Adriana Balduzzi, Maria Grazia Valsecchi, Thai Hoa Tran, Jan Zuna, Veronica Leoni, Gunnar Cario, Grazia Fazio, Virginie Gandemer, Sarah K Tasian, Inge M van der Sluis, Nicolò Peccatori, Rosanna Parasole, Stefania Monterisi, Mignon L Loh, Meenakshi Devidas, Stephen P Hunger, John A Kairalla, Paola De Lorenzo, Lewis B Silverman, Andrea Biondi
{"title":"Philadelphia chromosome-positive acute lymphoblastic leukaemia in children and adolescents: A changing treatment landscape and a methodological challenge.","authors":"Adriana Balduzzi, Maria Grazia Valsecchi, Thai Hoa Tran, Jan Zuna, Veronica Leoni, Gunnar Cario, Grazia Fazio, Virginie Gandemer, Sarah K Tasian, Inge M van der Sluis, Nicolò Peccatori, Rosanna Parasole, Stefania Monterisi, Mignon L Loh, Meenakshi Devidas, Stephen P Hunger, John A Kairalla, Paola De Lorenzo, Lewis B Silverman, Andrea Biondi","doi":"10.1111/bjh.70288","DOIUrl":"https://doi.org/10.1111/bjh.70288","url":null,"abstract":"","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-12-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145831830","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Several tools have been developed to provide prognostic indicators that are more accurate than chronological age alone because choosing the best treatment for older lymphoma patients is challenging. For older patients with diffuse large B-cell lymphoma (DLBCL), one of these prognostic indicators is patient fitness; its definition has been validated, its assessment is recommended by international guidelines and it is currently integrated into several clinical trials. In terms of fitness, frailty is emerging as a major barrier to curative options for older patients with Hodgkin lymphoma (HL) as well, and the preliminary data now available support geriatric assessment in this setting. This review presents and discusses the robust results on the prognostic value of assessing fitness achieved in DLBCL and the early findings of geriatric studies conducted in HL, including promising novel approaches to defining patient fitness.
{"title":"The importance of assessing the fitness of older patients with newly diagnosed diffuse large B-cell lymphoma and classic Hodgkin lymphoma.","authors":"Vittorio Ruggero Zilioli, Alessandra Tucci, Michele Spina, Francesco Merli, Annalisa Arcari","doi":"10.1111/bjh.70290","DOIUrl":"https://doi.org/10.1111/bjh.70290","url":null,"abstract":"<p><p>Several tools have been developed to provide prognostic indicators that are more accurate than chronological age alone because choosing the best treatment for older lymphoma patients is challenging. For older patients with diffuse large B-cell lymphoma (DLBCL), one of these prognostic indicators is patient fitness; its definition has been validated, its assessment is recommended by international guidelines and it is currently integrated into several clinical trials. In terms of fitness, frailty is emerging as a major barrier to curative options for older patients with Hodgkin lymphoma (HL) as well, and the preliminary data now available support geriatric assessment in this setting. This review presents and discusses the robust results on the prognostic value of assessing fitness achieved in DLBCL and the early findings of geriatric studies conducted in HL, including promising novel approaches to defining patient fitness.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-12-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145831836","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Maria L Andersson, Olina Lind, Kia Heimersson, Anna Berglöf, Teresa Del Peso Santos, Lucía Peña-Pérez, Qing Wang, Tom A Mulder, Rula Zain, Robert Månsson, Richard Rosenquist, C I Edvard Smith, Anders Österborg, Marzia Palma
{"title":"T-cell immunomodulation occurs with different time kinetics during acalabrutinib and zanubrutinib therapy in chronic lymphocytic leukaemia.","authors":"Maria L Andersson, Olina Lind, Kia Heimersson, Anna Berglöf, Teresa Del Peso Santos, Lucía Peña-Pérez, Qing Wang, Tom A Mulder, Rula Zain, Robert Månsson, Richard Rosenquist, C I Edvard Smith, Anders Österborg, Marzia Palma","doi":"10.1111/bjh.70293","DOIUrl":"https://doi.org/10.1111/bjh.70293","url":null,"abstract":"","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-12-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145831895","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Dong Hyun Kim, Sin Young Park, Hyunsoo Cho, Ja Min Byun, Dong-Yeop Shin, Youngil Koh, Inho Kim, Sung-Soo Yoon, Junshik Hong
Donor age is an important factor influencing outcomes after allogeneic haematopoietic stem cell transplantation (allo-HSCT), but its relevance across donor types and distinct disease biology in acute myeloid leukaemia (AML) and myelodysplastic syndrome (MDS) remains unclear. We analysed 320 patients with AML/MDS who underwent first allo-HSCT between 2016 and 2024. Donors were categorized as matched or mismatched and further stratified as young (<35 years) or old (≥35 years). Matched donors achieved superior survival compared with mismatched donors, while outcomes with young mismatched donors were intermediate and old mismatched donors were inferior driven by high non-relapse mortality. This effect was most evident in MDS, where young mismatched donors achieved survival similar to matched donors, while old mismatched donors were associated with inferior overall survival (hazard ratio [HR] 2.66) and relapse-free survival (HR 2.45). By contrast, in AML, survival did not differ by donor age; instead, disease biology dominated prognosis, with marrow blasts and European LeukemiaNet (ELN) risk strongly predicting outcomes. In conclusion, donor age interacts with donor type to shape allo-HSCT outcomes, with a marked impact in MDS but limited effect in AML. These findings suggest that old mismatched donors should be avoided in MDS and underscore the importance of disease-specific considerations in donor selection.
{"title":"Donor age and type in allogeneic haematopoietic stem cell transplantation: Strong effect in MDS, limited in AML.","authors":"Dong Hyun Kim, Sin Young Park, Hyunsoo Cho, Ja Min Byun, Dong-Yeop Shin, Youngil Koh, Inho Kim, Sung-Soo Yoon, Junshik Hong","doi":"10.1111/bjh.70303","DOIUrl":"https://doi.org/10.1111/bjh.70303","url":null,"abstract":"<p><p>Donor age is an important factor influencing outcomes after allogeneic haematopoietic stem cell transplantation (allo-HSCT), but its relevance across donor types and distinct disease biology in acute myeloid leukaemia (AML) and myelodysplastic syndrome (MDS) remains unclear. We analysed 320 patients with AML/MDS who underwent first allo-HSCT between 2016 and 2024. Donors were categorized as matched or mismatched and further stratified as young (<35 years) or old (≥35 years). Matched donors achieved superior survival compared with mismatched donors, while outcomes with young mismatched donors were intermediate and old mismatched donors were inferior driven by high non-relapse mortality. This effect was most evident in MDS, where young mismatched donors achieved survival similar to matched donors, while old mismatched donors were associated with inferior overall survival (hazard ratio [HR] 2.66) and relapse-free survival (HR 2.45). By contrast, in AML, survival did not differ by donor age; instead, disease biology dominated prognosis, with marrow blasts and European LeukemiaNet (ELN) risk strongly predicting outcomes. In conclusion, donor age interacts with donor type to shape allo-HSCT outcomes, with a marked impact in MDS but limited effect in AML. These findings suggest that old mismatched donors should be avoided in MDS and underscore the importance of disease-specific considerations in donor selection.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-12-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145814803","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
T-cell/histiocyte-rich large B-cell lymphoma (THRLBCL) is an uncommon aggressive large B-cell lymphoma variant which can develop synchronously or following a diagnosis of nodular lymphocyte predominant Hodgkin lymphoma (NLPHL). There is morphological, immunophenotypic and molecular overlap between THRLBCL and NLPHL suggesting that these two entities may lie on the same spectrum. Due to the rarity of THRLBCL, accurate diagnosis can be challenging and there is a paucity of data on which to base treatment decisions. The management has largely followed diffuse large B-cell lymphoma (DLBCL) with rituximab, cyclophosphamide, doxorubicin, vincristine and prednisone established as the standard of care in the first line, with outcomes comparable to international prognostic index matched DLBCL. In the relapsed/refractory (R/R) setting, there is no standard of care. There is a move towards inclusion of THRLBCL patients in clinical trials evaluating novel agents, although historically they were commonly excluded. Due to the small numbers included in clinical trials, it has been difficult to discern the effect of novel agents in this cohort. Thus, we are reliant on larger real-world datasets to inform our understanding. This review will examine the data available in the first line and R/R setting with a focus on immunotherapeutic approaches.
{"title":"T-cell/histiocyte-rich large B-cell lymphoma in the era of novel immunotherapy: A focused review.","authors":"Joleen P Choy, Chan Y Cheah","doi":"10.1111/bjh.70294","DOIUrl":"https://doi.org/10.1111/bjh.70294","url":null,"abstract":"<p><p>T-cell/histiocyte-rich large B-cell lymphoma (THRLBCL) is an uncommon aggressive large B-cell lymphoma variant which can develop synchronously or following a diagnosis of nodular lymphocyte predominant Hodgkin lymphoma (NLPHL). There is morphological, immunophenotypic and molecular overlap between THRLBCL and NLPHL suggesting that these two entities may lie on the same spectrum. Due to the rarity of THRLBCL, accurate diagnosis can be challenging and there is a paucity of data on which to base treatment decisions. The management has largely followed diffuse large B-cell lymphoma (DLBCL) with rituximab, cyclophosphamide, doxorubicin, vincristine and prednisone established as the standard of care in the first line, with outcomes comparable to international prognostic index matched DLBCL. In the relapsed/refractory (R/R) setting, there is no standard of care. There is a move towards inclusion of THRLBCL patients in clinical trials evaluating novel agents, although historically they were commonly excluded. Due to the small numbers included in clinical trials, it has been difficult to discern the effect of novel agents in this cohort. Thus, we are reliant on larger real-world datasets to inform our understanding. This review will examine the data available in the first line and R/R setting with a focus on immunotherapeutic approaches.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-12-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145814867","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Ashley Brown, Richard J Aspinall, Stephen T Barclay, Mark Gillyon-Powell, Katie Jeffery, Patrick T Kennedy, Neil McDougall, Tom Pembroke, Martin Scott, Susan Shapiro
This is a new British Society of Haematology (BSH) guideline which focuses on the management of hepatitis B and C (HBV and HCV) infection affecting people with bleeding disorders (PwBD), including those who cleared the infection many years previously. It is based on the European Association for the Study of the Liver (EASL) guidance and is a succinct practical guide for haematologists to support joint care with hepatology. It is compatible with the joint international guidance from the European Association for Haemophilia and Allied Disorders, European Haemophilia Consortium, International Society on Thrombosis and Haemostasis (ISTH) and World Federation of Hemophilia 2024. However, it provides more specific guidance on investigations for fibrosis/cirrhosis and thresholds for onwards referral to hepatology in those who cleared HCV historically (either through spontaneous clearance or following effective treatment). The aim of this guideline is to improve the management of this group of patients nationally as well as potentially supporting the management of this group of patients internationally. It also contributes to the delivery of recommendations made by the UK Infected Blood Inquiry 2024.
{"title":"Guideline for the management of hepatitis B and C infection and subsequent liver disease surveillance (where indicated) in people with congenital bleeding disorders: A joint guideline from the British Society of Haematology and the British Viral Hepatitis Group.","authors":"Ashley Brown, Richard J Aspinall, Stephen T Barclay, Mark Gillyon-Powell, Katie Jeffery, Patrick T Kennedy, Neil McDougall, Tom Pembroke, Martin Scott, Susan Shapiro","doi":"10.1111/bjh.70283","DOIUrl":"https://doi.org/10.1111/bjh.70283","url":null,"abstract":"<p><p>This is a new British Society of Haematology (BSH) guideline which focuses on the management of hepatitis B and C (HBV and HCV) infection affecting people with bleeding disorders (PwBD), including those who cleared the infection many years previously. It is based on the European Association for the Study of the Liver (EASL) guidance and is a succinct practical guide for haematologists to support joint care with hepatology. It is compatible with the joint international guidance from the European Association for Haemophilia and Allied Disorders, European Haemophilia Consortium, International Society on Thrombosis and Haemostasis (ISTH) and World Federation of Hemophilia 2024. However, it provides more specific guidance on investigations for fibrosis/cirrhosis and thresholds for onwards referral to hepatology in those who cleared HCV historically (either through spontaneous clearance or following effective treatment). The aim of this guideline is to improve the management of this group of patients nationally as well as potentially supporting the management of this group of patients internationally. It also contributes to the delivery of recommendations made by the UK Infected Blood Inquiry 2024.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-12-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145808933","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Maria Huguet, Mireia Morgades, Alfredo Rivas-Delgado, Mariana Bastos-Oreiro, Raúl Córdoba, Antonio Salar, Fátima de la Cruz-Vicente, Miguel Alcoceba, Blanca Ferrer-Lores, Ana Jiménez-Ubieto, Antonio Gutiérrez, María Infante, Sofía Huerga, Pau Abrisqueta, David Cruz, Carlos Montalbán, Ana Muntañola, Sonia González-de Villambrosia, Josep-Maria Ribera, José-Tomás Navarro
{"title":"Validation of prognostic scores in patients with HIV-related diffuse large B-cell lymphoma: The value of peripheral blood parameters-A study from the Spanish Lymphoma Group GELTAMO.","authors":"Maria Huguet, Mireia Morgades, Alfredo Rivas-Delgado, Mariana Bastos-Oreiro, Raúl Córdoba, Antonio Salar, Fátima de la Cruz-Vicente, Miguel Alcoceba, Blanca Ferrer-Lores, Ana Jiménez-Ubieto, Antonio Gutiérrez, María Infante, Sofía Huerga, Pau Abrisqueta, David Cruz, Carlos Montalbán, Ana Muntañola, Sonia González-de Villambrosia, Josep-Maria Ribera, José-Tomás Navarro","doi":"10.1111/bjh.70287","DOIUrl":"https://doi.org/10.1111/bjh.70287","url":null,"abstract":"","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-12-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145808942","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Malin Rasmussen, Nikolaj Mannering, Henrik Frederiksen
{"title":"Incidence of systemic lupus erythematosus in patients with immune thrombocytopenia: A nationwide study.","authors":"Malin Rasmussen, Nikolaj Mannering, Henrik Frederiksen","doi":"10.1111/bjh.70299","DOIUrl":"https://doi.org/10.1111/bjh.70299","url":null,"abstract":"","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-12-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145808980","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
David T Yeung, Andrew P Grigg, John Reynolds, Susan Branford, Deepmala Mazumdar, Ilona Cunningham, Jake Shortt, Philip Rowlings, Rosemary Harrup, David M Ross, David Kipp, Anthony K Mills, Christopher K Arthur, Anthony Schwarer, Kathryn Jackson, Nicholas Viiala, Robert Weinkove, Agnes S M Yong, Sher G Gazdar, Deborah White, Naranie Shanmuganathan, Timothy Hughes
We evaluated the tolerability and efficacy of pegylated interferon alfa-2B (peg-IFNα; PegIntron, MSD) combined with nilotinib in the Australasian Leukaemia and Lymphoma Group CML11 (Pinnacle) study. This phase II study started patients on nilotinib 300 mg twice daily. Subcutaneous peg-IFNα was added at 30-50 50 μg/week from 3 months until 24 months as tolerated. Sixty patients were enrolled with a median age of 48.5 years (range 19-72); 45% were female. With a median follow-up of 60 months, 40 patients (67%) remain on study. The proportion of patients who received ≥50% and ≥85% of their assigned peg-IFNα doses were 58% and 35% respectively. Common reasons for peg-IFNα discontinuation were mood disturbance (5), thyroid disease (4) and myalgia (4). The cumulative incidence of Major Molecular Response (MMR, BCR::ABL1≤0.1%) was 87% by 12 months; Molecular Response 4.5 (MR4.5, BCR::ABL1≤0.0032%) incidence at 24 and 60 months was 55% and 82% respectively. Thirty-seven patients (62%) had MR4.5 for >24 months, 14 of whom attempted treatment-free remission (TFR); 13 remained in TFR at a median follow-up of 32 months. CML11 demonstrated that peg-IFNα with nilotinib leads to high rates of molecular response, with tolerability similar to prior studies. Trial registration ANZCTRN12612000851864.
{"title":"The PINNACLE study: A multicentre phase II trial of nilotinib in combination with pegylated interferon-α2b in newly diagnosed chronic phase chronic myeloid leukaemia.","authors":"David T Yeung, Andrew P Grigg, John Reynolds, Susan Branford, Deepmala Mazumdar, Ilona Cunningham, Jake Shortt, Philip Rowlings, Rosemary Harrup, David M Ross, David Kipp, Anthony K Mills, Christopher K Arthur, Anthony Schwarer, Kathryn Jackson, Nicholas Viiala, Robert Weinkove, Agnes S M Yong, Sher G Gazdar, Deborah White, Naranie Shanmuganathan, Timothy Hughes","doi":"10.1111/bjh.70276","DOIUrl":"https://doi.org/10.1111/bjh.70276","url":null,"abstract":"<p><p>We evaluated the tolerability and efficacy of pegylated interferon alfa-2B (peg-IFNα; PegIntron, MSD) combined with nilotinib in the Australasian Leukaemia and Lymphoma Group CML11 (Pinnacle) study. This phase II study started patients on nilotinib 300 mg twice daily. Subcutaneous peg-IFNα was added at 30-50 50 μg/week from 3 months until 24 months as tolerated. Sixty patients were enrolled with a median age of 48.5 years (range 19-72); 45% were female. With a median follow-up of 60 months, 40 patients (67%) remain on study. The proportion of patients who received ≥50% and ≥85% of their assigned peg-IFNα doses were 58% and 35% respectively. Common reasons for peg-IFNα discontinuation were mood disturbance (5), thyroid disease (4) and myalgia (4). The cumulative incidence of Major Molecular Response (MMR, BCR::ABL1≤0.1%) was 87% by 12 months; Molecular Response 4.5 (MR4.5, BCR::ABL1≤0.0032%) incidence at 24 and 60 months was 55% and 82% respectively. Thirty-seven patients (62%) had MR4.5 for >24 months, 14 of whom attempted treatment-free remission (TFR); 13 remained in TFR at a median follow-up of 32 months. CML11 demonstrated that peg-IFNα with nilotinib leads to high rates of molecular response, with tolerability similar to prior studies. Trial registration ANZCTRN12612000851864.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-12-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145808898","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Augustin Boudry, Florian Chevillon, Alice Marceau-Renaut, Thorsten Braun, Thomas Boyer, Nathalie Helevaut, Elise Fournier, Sandrine Geffroy, Nicolas Boissel, Emmanuelle Clappier, Claude Preudhomme, Nicolas Duployez, Catherine Poirot, Laurène Fenwarth
Allogeneic haematopoietic stem cell transplantation (ASCT) is a curative treatment for acute myeloid leukaemia (AML) but carries a high risk of gonadotoxicity. Ovarian tissue cryopreservation (OTC) offers a fertility preservation option, yet its safety in AML remains uncertain due to the risk of leukaemic cell reintroduction. The FERTILAM pilot study evaluated measurable residual disease (MRD) in ovarian tissue collected at complete remission (CR) from nine AML patients undergoing OTC before ASCT. MRD was assessed using patient-specific clonal markers via droplet digital polymerase chain reaction on DNA and RNA from bone marrow (BM), ovarian cortex and medulla. At CR, MRD-DNA was detected in ovarian cortex of four of nine patients, all with concurrent MRD positivity in BM. Three patients were negative in both BM and ovarian tissue. Paired cortex/medulla analyses showed concordant MRD-DNA results in five of six patients. BM MRD-RNA and MRD-DNA were fully concordant, whereas two discrepancies were observed between MRD-DNA and MRD-RNA in ovarian tissue. These findings suggest potential leukaemic cell persistence in ovarian tissue despite CR and highlight the need for sensitive molecular assays to assess safety prior to ovarian tissue transplantation.
{"title":"Assessment of measurable residual disease in ovarian tissue collected for fertility preservation in patients in remission from acute myeloid leukaemia: A pilot study.","authors":"Augustin Boudry, Florian Chevillon, Alice Marceau-Renaut, Thorsten Braun, Thomas Boyer, Nathalie Helevaut, Elise Fournier, Sandrine Geffroy, Nicolas Boissel, Emmanuelle Clappier, Claude Preudhomme, Nicolas Duployez, Catherine Poirot, Laurène Fenwarth","doi":"10.1111/bjh.70289","DOIUrl":"https://doi.org/10.1111/bjh.70289","url":null,"abstract":"<p><p>Allogeneic haematopoietic stem cell transplantation (ASCT) is a curative treatment for acute myeloid leukaemia (AML) but carries a high risk of gonadotoxicity. Ovarian tissue cryopreservation (OTC) offers a fertility preservation option, yet its safety in AML remains uncertain due to the risk of leukaemic cell reintroduction. The FERTILAM pilot study evaluated measurable residual disease (MRD) in ovarian tissue collected at complete remission (CR) from nine AML patients undergoing OTC before ASCT. MRD was assessed using patient-specific clonal markers via droplet digital polymerase chain reaction on DNA and RNA from bone marrow (BM), ovarian cortex and medulla. At CR, MRD-DNA was detected in ovarian cortex of four of nine patients, all with concurrent MRD positivity in BM. Three patients were negative in both BM and ovarian tissue. Paired cortex/medulla analyses showed concordant MRD-DNA results in five of six patients. BM MRD-RNA and MRD-DNA were fully concordant, whereas two discrepancies were observed between MRD-DNA and MRD-RNA in ovarian tissue. These findings suggest potential leukaemic cell persistence in ovarian tissue despite CR and highlight the need for sensitive molecular assays to assess safety prior to ovarian tissue transplantation.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-12-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145792604","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}