Transplant center characteristics and survival after allogeneic hematopoietic cell transplantation in adults

Majhail, Navneet S.; Mau, Lih Wen; Chitphakdithai, Pintip; Denzen, Ellen M.; Joffe, Steven; Lee, Stephanie J.; LeMaistre, Charles F.; Loberiza, Fausto R.; Parsons, Susan K.; Repaczki-Jones, Ramona; Robinett, Pam; Rizzo, J. Douglas; Murphy, Elizabeth A.; Logan, Brent R.; Le‐Rademacher, Jennifer

doi:10.1038/s41409-019-0748-1

Cited by 38 publications

(19 citation statements)

References 48 publications

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“…Hematopoietic-cell transplantation (HCT) is an established procedure for many inherited or acquired disorders of the hematopoietic system, whether benign or neoplastic, including those of the immune system, and as enzyme replacement in metabolic disorders [1][2][3]. The activity survey of the European Society of Blood and Marrow Transplantation (EBMT), describing the status of HCT in Europe and affiliated countries, has become an instrument to observe trends and to monitor changes in the technology in Europe and neighboring countries [4][5][6][7][8][9][10][11][12][13][14]. The survey using a standardized structure captures the numbers of HCT from highly committed participating teams, divided by indication, donor type, and stem cell source.…”

Section: Introductionmentioning

confidence: 99%

The EBMT activity survey on hematopoietic-cell transplantation and cellular therapy 2018: CAR-T’s come into focus

Passweg¹,

Baldomero²,

Chabannon³

et al. 2020

Bone Marrow Transplant

164

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Hematopoietic-cell transplantation (HCT) is widely used for acquired and congenital disorders of the hematopoietic system. Number of transplants performed in Europe and associated countries continues to rise with 47,468 HCT in 42,901 patients [19,630 allogeneic (41%) and 27,838 autologous (59%)] reported by 701 centers in 50 countries in 2018. Main indications were myeloid malignancies 10,679 (25%; 97% allogeneic), lymphoid malignancies 27,318 (64%; 20% allogeneic), solid tumors 1625 (4%; 2.9% allogeneic), and nonmalignant disorders 3063 (7%; 81% allogeneic). This year's analysis focuses on cellular therapies with the marked growth in CAR T-cell therapies from 151 in 2017 to 301 patients reported in 2018. Other cellular therapy numbers show less significant changes. Important trends in HCT include a 49% increase in allogeneic HCT for chronic phase CML (although transplant numbers remain low) and a 24% increase in aplastic anemia. In autologous HCT, there is an ongoing increase in autoimmune diseases (by 19%), predominantly due to activity in multiple sclerosis. This annual report reflects current activity and highlights important trends, useful for health care planning.

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Section: Introductionmentioning

confidence: 99%

The EBMT activity survey on hematopoietic-cell transplantation and cellular therapy 2018: CAR-T’s come into focus

Passweg¹,

Baldomero²,

Chabannon³

et al. 2020

Bone Marrow Transplant

164

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“…Our symbolic two-step method was previously applied to a retrospective evaluation of bone marrow transplant center practices on post-transplant survival [ 4 ]. The retrospective data analysis was based on a subset of data obtained from a study conducted by Majhail et al [ 10 , 11 ] The subset comprised 3320 patients from 67 transplant centers across the United States (approximately 50 patients per center, on average). The continuous outcome of interest

was the probability of survival at 1-year based on pseudo-observations which represented the estimated value of the survival function at 1-year for each patient j in center i [ 12 ] and calculated using the R package pseudo .…”

Section: Resultsmentioning

confidence: 99%

Sample size planning in the design and analysis of cluster randomized trials using the symbolic two-step method

Zahrieh

Le‐Rademacher

2020

Contemporary Clinical Trials Communications

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Introduction Evidence that can be used to improve clinical practice patterns and processes is frequently generated through standard, parallel-arms cluster randomized trial (CRT) designs that test interventions implemented at the center-level. Although the primary endpoint of these trials is often a center-level outcome, patient-level factors may vary between centers and, consequently, may influence the center-level outcome. Furthermore, there may be important factors that predict the variation in the center-level outcome and this knowledge can help contextualize the trial results and inform practice patterns. Methods Our symbolic two-step method that applies symbolic data analysis to account for patient-level factors when estimating and testing a center-level effect on both the average center-level outcome and its variation was developed for such settings. Herein, we sought to extend the method to prospectively size a CRT so that the application of our method in data analysis is consistent with the design. Results Our formulaic approach to sample size planning incorporated predictive factors of the within-center variation and accounted for patient-level characteristics. The sample size approximation performed well in many different pragmatic settings. Conclusions Our symbolic two-step method provides an alternate approach in the design and analysis of CRTs evaluating novel improvement processes within care delivery research.

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“…Die Risikofaktoren, die die Prätransplantationsphase und deren Komplikationen bestimmen, können eingeteilt werden in: patientenspezifische Risikofaktoren: Alter, Allgemeinzustand (Komorbiditäten gemäß Eastern Cooperative Oncology Group [ECOG]) und Vortherapien; krankheitsspezifische Risikofaktoren: Entität, Mutationen und Krankheitsdynamik (Remission, Rezidiv, therapierefraktär); transplantationsspezifische Risikofaktoren: Spenderauswahl („human leukocyte antigen [HLA] matching“, verwandt vs. unverwandt), Intensität der Konditionierungstherapie, Stammzellquelle und GvHD-Prophylaxe [ 16 , 22 , 31 , 42 ]. …”

Section: Zeitlicher Ablauf Und Komplikationen Der Allogene Hsztunclassified

Der knochenmarktransplantierte Patient auf der Intensivstation

Stecher

Stemmler

Tischer

et al. 2021

Med Klin Intensivmed Notfmed

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Der knochenmarktransplantierte Patient auf der Intensivstation Hintergrund Die allogene hämatopoetische Stammzelltransplantation (HSZT) gehört zweifelsfrei zu den invasivsten und aggressivsten konservativen Maßnahmen im therapeutischen Spektrum der Hämatoonkologie. Die Indikationsstellung erfolgt nach Risikofaktoren sowie nach Erkrankungsverlauf unter konventioneller Therapie (z. B. rezidiviert, refraktär) und beinhaltet häufig Erkrankungen mit Hochrisikogenetik sowie nichtmaligne Erkrankungen wie z. B. die schwere aplastische Anämie oder Immundefekterkrankungen. Das Ziel einer allogenen HSZT ist die Bekämpfung der malignen/ nichtmalignen Grunderkrankung. Dies geschieht durch 1) eine chemo-und/oder strahlentherapeutische Vorbehandlung und 2) die Induktion einer Graft-versus-Leukämie-/-Lymphom(GvL)-Reaktion sowie 3) Rekonstitution einer gesunden Spenderhämatopoese bzw. eines funktionsfähigen Immunsystems mit Ausbildung einer Toleranz.

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Transplant center characteristics and survival after allogeneic hematopoietic cell transplantation in adults

Cited by 38 publications

References 48 publications

The EBMT activity survey on hematopoietic-cell transplantation and cellular therapy 2018: CAR-T’s come into focus

The EBMT activity survey on hematopoietic-cell transplantation and cellular therapy 2018: CAR-T’s come into focus

Sample size planning in the design and analysis of cluster randomized trials using the symbolic two-step method

Der knochenmarktransplantierte Patient auf der Intensivstation

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