Complications of Haploidentical Hematopoietic Cell Transplantation with Post-Transplant Cyclophosphamide—A Prospective Study on Behalf of the EBMT Transplant Complications Working Party

<b>Background:</b> Haploidentical hematopoietic cell transplantations (haplo-HCTs) with post-transplant cyclophosphamide (PT-Cy) are standard practice, but complications causing morbidity and mortality are not well described. <b>Methods:</b> The aim of this prospective non-interventional multicenter study was to document frequency of potential non-infectious and infection-related complications and main transplant outcomes after the first unmanipulated haplo-HCT with PT-Cy between 2017 and 2019 in 129 adult patients with hematological malignancies. The median follow-up was 37.3 months [95% CI: 34.3-39.7]. <b>Results:</b> The cumulative incidence (CI) of acute graft versus host disease (aGvHD) at day +100 was 22.4% grade II-IV [95% CI: 15.5-30.1] and 8.8% grade III-IV [95% CI: 4.6-14.6], respectively. The cumulative incidence of chronic GvHD (cGvHD) at 24 months was 25.8% [95% CI: 18.5-33.6]; extensive cGvHD was 10.9% [95% CI: 6.3-17.1], respectively. The most frequent non-infectious complications for the whole study population were mucositis-37.5% (<i>n</i> = 48); renal insufficiency-18% (<i>n</i> = 23); and cardiovascular complications-10.9% (<i>n</i> = 14). The following infection-related complications were diagnosed: bacterial in 84 (65.1%), viral in 66 (51.6%), and fungal in 24 (18.6%) recipients. Two-year OS was 58.1% [95% CI: 50.2-67.3]; NRM-27.1% [95% CI: 19.7-35]; PFS-50.4% [95% CI: 42.5-59.8]; and GRFS-38.8% [95% CI: 31.2-48.1]. About 50% of all deaths were directly caused by infection or infection-related conditions. <b>Conclusions:</b> Disease remission status at transplant significantly affected PFS, chronic GvHD, and GRFS. Although clinical applications of haplo-HCT with PTCy are widespread, the study confirms the need to reduce infection-related mortality after this type of GvHD prophylaxis.

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Journal
Cancers
Published
2025-12-18
DOI
https://doi.org/10.3390/cancers17244029
Primary Topic
Hematopoietic Stem Cell Transplantation
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article

Complications of Haploidentical Hematopoietic Cell Transplantation with Post-Transplant Cyclophosphamide—A Prospective Study on Behalf of the EBMT Transplant Complications Working Party

Hélène Schoemans, Ivan Moiseev, Jakob Robert Passweg, Mutlu Arat et al.
Cancers
Hematopoietic Stem Cell Transplantation
article

Complications of Haploidentical Hematopoietic Cell Transplantation with Post-Transplant Cyclophosphamide—A Prospective Study on Behalf of the EBMT Transplant Complications Working Party

Hélène Schoemans, Ivan Moiseev, Jakob Robert Passweg, Mutlu Arat, Pavel Jindra, Emmanuelle Polge, Urpu Salmenniemi, Rodrigo Martino, Alexander Kulagin, Matthias Eder, William Boreland, Christophe Peczynski, Grzegorz Władysław Basak, Alberto Mussetti, José Luis López Lorenzo, Zinaida Perić, Olaf Penack, Simona Sica, Charlotte Graham, Pascale Ambron, Agnieszka Tomaszewska, Mohamed-Amine Bekadja
article en

Abstract

<b>Background:</b> Haploidentical hematopoietic cell transplantations (haplo-HCTs) with post-transplant cyclophosphamide (PT-Cy) are standard practice, but complications causing morbidity and mortality are not well described. <b>Methods:</b> The aim of this prospective non-interventional multicenter study was to document frequency of potential non-infectious and infection-related complications and main transplant outcomes after the first unmanipulated haplo-HCT with PT-Cy between 2017 and 2019 in 129 adult patients with hematological malignancies. The median follow-up was 37.3 months [95% CI: 34.3-39.7]. <b>Results:</b> The cumulative incidence (CI) of acute graft versus host disease (aGvHD) at day +100 was 22.4% grade II-IV [95% CI: 15.5-30.1] and 8.8% grade III-IV [95% CI: 4.6-14.6], respectively. The cumulative incidence of chronic GvHD (cGvHD) at 24 months was 25.8% [95% CI: 18.5-33.6]; extensive cGvHD was 10.9% [95% CI: 6.3-17.1], respectively. The most frequent non-infectious complications for the whole study population were mucositis-37.5% (<i>n</i> = 48); renal insufficiency-18% (<i>n</i> = 23); and cardiovascular complications-10.9% (<i>n</i> = 14). The following infection-related complications were diagnosed: bacterial in 84 (65.1%), viral in 66 (51.6%), and fungal in 24 (18.6%) recipients. Two-year OS was 58.1% [95% CI: 50.2-67.3]; NRM-27.1% [95% CI: 19.7-35]; PFS-50.4% [95% CI: 42.5-59.8]; and GRFS-38.8% [95% CI: 31.2-48.1]. About 50% of all deaths were directly caused by infection or infection-related conditions. <b>Conclusions:</b> Disease remission status at transplant significantly affected PFS, chronic GvHD, and GRFS. Although clinical applications of haplo-HCT with PTCy are widespread, the study confirms the need to reduce infection-related mortality after this type of GvHD prophylaxis.

CancersVol. 17(24)
Università Cattolica del Sacro Cuore (IT), Bahçeşehir University (TR), Université Oran 1 Ahmed Ben Bella (DZ), University of Rijeka (HR), Inserm (FR), King's College London (GB), Charles University (CZ), Medical University of Warsaw (PL), First Pavlov State Medical University of St. Petersburg (RU), Hospital de Sant Pau (ES), University Hospital of Basel (CH), Medizinische Hochschule Hannover (DE), Sorbonne Université (FR), London Cancer (GB), Memorial Sisli Hospital (TR), Hospital Universitario Fundación Jiménez Díaz (ES), Centre de Recherche Saint-Antoine (FR), Duran i Reynals Hospital (ES), Cancer Society of Finland (FI), Charité - Universitätsmedizin Berlin (DE)
Openalex Percentile: Top 39%
Hematopoietic Stem Cell Transplantation
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