Nico Gagelmann Profile picture
@TheEBMT_Trainee co-chair. Hematology. Medicine. Science. Train. Think. Implement an integral actuality.

Nov 10, 2022, 16 tweets

A phrase many of us have used:
"Allogeneic BMT is the only curative treatment for various diseases but associated with morbidity/mortality."

Evidence & local practice are often heterogeneous. Can this year's @ASH_hematology meeting guide us?

🧵with #ASH22 abstracts

#MedTwitter

Mini intro to BMT:
-replaces damaged blood or bone marrow cells with healthy ones from donor
-source: peripheral blood or marrow (usually hip)
-matched related, matched unrelated (MUD), mismatched related, mismatched unrelated donors

In that spirit: go register & save lives!
/1

Let's categorize the #ASH22 thread chronologically along the path to and from allogeneic BMT:
-to transplant or not to transplant?
-donors
-conditioning
-graft-versus-host disease (GVHD)
-follow-up
Focus on adults, not a pediatrician. Respect to all in #Pediatrics btw!
/2

To BMT or to not BMT? part 1 #ASH22
-plenary
-chemo (high-dose cytarabine and mitoxantrone) before BMT with no survival advantage
-watchful waiting followed by sequential conditioning and BMT with comparable survival
-start donor search at diagnosis! ash.confex.com/ash/2022/webpr…
/3

To BMT or to not BMT? part 2 #ASH22
-randomized trial, n=245
-BMT is superior to non-BMT consolidation treatment for leukemia-free survival in elderly acute myeloid leukemia patients in first complete remission, 5year follow-up ash.confex.com/ash/2022/webpr…
/4

Which donors? #ASH22
-study from @CIBMTR
-significant relapse reduction with younger MUD versus older matched sibling donor
-higher treatment-related mortality with younger MUDs has decreased in recent years ash.confex.com/ash/2022/webpr…
/5

Which conditioning? #ASH22
-novel regimen with fludarabine, cyclophosphamide, rituximab in severe aplastic anemia
-0% non-relapse mortality and 100% disease-free survival with low GVHD rates at 1-year post-transplant ash.confex.com/ash/2022/webpr…
/6

Which GVHD prophylaxis? part 1 #ASH22
-@CIBMTR
-no significant difference in late graft failure rates when utilizing a haploidentical versus MUD with postBMT cyclophosphamide in patients undergoing reduced intensity BMT for AML, ALL or MDS ash.confex.com/ash/2022/webpr…
/7

Which GVHD prophylaxis? part 2 #ASH22
-@TheEBMT study
-methotrexate + calcineurin inhibitor prophylaxis was associated with favorable early survival and treatment-related mortality in patients with chronic myeloid malignancies or secondary AML ash.confex.com/ash/2022/webpr…
/8

Which GVHD prophylaxis? part 3 #ASH22
-using abatacept compared with ATG or postBMT cyclophosphamide appears to improve survival
-compelling results but validation and longer follow-up needed ash.confex.com/ash/2022/webpr…
/9

How to treat GVHD? #ASH22
-ruxolitinb (5 mg/day) + methylprednisolone (1 mg/kg) as effective first-line therapy
-high overall response rate
ash.confex.com/ash/2022/webpr…
/10

Maintenance after BMT? #ASH22
-pilot study of enasidenib maintenance therapy resulted in promisinge survival outcomes in AML patients carrying IDH2 mutations
-however, treatment delay and dose reductions were common
ash.confex.com/ash/2022/webpr…
/11

How to care after? #ASH22
-compared to usual care, shared care with local providers led to improved quality of life
-results a no-brainer but possibly for US system quite relevant
->in care, we need to take into account different healthcare systems! ash.confex.com/ash/2022/webpr…
/12

High-risk & long-term outcome? #ASH22
-BMT is associated with improved long-term outcomes in patients with TP53 AML
-significantly better among patients who were in complete remission at day 100 post BMT or had cGVHD
-should be offered to eligible patients ash.confex.com/ash/2022/webpr…

In conclusion, timing of BMT is everything and disease control the most important factor for success. We have new options for GVHD, time for large comparative trials to finally standardize our BMT community. Still, let's not forget the different healthcare realities we live in.

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