Standardizing Definitions of Hematopoietic Recovery, Graft Rejection, Graft Failure, Poor Graft Function, and Donor Chimerism in Allogeneic Hematopoietic Cell Transplantation: A Report on Behalf of the American Society for Transplantation and Cellular Therapy

Kharfan‐Dabaja, Mohamed A.; Kumar, Amit; Ayala, Ernesto; Aljurf, Mahmoud; Nishihori, Taiga; Marsh, Rebecca; Burroughs, Lauri M.; Majhail, Navneet S.; Al-Homsi, A. Samer; Al-Kadhimi, Zaid; Bar, Merav; Bertaina, Alice; Boelens, Jaap Jan; Champlin, Richard E.; Chaudhury, Sonali; DeFilipp, Zachariah; Dholaria, Bhagirathbhai; El‐Jawahri, Areej; Fanning, Suzanne R.; Fraint, Ellen; Gergis, Usama; Giralt, Sergío; Hamilton, Betty K.; Hashmi, Shahrukh K.; Horn, Biljana; Inamoto, Yoshihiro; Jacobsohn, David A.; Jain, Tania; Johnston, Laura; Kanate, Abraham S.; Kansagra, Ankit; Kassim, Adetola A.; Kean, Leslie S.; Kitko, Carrie L.; Knight‐Perry, Jessica; Kurtzberg, Joanne; Liu, Hien; MacMillan, Margaret L.; Mahmoudjafari, Zahra; Mielcarek, Marco; Mohty, Mohamad; Nagler, Arnon; Nemecek, Eneida R.; Olson, Timothy S.; Oran, Betül; Perales, Miguel Angel; Prockop, Susan E.; Pulsipher, Michael A.; Pusic, Iskra; Riches, Marcie L.; Rodriguez, Cesar; Romee, Rizwan; Rondón, Gabriela; Saad, Ayman; Shah, Nina; Shaw, Peter J.; Shenoy, Shalini; Sierra, Jorge; Talano, Julie; Verneris, Michael R.; Veys, Paul; Wagner, John E.; Savani, Bipin N.; Hamadani, Mehdi; Carpenter, Paul A.

doi:10.1016/j.jtct.2021.04.007

Cited by 91 publications

(88 citation statements)

References 15 publications

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“…Two patients have mixed chimerism at last follow-up but have normal enzyme and low psychosine levels compared with baseline. 24 Five children have normal immune function and require no transplant-related medications. One child (Patient 2), who is currently 58 months post-HCT, has needed intermittent immunosuppression for autoimmune hemolytic anemia.…”

Section: Resultsmentioning

confidence: 99%

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Benefits of newborn screening and hematopoietic cell transplant in infantile Krabbe disease

et al. 2022

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Infantile Krabbe Disease (IKD) can be treated with hematopoietic cell transplantation (HCT) if done during the first weeks of life before symptoms develop. To facilitate this, newborn screening (NBS) has been instituted in eight U.S. states. An application to add KD to the Recommended NBS Panel (RUSP) is currently under review. In this report, the outcomes of newborns with IKD diagnosed through NBS and treated with HCT are presented. The unique challenges associated with NBS for this disease are discussed including opportunities for earlier diagnosis and streamlining treatment referrals. This is a retrospective review of infants with IKD detected by NBS and referred for HCT. The timing from diagnosis to HCT were examined and both HCT and neurodevelopmental outcomes are described. Six infants were diagnosed and referred for HCT. Neurologic testing before HCT revealed evidence of active IKD in all infants. All underwent HCT between 24-40 days of age, successfully engrafted, and are alive 30-58 months later (median, 47.5 months). All are gaining developmental milestones albeit at a slower pace than unaffected age-matched peers. Gross motor function is most notably affected. NBS for these patients enabled early access to HCT, the only currently available treatment for infants with IKD. All children are alive and have derived developmental and neurologic benefits from timely HCT. Long-term follow up is ongoing. Optimization of HCT and further development of emerging therapies, all of which must be delivered early in life, are expected to further improve outcomes of infants with IKD.

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Section: Resultsmentioning

confidence: 99%

“…14 Standard definitions were used for neutrophil and platelet engraftment. 24 Full donor chimerism was defined as >95% donor cells in both myeloid and lymphoid lineages. 24 Acute and chronic graft versus host disease was graded according to consensus criteria.…”

Section: Methodsmentioning

confidence: 99%

Benefits of newborn screening and hematopoietic cell transplant in infantile Krabbe disease

et al. 2022

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“…Poor graft function (PGF) was defined as sustained cytopenia of 2 or 3 lineages (neutrophil count <.5 × 10 9 /L, hemoglobin < 70 g/L, and platelet count < 20 × 10 9 /L) for over 2 weeks with full donor chimerism of > 95%, hypoplastic-aplastic BM, and absence of severe GvHD, active infection and drug toxicity. Primary PGF referred to PGF that failed to achieve initial engraftment, and sPGF was defined as a decrease of blood counts after prompt recovery ( 16 , 17 ). Chimerism analysis was evaluated using PB at 1, 2, 3, 6, 12 months post-transplantation and at annual outpatient visits thereafter.…”

Section: Methodsmentioning

confidence: 99%

The Incidence, Outcomes, and Risk Factors of Secondary Poor Graft Function in Haploidentical Hematopoietic Stem Cell Transplantation for Acquired Aplastic Anemia

et al. 2022

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Secondary poor graft function (sPGF) increases the risk of life-threatening complications after hematopoietic stem cell transplantation (HSCT). The incidence, clinical outcomes, and risk factors of sPGF have not been elucidated in haploidentical (haplo-) HSCT for acquired aplastic anemia (AA) patients. We retrospectively reviewed 423 consecutive AA patients who underwent haplo-HSCT between January 2006 and December 2020 and report a 3-year cumulative incidence of 4.62% (95% confidence interval [CI]: 3.92%-10.23%) of sPGF. While no primary PGF occurred. The median time to sPGF was 121 days (range 30-626 days) after transplantation. To clarify the risk factors for sPGF, 17 sPGF cases and 382 without PGF were further analyzed. Compared to patients without PGF, the 2-year overall survival was significantly poorer for sPGF patients (67.7% vs 90.8%, p =.002). Twelve sPGF patients were alive until the last follow-up, and 7 achieved transfusion independency. The multivariable analyses revealed that later neutrophil engraftment (OR 2.819, p=.049) and a history of refractory cytomegalovirus viremia (OR=7.038, p=.002) post-transplantation were associated with sPGF. There was weak evidence that a history of grade 3-4 acute graft-versus-host disease increased the risk of sPGF (p=.063). We advocated better post-transplantation strategies to balance the risk of immunosuppression and viral reactivation for haplo-HSCT in AA patients.

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“…It usually refers to persistent cytopenia in more than one cell lineage, accompanied by a hypoplastic/aplastic bone marrow and complete donor chimerism in the absence of relapse [ 13 ]. There is usually a dependence on blood and/or platelet transfusions and/or growth factor support in the absence of other explanations such as disease relapse, drugs or infections [ 14 ].…”

Section: Causes Of Thrombocytopenia After Allo-sctmentioning

confidence: 99%

Thrombocytopenia and Therapeutic Strategies after Allogeneic Hematopoietic Stem Cell Transplantation

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Canaro

Bastida

et al. 2022

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Thrombocytopenia following allogeneic hematopoietic stem cell transplantation is a usual complication and can lead to high morbidity and mortality. New strategies, such as the use of another graft versus host-disease prophylaxis, alternative donors, and management of infections, have improved the survival of these patients. The mechanisms are unknown; therefore, the identification of new strategies to manage this potentially serious problem is needed. Thrombopoietin receptor agonists are currently available to stimulate platelet production. Some small retrospective studies have reported their potential efficacy in an allogeneic stem cell transplant setting, confirming good tolerability. Recent studies with higher numbers of patients also support their safety and efficacy in this setting, hence establishing the use of these drugs as a promising strategy for this post-transplant complication. However, prospective trials are needed to confirm these results.

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Standardizing Definitions of Hematopoietic Recovery, Graft Rejection, Graft Failure, Poor Graft Function, and Donor Chimerism in Allogeneic Hematopoietic Cell Transplantation: A Report on Behalf of the American Society for Transplantation and Cellular Therapy

Cited by 91 publications

References 15 publications

Benefits of newborn screening and hematopoietic cell transplant in infantile Krabbe disease

Benefits of newborn screening and hematopoietic cell transplant in infantile Krabbe disease

The Incidence, Outcomes, and Risk Factors of Secondary Poor Graft Function in Haploidentical Hematopoietic Stem Cell Transplantation for Acquired Aplastic Anemia

Thrombocytopenia and Therapeutic Strategies after Allogeneic Hematopoietic Stem Cell Transplantation

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