Figure 1 - available via license: Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International
Content may be subject to copyright.
Cumulative incidence of all-cause late mortality of 2-year survivors after allogeneic BMT in childhood for all bone marrow failure syndromes (n = 120) and for Fanconi anemia (n = 104).
Source publication
Children with bone marrow failure syndromes and severe aplastic anemia (SAA) are treated with allogeneic blood or marrow transplantation (BMT). However, there is a paucity of studies examining late mortality risk after allogeneic BMT performed in childhood for bone marrow failure syndromes and SAA and evaluating how this risk differs between these...
Contexts in source publication
Context 1
... patients with SAA, 19 (12.9%) had died after surviving for 2 years post-BMT at a median age of 29.8 years (range, 7.8 to 54.1 years). The cumulative mortality at 10 years and 15 years after BMT was 7.7% and 13.6%, respectively, for the bone marrow failure syndrome cohort and 4.2% and 6.9%, respectively, for the SAA cohort ( Figure 1A). The cumulative mortality after BMT for Fanconi anemia and other bone marrow failure syndromes is shown in Figure 1B. ...
Context 2
... cumulative mortality at 10 years and 15 years after BMT was 7.7% and 13.6%, respectively, for the bone marrow failure syndrome cohort and 4.2% and 6.9%, respectively, for the SAA cohort ( Figure 1A). The cumulative mortality after BMT for Fanconi anemia and other bone marrow failure syndromes is shown in Figure 1B. Table 2 presents the SMRs in this cohort using age-, sex-, and calendar-specific rates from the general US population. ...
Citations
... Recently published studies analyzing aspects of late posttransplant mortality showed a decrease over the last three decades in late mortality in patients after allogeneic HCT during childhood [2], and also a significant role of fatal infections in late phase, contributing to one-third of all deaths in this phase [3]. ...
We previously analyzed trends in incidence and factors associated with lethal complications in ALL/AML/CML patients (causes of deaths; COD-1 study). The objective of this study was the analysis of incidence and specific causes of death after HCT, with focus on infectious deaths in two time periods, 1980–2001 (cohort-1) and 2002–2015 (cohort-2). All patients with HCT for lymphoma, plasma cell disorders, chronic leukemia (except CML), myelodysplastic/myeloproliferative disorders, registered in the EBMT-ProMISe-database were included (n = 232,618) (COD-2 study). Results were compared to those in the ALL/AML/CML COD-1 study. Mortality from bacterial, viral, fungal, and parasitic infections decreased in very early, early and intermediate phases. In the late phase, mortality from bacterial infections increased, while mortality from fungal, viral, or unknown infectious etiology did not change. This pattern was similar for allo- and auto-HCT in COD-1 and COD-2 studies, with a distinct and constant lower incidence of all types of infections at all phases, after auto-HCT. In conclusion, infections were the main cause of death before day +100, followed by relapse. Mortality from infectious deaths significantly decreased, except late phase. Post-transplant mortality has significantly decreased in all phases, from all causes after auto-HCT; it has decreased in all phases after allo-HCT except late phase.
... Platelet transfusion can be administered to prevent or treat bleeding due to thrombocytopenia (132). In the case of acquired aplastic anemia, a condition associated with autoimmunity, immunosuppressive therapy with antithymocyte globulin and cyclosporine A is used (132,135,136). So far, this therapy has not been associated with reduced fertility in women, but the exact effect on male fertility has yet to be explored (137). ...
Due to the growing number of young patients at risk of germ cell loss, there is a need to preserve spermatogonial stem cells for patients who are not able to bank spermatozoa. Worldwide, more and more clinics are implementing testicular tissue (TT) banking programs, making it a novel, yet indispensable, discipline in the field of fertility preservation. Previously, TT cryopreservation was predominantly offered to young cancer patients before starting gonadotoxic chemo- or radiotherapy. Nowadays, most centers also bank TT from patients with non-malignant conditions who need gonadotoxic conditioning therapy prior to hematopoietic stem cell (HSCT) or bone marrow transplantation (BMT). Additionally, some centers include patients who suffer from genetic or developmental disorders associated with prepubertal germ cell loss or patients who already had a previous round of chemo- or radiotherapy. It is important to note that the surgical removal of TT is an invasive procedure. Moreover, TT cryopreservation is still considered experimental as restoration methods are not yet clinically available. For this reason, TT banking should preferably only be offered to patients who are at significant risk of becoming infertile. In our view, TT cryopreservation is recommended for young cancer patients in need of high-risk chemo- and/or radiotherapy, regardless of previous low-risk treatment. Likewise, TT banking is advised for patients with non-malignant disorders such as sickle cell disease, beta-thalassemia, and bone marrow failure, who need high-risk conditioning therapy before HSCT/BMT. TT retrieval during orchidopexy is also proposed for patients with bilateral cryptorchidism. Since patients with a medium- to low-risk treatment generally maintain their fertility, TT banking is not advised for this group. Also for Klinefelter patients, TT banking is not recommended as it does not give better outcomes than a testicular sperm extraction later in life.
... En esta serie se observó EICH agudo y crónico en el 29% de los pacientes, similar a lo reportado en la literatura con diferentes tipos de donante, que están entre 21,1-47,7% 26,34,37 . El estudio realizado por Sallfors-Holmqvist et al (BMTSS-2), entre 1974 y 2010, en el cual incluyeron pacientes menores de 21 años llevados a TPH debido a SFM, obtuvieron una SG de 86,4% a los 15 años posterior al TPH 38 . En nuestro caso alcanzamos una SG del 84% a 5 años, y específicamente 86% en el grupo de SFMA. ...
Los síndromes de falla medular (SFM) son trastornos infrecuentes, con una incidencia anual de 2-4 casos por millón. Las opciones de tratamiento incluyen terapia de inmunosupresión (TIS) y restauración de la hematopoyesis con trasplante de progenitores hematopoyéticas (TPH).Objetivo: Analizar los desenlaces de pacientes pediátricos diagnosticados con SFM tratados en una institución de alta complejidad.Pacientes y Método: Estudio retrospectivo de pacientes pediátricos con diagnóstico de SFM que consultaron a la Fundación Valle del Lili, Cali. Se realizo análisis estadístico descriptivo según SFM adquirida (SFMA) y SFM congénita (SFMC). Los desenlaces incluyeron: tratamiento, complicaciones, supervivencia global (SG) en los trasplantados, calculada con el método KaplanMeier.Resultados: Se incluyeron 24 pacientes con SFM, edad 6,5 ± 4 años, 50% mujeres. El 58% fueron SFMC, 9 con anemia de Fanconi, 2 disqueratosis congénita, 2 trombocitopenia amegacariocítica congénita, uno anemia Diamond-Blackfan. Doce pacientes con TPH tuvieron SG a 5 años de 83%. SFMA correspondió al 42%, 6 recibieron TIS-TPH, 3 TIS y 1 TPH, la SG del grupo con TIS-TPH fue 86%. Seis pacientes fallecieron, 4/6 relacionadas con infección.Conclusiones: En esta serie fue mayor el número de casos con SFMC. La SG de los pacientes llevados a TPH es comparable con la reportada en estudios recientes. La causa de muerte predominante fue infecciosa que también se ha reportado previamente. El tratamiento instaurado en los pacientes de esta serie mostró resultados favorables en un centro de alta complejidad en un país latinoamericano.
... En esta serie se observó EICH agudo y crónico en el 29% de los pacientes, similar a lo reportado en la literatura con diferentes tipos de donante, que están entre 21,1-47,7% 26,34,37 . El estudio realizado por Sallfors-Holmqvist et al (BMTSS-2), entre 1974 y 2010, en el cual incluyeron pacientes menores de 21 años llevados a TPH debido a SFM, obtuvieron una SG de 86,4% a los 15 años posterior al TPH 38 . En nuestro caso alcanzamos una SG del 84% a 5 años, y específicamente 86% en el grupo de SFMA. ...
Introduction:
Bone marrow failure (BMF) syndromes are rare disorders with an annual incidence of 2-4 cases per million. Treatment options include immunosuppressive therapy (IST) and hematopoietic stem cell transplantation (HSCT).
Objective:
To analyze the outcomes of pediatric patients diagnosed with BMF treated in a tertiary care center.
Patients and Method:
Retrospective study of pediatric patients diagnosed with BMF who consulted at Fundación Valle de Lili, Cali. Descriptive statistical analysis was performed according to Acquired BMF (ABMF) and Inherited BMF (IBMF). The outcomes include treatment, complications, overall survival (OS) in transplant patients, calculated using the Kaplan Meier method.
Results:
We included 24 patients with BMF, average age 6.5 ± 4 years, and 50% were women. 58% presented IBMF, 9 with Fanconi anemia (FA), 2 dyskeratosis congenita, 2 congenital amegakaryocytic thrombocytopenia, and 1 presented Diamond-Blackfan anemia. 12 patients treated with HSCT had a 5-year OS of 83%. ABMF represented 42%. 6 patients received IST-HSCT, 3 received IST, and 1 received HSCT. The OS of the IST-HSCT group was 86%. Six patients died, four of them related to infection.
Conclusions:
In this series, there was a higher number of cases with IBMF. The OS of patients treated with HSCT is similar to that reported in recent studies. The most frequent cause of death was of infectious origin which has also been previously reported. The treatment esta blished in the patients showed favorable results in a Latin American tertiary care center.
Conditional on surviving the first 2 to 5 years after allogeneic blood or marrow transplantation (BMT), the 10-year overall survival approaches 80%. Nonetheless, the risk of late mortality remains higher than the age- and sex-matched general population for several years after BMT. The higher mortality rates in transplant recipients translate into shorter projected life expectancies compared with the general population. Risk of relapse-related mortality reaches a plateau within 10 years after BMT. With increasing time from BMT, nonrelapse-related mortality becomes the leading cause of death, and continues to increase with time after BMT. The major causes of nonrelapse mortality include infection (with or without chronic graft-versus-host disease), subsequent neoplasms, and cardiopulmonary compromise. In this review, findings from large cohorts are summarized, identifying opportunities for risk-based anticipatory intervention strategies to reduce mortality.
