Citations 2
Allogeneic haematopoietic cell transplantation in advanced systemic mastocytosis in the new era: A CIBMTR study
Üstün, Celalettin , Zhang, Mei‐Jie , Peterson, Andrew C. , Baek, Alexander, Coltoff, Alex, Gandhi, Arpita , Ho, Vincent T., Lin, Adam, Gowda, Lohith , Mensah, Felix, Nadiminti, Kalyan , Trunk, Andrew D. , Uberti, Joseph P., Marcucci, Guido , Akın, Cem , Pullarkat, Vinod , Juckett, Mark, Saber, Wael , Burns, Linda J. , Alkhateeb, Hassan B , Borthakur, Gautam , Nadiminti, Kalyan V , Nishihori, Taiga , Trunk, Andrew , Hamadani, Mehdi , Oran, Betul , Peterson, Andrew, Agha, Mounzer , Alkhateeb, Hassan B. , Chhabra, Saurabh , Coltoff, Alex, Lima, Marcos de , Kassim, Adetola A , Lin, Adam, Borthakur, Gautam , DeAngelo, Daniel J. , McGuirk, Joseph P. , Nishihori, Taiga , Pantin, Jeremy , Gotlib, Jason , Akin, Cem , Hamadani, Mehdi , Valent, Peter , Grunwald, Michael R. , Oran, Betül
British Journal of Haematology | September 23, 2025 | Volume: 207 | Issue: 6 | Pages: 2486-2495
Summary We investigated the outcomes of patients with advanced systemic mastocytosis (AdvSM) undergoing allogeneic haematopoietic cell therapy (alloHCT) in the US. Twenty‐seven adult (median age, 58 years) patients with AdvSM received alloHCT from 2014 to 2021. Most patients were white (93%), male (63%) and had systemic mastocytosis (SM) with associated haematological neoplasm (SM‐AHN, 70%), received peripheral blood grafts (89%) and non‐myeloablative regimens. KIT mutation was detected in 14 of the reported 16 patients (87.5%). Post‐transplantation cyclophosphamide (PTCy) was used in approximately 50% of the cohort. Approximately 1 year after alloHCT, median bone marrow mast cell burden decreased from 15% pre‐alloHCT to 1.5% (range 0–8) at 1 year post‐alloHCT. The median serum tryptase decreased from 55 ng/mL (range 3–400 ng/mL) pre‐alloHCT to 18 ng/mL (range 0–481 ng/mL) at 1 year post‐alloHCT. At 1 year, progression‐free survival (PFS) and overall survival (OS) were 59% (95% confidence interval [CI], 41%–77%) and 74% (95% CI, 56%–89%) respectively. In the subset of patients with SM‐AHN, 1‐year PFS and OS were 74% (52%–91%) and 79% (95% CI, 58%–94%) respectively. The cumulative incidence of relapse/progression for the entire population of patients with SM ( n = 27) was 4% (95% CI, 0–14) at 100 days and 15% (95% CI, 4–31) at 1 year. For the subset of patients with AHN, the cumulative incidence was 11% (95% CI, 1–28) at 100 days and 21% (95% CI, 6–42) at 1 year. Relapse/progression was the cause of death in six of 12 patients (5 died of AHN and 1 died of SM progression). Eighteen and nine patients received a KIT inhibitor before alloHCT and after alloHCT respectively. In addition, six patients received a KIT inhibitor for the treatment of relapse/progression of SM after alloHCT. Although it is difficult to delineate the exact effect of alloHCT and KIT inhibitors because of the size of the study in this era, the use of alloHCT and KIT inhibitors pre‐ and post‐ alloHCT is increasing. In the near future, larger studies are required to provide further insights into this subject.
Donor types and outcomes of transplantation in myelofibrosis: a CIBMTR study
Jain, Tania , Estrada‐Merly, Noel, Salas, María Queralt , Kim, Soyoung , DeVos, Jakob D. , Chen, Min , Fang, Xi , Kumar, Rajat , Elmariah, Hany , Aljurf, Mahmoud , Badar, Talha , Ballen, Karen K. , Bredeson, Christopher , DeFilipp, Zachariah , Farhadfar, Nosha , Farhan, Shatha , Gandhi, Arpita , Ganguly, Siddhartha , Hamilton, Betty K. , Inamoto, Yoshihiro , Jamy, Omer , Kharfan‐Dabaja, Mohamed A. , Krem, Maxwell M. , Lad, Deepesh , Malone, Adriana K. , Pawarode, Attaphol , Poel, Marjolein van der , Rizzieri, David A. , Savoie, Mary Lynn , Seo, Sachiko , Solh, Melhem , Üstün, Celalettin , Verdonck, Leo F. , Wirk, Baldeep , Bejanyan, Nelli , Jones, Richard J., Nakamura, Ryotaro , Scott, Bart L. , Saber, Wael , Gupta, Vikas , Salas, M Queralt , Elmariah, Hany , Bacher, Ulrike , Badar, Talha , Bhatt, Vijaya Raj , DeFilipp, Zachariah , Dholaria, Bhagirathbhai R , Farhan, Shatha , Hamad, Nada , Hamilton, Betty K , Inamoto, Yoshihiro , Jamy, Omer , Juckett, Mark , Kharfan-Dabaja, Mohamed A , Krem, Maxwell M , Al Malki, Monzr M , Malone, Adriana K , Murthy, Hemant S , Patel, Sagar , Perales, Miguel-Angel , Ringden, Olov , Rovó, Alicia , Savani, Bipin N , Savoie, Mary Lynn , Seo, Sachiko , Ustun, Celalettin , Verdonck, Leo F , Bejanyan, Nelli , Nishihori, Taiga , Nakamura, Ryotaro , Scott, Bart , Gupta, Vikas , Jain, Tania , Kim, Soyoung , Andrade‐Campos, Marcio , Agrawal, Vaibhav , Badawy, Sherif M. , Beitinjaneh, Amer , Bhatt, Vijaya Raj , Dholaria, Bhagirathbhai , Gergis, Usama , Grunwald, Michael R. , Hamad, Nada , Iqbal, Madiha , Liesveld, Jane L. , Malki, Monzr M. Al , Murthy, Hemant S. , Ortí, Guillermo, Patel, Sagar S. , Perales, Miguel‐Angel , Ringdén, Olle , Savani, Bipin N. , Wingard, John R. , Nishihori, Taiga , Oran, Betül
Blood Advances | June 25, 2024 | Volume: 8 | Issue: 16 | Pages: 4281-4293
10.1182/bloodadvances.2024013451
Abstract We evaluate the impact of donor types on outcomes of hematopoietic cell transplantation (HCT) in myelofibrosis, using the Center for International Blood and Marrow Transplant Research registry data for HCTs done between 2013 and 2019. In all 1597 patients, the use of haploidentical donors increased from 3% in 2013 to 19% in 2019. In study-eligible 1032 patients who received peripheral blood grafts for chronic-phase myelofibrosis, 38% of recipients of haploidentical HCT were non-White/Caucasian. Matched sibling donor (MSD)–HCTs were associated with superior overall survival (OS) in the first 3 months (haploidentical hazard ratio [HR], 5.80 [95% confidence interval (CI), 2.52-13.35]; matched unrelated (MUD) HR, 4.50 [95% CI, 2.24-9.03]; mismatched unrelated HR, 5.13 [95% CI, 1.44-18.31]; P < .001). This difference in OS aligns with lower graft failure with MSD (haploidentical HR, 6.11 [95% CI, 2.98-12.54]; matched unrelated HR, 2.33 [95% CI, 1.20-4.51]; mismatched unrelated HR, 1.82 [95% CI, 0.58-5.72]). There was no significant difference in OS among haploidentical, MUD, and mismatched unrelated donor HCTs in the first 3 months. Donor type was not associated with differences in OS beyond 3 months after HCT, relapse, disease-free survival, or OS among patients who underwent HCT within 24 months of diagnosis. Patients who experienced graft failure had more advanced disease and commonly used nonmyeloablative conditioning. Although MSD-HCTs were superior, there is no significant difference in HCT outcomes from haploidentical and MUDs. These results establish haploidentical HCT with posttransplantation cyclophosphamide as a viable option in myelofibrosis, especially for ethnic minorities underrepresented in the donor registries.