplastic anemia on chronic cyclosporine treatment? Thank you! (O. Naveiras, Lausanne, Switzerland)Posted onNovember 15, 2020 3:19 pm

The second case still showed high retics and high LDH on APL2 despite improvement in Hgb. What are your thoughts on why these did not normalize?Posted onNovember 15, 2020 3:13 pm

Dr. Hillmen: What is your approach on whether providing prophylactic anticoagulation to patients who can not access or have a major contraindication to complement inhibition and have extremely large clones (eg our case: 99% PNH clone on 69yo patient with immune-deficiency, complex infectious history and no history of thrombosis, full transfusison dependency) ?Posted onNovember 15, 2020 3:12 pm

************** session 8 ***************Posted onNovember 15, 2020 2:44 pm

Sixteen year old with PNH presented with severe pancytopenia. Moderate response to Eculizumab Hb max; 10-g. and plts max: 70K. Developed EVH. Steroid moderate response in 2 weeks, rise in Hb 2-g and rise in plts 30K. Then started losing the response. Currently Hb: 8.3, plts: 44K. NGS panel for MDS and AA was negative in the beginning. Her BMA 90% cellularity. Transplant is offered. We asked for APL2 awaiting response from APELLIS. Q: Do you agree with APL2 treatment? We are tapering the steroid dose now, which she is on 1-mg/kg/d currently. Do you recommend EPO, Eltrombopag for this patient? How concerned are you if she starts on APL with respect to infections? Thank you. Posted onNovember 15, 2020 2:39 pm

Sixteen year old with PNH presented with severe pancytopenia. Moderate response to Eculizumab Hb max; 10-g. and plts max: 70K. Developed EVH. Steroid moderate response in 2 weeks, rise in Hb 2-g and rise in plts 30K. Then started losing the response. Currently Hb: 8.3, plts: 44K. NGS panel for MDS and AA was negative in the beginning. Her BMA 90% cellularity. Transplant is offered. We asked for APL2 awaiting response from APELLIS. Q: Do you agree with APL2 treatment? We are tapering the steroid dose now, which she is on 1-mg/kg/d currently. Do you recommend EPO, Eltrombopag for this patient? How concerned are you if she starts on APL with respect to infections? Thank you. Posted onNovember 15, 2020 2:38 pm

I am from Egypt pediatrics treating children with BMF want to share in trials in aplastic anemia Posted onNovember 15, 2020 2:35 pm

I am from Egypt pediatrics treating children with BMF want to share in trials in aplastic anemia Posted onNovember 15, 2020 2:35 pm

I am from Egypt pediatrics treating children with BMF want to share in trials in aplastic anemia Posted onNovember 15, 2020 2:35 pm

I am from Egypt pediatrics treating children with BMF want to share in trials in aplastic anemia Posted onNovember 15, 2020 2:35 pm

I am from Egypt pediatrics treating children with BMF want to share in trials in aplastic anemia Posted onNovember 15, 2020 2:35 pm

I am from Egypt pediatrics treating children with BMF want to share in trials in aplastic anemia Posted onNovember 15, 2020 2:35 pm

I am from Egypt pediatrics treating children with BMF want to share in trials in aplastic anemia Posted onNovember 15, 2020 2:35 pm

For our protocol. Andrea describes the goal, but for now needs a center to distribute drugs, and only Jamaica participating as of now .Posted onNovember 15, 2020 2:33 pm

About supportive therapy, what anti fungal/anti-bacterial prophylaxis are you using in SAA with N count < 200? Thank you (Giovanni Del Borrello) Posted onNovember 15, 2020 2:32 pm

is it possible to inter in this trialPosted onNovember 15, 2020 2:31 pm

is it possible to inter in this trialPosted onNovember 15, 2020 2:31 pm

Posted onNovember 15, 2020 2:30 pm

Great discussion, great people on the podium ! :-) Posted onNovember 15, 2020 2:19 pm

Is Eltromnobag and cyclosporine oral treatment in developing counteries as 1st line in severe aplastic anemia in children unable to get IST ATG/cyclosporine and with no MRD ? Posted onNovember 15, 2020 2:18 pm

Follow up - I do have her on single agent eltrombopag now. So far no response. J Grossman - CanadaPosted onNovember 15, 2020 2:09 pm

To Dr Lefloque--you should test in the immune AA mouse model!Posted onNovember 15, 2020 2:04 pm

for moderate aplastic anemia, what would be the appropriate time to consider transplant ? K. Nadiminti - Wisconsin, USAPosted onNovember 15, 2020 2:04 pm

To everybody: do you agree that after triple therapy refractory patients (younger than 50? or 40? or 60?) should move to SCT with the best available donor? AntonioPosted onNovember 15, 2020 2:04 pm

Posted onNovember 15, 2020 2:03 pm

to Britta: What is the estimated incidence of mAA in Europe (as apposed to sAA) ? Thanks TimPosted onNovember 15, 2020 2:03 pm

My question to Britta Höchsmann. What is the follow-up schedule of patient with Moderate Aplastic Anemia to investigate the risk of evolution in Severe Aplastic Anemia or MDS? Gianluca Dell'Orso, GenoaPosted onNovember 15, 2020 2:02 pm

Carmem Bonfim: excellent session. Thank you all. Regis, I saw that you included some patients between 15 and 18 years old in the RACE trial. What about the response of eltrombopag in children and adolescents in this trial? Can you speculate why they may not have the same response as adults?Posted onNovember 15, 2020 2:02 pm

To Regis: how much follow up is required by EMA before we can state that Clonal evolution rates are not different between the two arms in the RACE trial ? Thanks Tim BrümmendorfPosted onNovember 15, 2020 2:01 pm

Conditioning in children MSD Cy/Flu versus CY/ ATG Posted onNovember 15, 2020 2:01 pm

I have a 71yo patient diagnosed with aplastic anemia who also had a concomitant monoclonal B cell lymphocytosis on her pre treatment biopsies. She failed to respond to ATG/ CsA (we can't get E-pag up front here). Could the MBL be playing a role here? Would there be any point in giving ritux? Jennifer Grossman Posted onNovember 15, 2020 2:00 pm

Is the Regeneron compound already in clinical development ? Superb session ! Thanks Tim BrümmendorfPosted onNovember 15, 2020 1:59 pm

What is the treatment algorithm for moderate aplastic anaemia in paediatrics: upfront BMT? - Josu de la FuentePosted onNovember 15, 2020 1:59 pm

To Andrea Bacigalupo: what is the actual role of haplo-SCT in the current treatment algorithm? Actually we still have concerns to use a 9/10 UD in the setting of second-line treatment, but here we see that we can use haplo even upfrontPosted onNovember 15, 2020 1:56 pm

Dr Dufour: In pediatric patient, who has not donor to BMT, you would recommend the use of IS + eltrombopag although its effectiviness has not been shown to be effective as in adults?Posted onNovember 15, 2020 1:55 pm

Rosario Notaro for Dr. Le Floc'h very nice approach. You have shown data after about 6 months of follow up. What about longer follow up?Posted onNovember 15, 2020 1:54 pm

RACE trial: Follow up beyond month 6: Proportion of patients who are off Eltrombopag - and in stabile remission. Hubert SchrezenmeierPosted onNovember 15, 2020 1:23 pm

Bravo, Regis! Terrific talk and satisfying conclusion, of course. Heroic and successful effort by you and Antonio. N.Posted onNovember 15, 2020 1:21 pm

RACE Trial: are there already data available on overall survival ? Hubert SchrezenmeierPosted onNovember 15, 2020 1:20 pm

Posted onNovember 15, 2020 1:20 pm

*********** Session ************ Posted onNovember 15, 2020 1:05 pm

Would you use APL2 in acute situations such as thrombosis in naive patients?Posted onNovember 15, 2020 1:00 pm

Great talk and discussion guys! talk soon RégisPosted onNovember 15, 2020 12:59 pm

followup Q- how would you manage this new breakthrough? advance the C3inh or extra dose of C5inhPosted onNovember 15, 2020 12:53 pm

What is the role of >90% red cell clone in BTH in the context of proximal inhibitors? Is this the major reason for BTH and its serious compl? Thanks AustinPosted onNovember 15, 2020 12:50 pm

Those Hb curves in the two groups are more than impressive: wonderful news! Any problem arising in patients who must by now have nearly 100% PNH red cells?Posted onNovember 15, 2020 12:42 pm

*********** Day 3 **********Posted onNovember 15, 2020 11:34 am

************ Day 3 ***********Posted onNovember 14, 2020 7:05 pm

Aikaterina Poulaki: have you tested how modulation of Redox status in high blast patients (eg. FCCP, DNP) affects their capacity of differentiation ? O. Naveiras (Lausanne, Switterland)Posted onNovember 14, 2020 5:56 pm

Marc Raaijmakers to Alan, Thanks for an excellent talk. What are your thoughts on how the series of events caused by cGAS-STING-NLRP3 activation may contribute to competitive advantage and clonal evolution? Posted onNovember 14, 2020 5:49 pm

Dr. Panse, thank you very much for the presentation and valuable work behind it. There is no doubt about the utility of the tool in clinical trials. How do you propose using it for every-day clinical practice. Have you tested it locally for outpatient treatment choice or adaptation? Olaia Naveiras (Lausanne, Switzerland)Posted onNovember 14, 2020 5:35 pm

@Jens Panse- how often would you recommend doing the QLG-AA/PNH, for AA and PNH? Is there any specific critical time points for doing this in particular for AA. Many thanks AustinPosted onNovember 14, 2020 5:34 pm

can you comment on the few patients harboring meanigful PNH clones only in granulocytes (and possibly platelets)? and their association with thrombosis? antonioPosted onNovember 14, 2020 4:37 pm

What is the selective pressure for PIG-A somatic mutations in the 30% of PNH patients with no concurrent BM failure? Posted onNovember 14, 2020 4:36 pm

Wonderful talk-latest insight into the identity of the GP1 linked target in aplastic anemia? Alan WPosted onNovember 14, 2020 4:34 pm

An amazing lecture. Thank you Posted onNovember 14, 2020 4:28 pm

Many patients have a stable balance between type II and III cells over long periods of time. Why is there not a selective increase in Type III cells compared to Type II as the partially deficient cells express some GPI-molecules but the Type III don't? (Pete Hillmen)Posted onNovember 14, 2020 4:27 pm

Sorry, I forgot to identify myself, Tim Brümmendorf, This for the answer !Posted onNovember 14, 2020 4:24 pm

@Lucio Luzatto: great talk, thank you very much ! Yesterday, we heard of a joint c-MPL and PiGV mutation in another PNH/MPN overlap syndrome, where mutant c-MPL was suspected to be the driver of clonal evolution of the PIGV-clone. Have you seen c-MPL mutated MPN/PNH overlaps also in association with classical PIGA-mutated PNH ?Posted onNovember 14, 2020 4:17 pm

***********************Posted onNovember 14, 2020 3:54 pm

Why no hemolysis with PIGM mutations?Posted onNovember 14, 2020 3:54 pm

Can you compare C3 and/or factor B /factor D inhibitors with rare genetically determined deficiency of these proteins?Posted onNovember 14, 2020 3:34 pm

dr Peffault In pediatrician patient <18 years, who has not donnors for transplant and use IS, Do you use hATG + CsA whithout Eltrombopag?Posted onNovember 14, 2020 3:34 pm

In young aplastic anemia patients <40yo relapsing during Ciclosporine tapering should alternative donors be considered (if no MUD)? Posted onNovember 14, 2020 3:29 pm

For a young patient aged 15-20 with SAA without MSD would u choose IST + ELT or rapidly available 10/10 MUD? N BuchbinderPosted onNovember 14, 2020 3:28 pm

With patients on eltrombopag and ciclosporin do you have a preference which drug to wean first?Posted onNovember 14, 2020 3:25 pm

In patients treated for severe aplastic anemia with IST, have you seen significant expansion of PNH clone enough to cause hemolytic anemia? If so, have you treated such patients with eculizumab? - Abhishek Mangaonkar (Mayo Clinic - Rochester)Posted onNovember 14, 2020 3:23 pm

Great Symposium, again !!! TimPosted onNovember 14, 2020 3:20 pm

@Antonio: any infectious complications under LNP023 ?Posted onNovember 14, 2020 3:15 pm

Regis: When do you start ELT first line outside clinical trials now: day 1 or day 14 ? Thanks TimPosted onNovember 14, 2020 3:00 pm

********* Sympo ********Posted onNovember 14, 2020 2:56 pm

In pediatrics patients, <18 years do you recomend use Eltrombopag?Posted onNovember 14, 2020 2:35 pm

For Dr.Notaro: can you further speculate on the data showing GPI-anchor as target of the immune-mediated attack in AA (and PNH)?Posted onNovember 14, 2020 2:34 pm

Great session and great discussion alltogether !!! Congrats, Tim !Posted onNovember 14, 2020 2:30 pm

To Austin (and everybody): somatic mutations are frequent in AA, but emergind data clearly show that in addition to "myeloid cancer genes" they may affect "immune-privilege genes", such as PIGA or HLA. Can we conclude that we are looking at standard rate of mutations, keeping in mind that these 2 types are simply the ones that we may detect because they lead to expansions of mutant clones? Thus, are we overestimating the risk of MDS or leukemia by simply looking to NGS data? AntonioPosted onNovember 14, 2020 2:13 pm

For Austin: If I recall correctly, adenosine deaminase 2 is measured in plasma and is secreted from lymphocytes. Therefore, are lower levels observed in patients with SAA and marked lymphopenia regardless of ADA2 germline pathogenic variants? Alison Bertuch, Houston Posted onNovember 14, 2020 2:12 pm

Is there a specific PNH clone threshold (%) in bone marrow failure presentation that changes your management of immune aplastic anaemia: 1. use ATG rather than alemtuzumab in a BMT or not to consider IST treatment 2. adopt an alternative donor BMT in a patient without a matched related or unrelated donor What is the consensus of the panel? Josu de la FuentePosted onNovember 14, 2020 2:11 pm

@Austin: when/why do you use PCR (as opposed to flow-FISH) to exclude TBD at diagnostic work-up of AA ? Thanks Tim BrümmendorfPosted onNovember 14, 2020 2:00 pm

@Rosario: excellent talk ! Yesterday, we had an exciting poster presented (poster#21) that showed the coincidence of a PIGV mutation and a driving JAK2-mutation in a patient with concomitant PNH and ET. The idea was that JAK2 was driving the PIGV clone. Have you seen this as a driving event in PIGA-based PNH as well ? Thanks Tim BrümmendorfPosted onNovember 14, 2020 1:57 pm

For panel (from Olaia Naveiras, Lausanne Switzerland): Have you observed a clinical overlap of SAA and late onset telomere biology disorders? Else how would you interpret this case? Young (29yo) patient with classic presentation of SAA (with autoimmune hepatitis) and partial response to hATG/Cs/eltrombopag (full response for neutrophils), Telomere attrition was detected and confirmed in the parents. No donor available. He remained fully dependent on platelet transfussions and partially transfussion-dependent for RBCs. He was started in danazol. Platelet transfussion independency was reached 18 months after danazol introduction. Ciclosporine and eltrombopag have not yet been fully tampered due to neutrophil dependency. Cytogenetics are normal at the last BM control (24 months after danazol introduction). Thank you for the great session!Posted onNovember 14, 2020 1:55 pm

To Neal Young: Thank you for the outstanding review! Why - if evasion of IFNg competition for the MBL-receptor binding by eltrombopag is one of the major mechanisms of action in AA patients does romiplostim also seem to work, even in patients not responding to eltrombopag. And is the timing of the addition of eltrombopag to IST essential? Jens Panse Posted onNovember 14, 2020 1:54 pm

@Neal: excellent talk ! Do you thinks the the lower risk of clonal evolution for patients under IST + ELT as opposed to ELT monotherapy (and probably also IST alone) would also hold true fror the combination of CSA + ELT (compared to ELT or CSA mono therapy) in elderly, frail AA patients (who are in any way at higher risk of clonal evolution ? Thanks Tim BrümmendorfPosted onNovember 14, 2020 1:40 pm

What would you recommend for a 25 y/o male who relapsed 2 years post sib allogeneic HCT, did not respond to salvage with eltrombopag and cyclosporine and had a second allogeneic HCT from the same sibling. no GVHD - prolong CSP duration? add sirolimus? Posted onNovember 14, 2020 1:33 pm

Is it possible to accelerate clonal evolution with immunosuppressive therapy in severe aplastic anemia? (Abhishek Mangaonkar - Mayo Clinic)Posted onNovember 14, 2020 1:23 pm

Thanks Neal wonderful and inspiring talk as usual! You clearly showed the role of immune-mediated damage and the benefit of HSC-targeted therapy with EPAG. 1. Can you further comment on the trend of using ATG-free regimens (fine during the covid pandemic… but I see the risk of less robust and curative responses in the real life). 2. Really impressed by the concept of IST-boost with sirolimus: any easy biomarker to dissect between patients who may benefit from IST-boost vs those who can better benefit from extended EPAG? Antonio RisitanoPosted onNovember 14, 2020 1:23 pm

********** Session 5 *************Posted onNovember 14, 2020 1:08 pm

You are quite right: I ave patients in Tanzania and I ahve requested eculizumab on compassionate basis but to no avialPosted onNovember 14, 2020 1:05 pm

great symposium in a very important topic, congratulations !!!!Posted onNovember 14, 2020 1:03 pm

Is the questionnaire already used by physicians regularly? (Magdalena Howe)Posted onNovember 14, 2020 12:50 pm

testPosted onNovember 14, 2020 12:49 pm

no question, just a feedback: Liebe Frau Burmester, eine sehr schöne klare Darstellung, die anderen Kollegen sicherlich die Relevanz der Zusammenarbeit näher bringt. Ganz liebe Grüße Britta Höchsmann Posted onNovember 14, 2020 12:42 pm

Could you comment on comparative infectious risk of the different strategies and specially of anti factor D treatment, namely for a PNH patient with high infectious risk, non transplant eligible, and with moderate neutropenia and thrombocytopenia. Thank youPosted onNovember 14, 2020 12:27 pm

Which PROM questionnaire is being used in COMMODORE ?Posted onNovember 14, 2020 12:25 pm

how many mints to startePosted onNovember 14, 2020 11:06 am

********* Day 2 **********Posted onNovember 14, 2020 10:46 am

Very interesting!! New mechanism of clonal selection I think there is no oncogene in most cases of PIGA mutations : the immune escape is correct i Most patients with ONH do NOT evolve to MDS/AML Posted onNovember 13, 2020 5:21 pm

************* Posters Posted onNovember 13, 2020 4:26 pm

Very well done ! excellent session Best to all Carlo Posted onNovember 13, 2020 4:12 pm

do drugs like pirfenidone and nintedanib have a role in STS ILD? Posted onNovember 13, 2020 4:02 pm

For HSCT, I have a concern with using siblings who do not carry the TBD mutation found in their affected sibling, but have short telomeres <1%. What do you think?Posted onNovember 13, 2020 3:51 pm

to Alison, why do you think very long telomeres are associated with development of cancer? Posted onNovember 13, 2020 3:49 pm

Carmem Bonfim Curitiba brazil - Do you give any treatment to prevent/treat pulmonary fibrosis after transplant? is there anything effective to prevent this complications? Posted onNovember 13, 2020 3:34 pm

Do you think solid cancer surveillance is indicated in patients with DC? There is a significant increased risk, but the chance per organ is small. Marjolijn JongmansPosted onNovember 13, 2020 3:33 pm

Excellent talks my friends! Question 1 to all from Carlo Dufour. We know that rate of growth is not uniform in pediatric age (top first 3 years , slows down until puberty and that rises again at puberty. Has any of you observed fluctuation of TL, reflecting these different rates, in normal control in ages 0-21 yrs? Question 2. from Carlo to all. have you noted differences in TL measurement in MNC vs different lymphocyte subset?Posted onNovember 13, 2020 3:33 pm

To Indejeet Dokal Can you tease out phenotypes that relate to the ribosome defect vs telomere shortening for the relevant genesPosted onNovember 13, 2020 3:17 pm

Can you elaborate a bit on the anticipation of short telomeres from generation to generation? Why are these shorter in the next generation (Marrie Bruin, Princess Maxima Center) Posted onNovember 13, 2020 3:13 pm

Can telomere length measured by flow FISH determine a diagnostic boundary for DC, even if age adjusted, that allows diagnosis in BM failure even in NGS does not determine a genetic pathogenic mutation? I assume that this requires <0.4th centile but where is the boundary? - Josu de la FuentePosted onNovember 13, 2020 2:53 pm

****** Session 4 **********Posted onNovember 13, 2020 2:53 pm

Are eIF6 mutations of clonal origins or independently develop in multiple HSC Christian BrendelPosted onNovember 13, 2020 2:31 pm

For Yigal Dror: Does Leucine act differently (is less effective) in younger vs. older patients with DBA ?Posted onNovember 13, 2020 1:53 pm

For Yigal Dror: What specific adult, long-term monitoring do you recommend for non-severe, non-transplanted DBA/SDS (e.g. frequency of BM monitoring and of non-hematological cancer surveillance)? Posted onNovember 13, 2020 1:53 pm

Beautiful talk by Alan Warren: has eIF6 (in analogy to other eIFs) also a role for initiation of translation itself (and if so, is this affected by the mutations as well) or only for ribosomal assembly ? Tim BrümmendorfPosted onNovember 13, 2020 1:36 pm

Do eIF6 mutations reduce or eliminate MDS/AML risk. Christian Brendel Posted onNovember 13, 2020 1:35 pm

For Vijay. Two questions from Olaia Naveiras (Lausanne, Switzerland) - In your DBA patient cohort, could you see a relationship of global transcipt reduction and severity of the DBA phenotype? - Does corticoid treatment rescue global transciptome or qualitatively increases GATA1 expression only ?Posted onNovember 13, 2020 1:29 pm

********* Session 3 ************Posted onNovember 13, 2020 12:53 pm

Thank you all!Posted onNovember 13, 2020 12:04 pm

Thank you very muchPosted onNovember 13, 2020 11:55 am

Thank you very muchPosted onNovember 13, 2020 11:55 am

Is early transplantation once BMF appear is better or late to delay clonal disease appearance? Posted onNovember 13, 2020 11:49 am

Posted onNovember 13, 2020 11:35 am

These were excellent talks: understanding and treating FA has come a long way since I diagnosed my first patient with this condition. THANK YOUPosted onNovember 13, 2020 11:35 am

Is Radiation small dose in conditioning in pediatrics is tolerated better than the non radiation conditioning ?Posted onNovember 13, 2020 11:34 am

Posted onNovember 13, 2020 11:31 am

Is MTX prophylaxis of n GVHD is better to be avoided in Fanconi?Posted onNovember 13, 2020 11:31 am

Questions from Marc Raaijmakers to Juan: Juan: can you speculate/elaborate on the fate of non-corrected hematopoietic stem/progenitor cells and their risk for leukemic evolution? Posted onNovember 13, 2020 11:18 am

How do the engraftment kinetics and clonal dynamics of gene-therapy-modified FA cells compare to those observed in gene therapy studies for other diseases?Posted onNovember 13, 2020 11:13 am

Could addition of busulfan 2 mg/kg to conditioning regimens further reduce the risk of relapse in FLAG aplastic phase transplantation in transformed FA patients? (Josu de la Fuente)Posted onNovember 13, 2020 11:13 am

Is there are role for 9/10 BMT in Fanconi anaemia and if so, what should the conditioning regimen be? (Josu de la Fuente)Posted onNovember 13, 2020 11:11 am

Question from Marc Raaijmakers to Jean Soulier: Jean: is the occurence of 1q+ associated with a (transient) amelioration/ improvement in hematopoiesis (peripheral blood numbers)? (since it suppresses TP53 signaling via MDM4)? In general: Is temporal improvement in hematopoiesis observed in FA, and, if so, is it associated with later leukemic evolution?Posted onNovember 13, 2020 10:41 am

******** Session 2 ************* Posted onNovember 13, 2020 10:27 am

IS aquired Fanconi anemia is now a possibility ?Posted onNovember 13, 2020 10:01 am

Has formaldehyde have a role in ribosomal biogenesis disruption?Posted onNovember 13, 2020 9:59 am

To all: Fantastic talks!Posted onNovember 13, 2020 9:57 am

Question from Marc Raaijmakers to Markus Markus: Given the wide repertoire of inflammatory molecules that can be secreted by neutrophils/myeloid cells, are we looking at the tip of the iceberg focusing on IL1 and should we ultimately aim for targeting 'master regulators of inflammation' to attenuate aging? If so, what would be candidates?Posted onNovember 13, 2020 9:57 am

Question for KJ : what type of cancer do the Aldh2-5-/- mice develop ? Jean SoulierPosted onNovember 13, 2020 9:52 am

Question to Markus Manz: do you see a potential future role for anti-IL1R treatement in fore example (inherited) bone marrow failure syndromes? Sincerely, Anna Aalbers, fellow hematology.Posted onNovember 13, 2020 9:49 am

Question from Marc Raaijmakers to Ashvind: Ashvind: Can you speculate on the mechanism behind the association between positive blood cultures early after transplant and graft failure? Posted onNovember 13, 2020 9:49 am

Question from Marc Raaijmakers to Ketan Patel: Ketan: Is it reasonable to hypothesize that differing levels of aldehydes play a role in human bone marrow failure, outside of the Fanconi context? For example, in aging associated failure and CHIP? Can we measure aldehydes in humans?Posted onNovember 13, 2020 9:46 am

Thank you for the great presentation. Which antibiotics were used to revert the IL-1b mediated myeloid bias? Did you control for changes in myeloid bias impossed by the antibiotics themselves in vitro, as antibiotics and specially tetracyclines can induce mild mitochontrial stress, which we have been associated to HSC rejuvenation. With best regards. Olaia NaveirasPosted onNovember 13, 2020 9:34 am

Great talk! Are any of the congenital anomalies of FA found in the children with BMF with cachexia? Alison BertuchPosted onNovember 13, 2020 9:16 am

To Patel (from Miriam Erlacher, Freiburg): Did Patients with mutations in both ALDH2 and ADH5 show increased treatment toxicity during conditioning for HSCT?Posted onNovember 13, 2020 9:14 am

Beautiful data. Thank you. Have you looked at how Netrin expression changes during the revascularization that occurs after chemo- or radiation induced aplasia? Olaia NaveirasPosted onNovember 13, 2020 8:59 am

Session 1 ********* Posted onNovember 13, 2020 7:59 am

asdasdPosted onNovember 12, 2020 8:58 pm

hey hey!Posted onNovember 7, 2020 3:36 pm

HiPosted onOctober 25, 2020 10:49 am

helloPosted onOctober 25, 2020 10:49 am

Test questionPosted onOctober 13, 2020 5:57 pm

HelloPosted onOctober 13, 2020 5:57 pm