Trasplante de médula ósea haploidéntico en un paciente pediátrico con síndrome de Wiskott-Aldrich. A propósito de un caso / Haploidentical bone marrow transplantation in a pediatric patient with Wiskott-Aldrich syndrome. A case report

El síndrome de Wiskott-Aldrich es un error innato de la inmunidad de herencia ligada al cromosoma X, producido por variantes en el gen que codifica la proteína del síndrome de Wiskott-Aldrich (WASp). Reportamos el caso clínico de un paciente de 18 meses con diagnóstico de Wiskott-Aldrich que no presentaba donante antígeno leucocitario humano (HLA) idéntico y recibió un trasplante de células progenitoras hematopoyéticas (TCPH) con donante familiar haploidéntico. La profilaxis para enfermedad de injerto contra huésped incluyó ciclofosfamida (PT-Cy). El quimerismo del día +30 fue 100 % del donante y la evaluación postrasplante de la expresión de la proteína WAS fue normal. Actualmente, a 32 meses del trasplante, presenta reconstitución hematológica e inmunológica y quimerismo completo sin evidencia de enfermedad injerto contra huésped. El TCPH haploidéntico con PT-Cy se mostró factible y seguro en este caso de síndrome de WiskottAldrich en el que no se disponía de un donante HLA idéntico.

Wiskott-Aldrich syndrome (WAS) is an X-linked genetic disorder caused by mutations in the gene that encodes the Wiskott-Aldrich syndrome protein (WASp). Here, we report the clinical case of an 18-month-old boy diagnosed with Wiskott-Aldrich syndrome, who did not have an HLA-matched related or unrelated donor and was treated successfully with a hematopoietic stem cell transplant (HSCT) from a haploidentical family donor. Graft-versus-host disease (GvHD) prophylaxis included post-transplant cyclophosphamide (PT-Cy). At day +30, the peripheral blood-nucleated cell chimerism was 100% and the WAS protein had a normal expression. Currently, at month 32 post-transplant, the patient has hematological and immune reconstitution and complete donor chimerism without evidence of GvHD. HSCT with PT-Cy was a feasible and safe option for this patient with WAS, in which an HLA matched donor was not available.

Calidad de vida y toxicidad financiera de los receptores de trasplantes de células madre hematopoyéticas en COVID-19 / Quality of life and financial toxicity of hematopoietic stem cell transplant recipients in COVID-19 / Qualidade de vida e toxicidade financeira dos transplantados de células-tronco hematopoéticas na COVID-19

Objetivo: evaluar y correlacionar la calidad de vida y la toxicidad financiera de pacientes adultos sometidos a trasplante de células madre hematopoyéticas durante el período de la pandemia de COVID-19. Método: estudio observacional, analítico, realizado con 35 pacientes en un hospital de referencia para trasplante en Latinoamérica. Para la recolección de datos, se utilizaron los cuestionarios Functional Assessment Cancer Therapy Bone Marrow Transplantation y el COmprehensive Score for financial Toxicity. Para el análisis de los datos se utilizaron las pruebas de correlación de Spearman y Mann-Whitney. Resultados: la calidad de vida general durante la COVID-19 mostró un puntaje bajo (67,09/108) con mayor deterioro en el bienestar funcional (14,47/28), bienestar social (16,76/28) y preocupaciones adicionales (23,41/40). Los promedios del grupo alogénico fueron inferiores a los del grupo autólogo en todos los dominios, presentando diferencia significativa en relación a preocupaciones adicionales (p=0,01) y en el índice de evaluación del tratamiento (p=0,04). Se consideró que la toxicidad financiera tenía un impacto leve (22.11/44). Se observó una relación, aunque no significativa, entre la calidad de vida y la toxicidad financiera (p=0,051). Conclusión: la calidad de vida de la muestra fue baja; existe una correlación entre la calidad de vida y la toxicidad financiera, aunque no significativa. Cuanto mayor es la toxicidad financiera, menor es la calidad de vida.

Objective: to evaluate and correlate the quality of life and financial toxicity of adult patients undergoing hematopoietic stem cell transplantation during the COVID-19 pandemic. Method: observational, analytical study, carried out with 35 patients in a reference hospital for transplantation in Latin America. For data collection, the Functional Assessment Cancer Therapy Bone Marrow Transplantation and COmprehensive Score for Financial Toxicity questionnaires were used. Spearman and Mann-Whitney correlation tests were used for data analysis. Results: general quality of life during COVID-19 had a low score (67.09/108) with greater impairment in functional well-being (14.47/28), social well-being (16.76/28) and additional concerns (23.41/40). The means of the allogeneic group were lower than those of the autologous group in all domains, showing a significant difference in relation to additional concerns (p=0.01) and in the treatment evaluation index (p=0.04). Financial toxicity was considered to have a slight impact (22.11/44). There was a relationship, albeit not significant, between quality of life and financial toxicity (p=0.051). Conclusion: the quality of life of the sample was low; there is a correlation between quality of life and financial toxicity, although not significant. The higher the financial toxicity, the lower the quality of life.

Objetivo: avaliar e correlacionar a qualidade de vida e a toxicidade financeira dos pacientes adultos submetidos ao transplante de células-tronco hematopoéticas no período da pandemia de COVID-19. Método: estudo observacional, analítico, realizado com 35 pacientes em um hospital de referência para o transplante na América Latina. Para coleta de dados, utilizaram-se os questionários Functional Assessment Cancer Therapy Bone Marrow Transplantation e COmprehensive Score for financial Toxicity. Na análise dos dados empregaram-se os testes de correlação de Spearman e Mann-Whitney. Resultados: a qualidade de vida geral, durante a COVID-19, apresentou baixo escore (67,09/108), com maior comprometimento nas funções bem-estar funcional (14,47/28), social (16,76/28) e preocupações adicionais (23,41/40). As médias do grupo alogênico foram inferiores às do autólogo em todos os domínios, apresentando diferença significativa em relação às preocupações adicionais (p=0,01) e ao índice de avaliação do tratamento (p=0,04). A toxicidade financeira foi considerada de impacto leve (22,11/44). Observou-se relação, ainda que não significativa, entre a qualidade de vida e a toxicidade financeira (p=0,051). Conclusão: a qualidade de vida da amostra foi baixa, logo há uma correlação entre qualidade de vida e a toxicidade financeira, embora não significativa. Quanto maior a toxicidade financeira, menor a qualidade de vida.

¿Qué hay de nuevo en el tratamiento de amiloidosis por cadenas livianas? Parte 1: cadenas livianas / What is new in light chain amyloidosis treatment? Part 1: lightweight chains

La amiloidosis AL es una enfermedad debida al depósito, en órganos y tejidos, de fibrillas formadas por cadenas livianas producidas de forma patológica por plasmocitos clonales. Su tratamiento actualmente está orientado a erradicar el clon de células plasmáticas; este históricamente se extrapoló de tratamientos disponibles y estudiados para otras discrasias sanguíneas. En el año 2020, el Grupo de Estudio de Amiloidosis (GEA) confeccionó distintas guías de práctica clínica para el tratamiento de la amiloidosis AL. Desde entonces se han publicado ensayos clínicos que arrojan contundencia al conocimiento disponible hasta el momento, y están en desarrollo nuevas líneas de investigación que robustecen y estimulan el estudio en el área. En esta revisión se realiza una actualización de las guías existentes en lo que respecta al tratamiento de la amiloidosis por cadenas livianas.Como evidencia de relevancia, en el último año estuvieron disponibles resultados de ensayos clínicos que respaldan el uso de esquemas basados en daratumumab (un anticuerpo monoclonal anti-CD38+) para pacientes con diagnóstico reciente de amiloidosis AL como primera línea. Además, para el tratamiento de la amiloidosis AL refractaria o recaída, la disponibilidad de bibliografía respaldatoria es escasa y extrapolada del tratamiento del mieloma múltiple; sin embargo, actualmente existe evidencia de calidad para recomendar el uso de ixazomib, un inhibidor de proteosoma reversible por vía oral disponible en la Argentina desde 2020. Por último, se mencionan algunas líneas de investigación con otros anticuerpos monoclonales y terapéuticas basadas en el uso de CAR-T cells. (AU)

AL amyloidosis is a disease caused by the deposit in different organs and tissues of protein fibrils formed by light chains synthetized by pathological clonal plasma cells. Its treatment is currently aimed at eradicating this plasma cell clone and it has been historically extrapolated from available and validated treatments for other blood dyscrasias. In 2020, the Amyloidosis Study Group prepared different clinical practice guidelines for the treatment of AL amyloidosis.Since then, clinical trials have been published that confirm and strengthen the knowledge available up to now, and new lines of research are being developed that stimulate study in the area. In this review, an update of the existing guidelines regarding the treatment of AL amyloidosis is made. As relevant evidence, in the last year, results of clinical trials have been made available that support the use of regimens based on Daratumumab (an anti-CD38+ monoclonal antibody) for patients with newly diagnosed AL amyloidosis as first line therapy. In addition, for the treatment of refractory or relapsed AL amyloidosis, where the availability of supporting literature is scant and extrapolated from the treatment of multiple myeloma, there is currently quality evidence to recommend the use of ixazomib, an oral reversible proteasome inhibitor, only available in Argentina since 2020. Finally, some research lines exploring the efficacy of other monoclonal antibodies and therapeutic experiments based on the use of CAR-T cells are mentioned. (AU)

Outcomes and survival predictors of Latin American older adults with acute myeloid leukemia: Data from a single center

ABSTRACT Introduction: Acute myeloid leukemia (AML) is most commonly presented in older adults; however, it appears 10 years earlier in Latin American countries. Clinical evolution in older adults from this populations has not been characterized. We analyzed outcomes and survival predictors. Methods: Patients ≥ 55 years old diagnosed with AML at a hematology referral center from 2005 to 2020 receiving intensive chemotherapy (IC), low-dose cytarabine (LDAC) and best supportive care (BSC) were included. Survival analysis included the Kaplan-Meier and Cox models and the cumulative incidence of relapse (CIR). Results: Seventy-five adults were included and the overall survival (OS) was 4.87, 1.67 and 1.16 months, using IC, LDAC and BSC, respectively. The IC led to a higher OS (p < 0.001) and was a protective factor for early death, at a cost of more days spent hospitalized and more non-fatal treatment complications; non-significant differences were found between the LDAC and BSC. Eight (10.7%) patients underwent hematopoietic cell transplantation, with a higher OS (p = 0.013). Twenty (26.7%) patients achieved complete remission; 12 (60%) relapsed with a 6-month CIR of 57.9% in those < 70 years old vs. 86.5% in those ≥ 70 years old, p = 0.034. Multivariate analysis showed the white blood cell count (WBC) and IC had a significant impact on the patient survival, whereas chronological age and the Charlson comorbidity index (CCI) did not. Conclusion: AML in low-middle income countries demands a different approach; the IC improves survival, even with a high incidence of relapse, and should be offered as first-line treatment. Eligibility criteria should include WBC and a multidimensional evaluation. The age per se and the CCI should not be exclusion criteria to consider IC.

Effectiveness of the peripherally inserted central catheter for hematopoietic stem cell transplantation: a systematic review protocol

OBJECTIVE: to assess the effectiveness and safety of the peripherally inserted central catheter for hematopoietic stem cell transplantation. METHOD: this review will follow the recommendations of the Preferred Reporting Items for Systematic Reviews and Meta-Analyses, and the search steps will be presented through the flow diagram. The search strategy aims to locate both published and unpublished studies. No time or language restrictions will be applied. The review will consider experimental and observational studies that include adult and pediatric patients undergoing hematopoietic stem cell transplantation. Patients using peripherally inserted central catheters will be compared with those using other central catheters.

Perfil de liberación de biomarcadores miocárdicos en pacientes con enfermedades hemato-oncológicas que reciben trasplante de progenitores hematopoyéticos / Profile or myocardial biomarkers release in patients with hemato-oncological diseases who receive hematopoietic stem cell transplantation / Perfil de liberação de biomarcadores miocárdicos em pacientes com doenças hemato-oncológicas que recebem transplante de células-tronco hematopoéticas

Introducción: las enfermedades cardiovasculares (CV) son la primera causa de muerte en quienes sobreviven al cáncer. Aunque el trasplante de progenitores hematopoyéticos (TPH) se asocia con grados variables de cardiotoxicidad, estas complicaciones han sido escasamente caracterizadas. Objetivo: analizar el perfil de liberación de biomarcadores miocárdicos como potenciales indicadores subclínicos de cardiotoxicidad en pacientes sometidos a TPH. Material y método: estudio descriptivo, analítico, prospectivo transversal y unicéntrico, reclutando pacientes derivados a la policlínica de cardio-oncología, con indicación de TPH en octubre de 2018-marzo de 2020. Se realizaron controles clínicos, ECG, bioquímicos (troponina I TnI y péptido natriurético del tipo BBNP) e imagenológicos según algoritmo de seguimiento. Las variables discretas se presentan como n (%) y las continuas mediante media ± DE o mediana RIQ. Los valores evolutivos de biomarcadores séricos se compararon mediante test de Friedman. La fracciónde eyección del VI (FEVI) basal se comparó con la de los 3 meses del TPH mediante test de Wilcoxon. Resultados: se incluyeron 19 pacientes, 37% mujeres, de 43,8 ± 15,7 años. No se detectaron modificaciones significativas de la FEVI en los controles evolutivos. En ningún caso se observó aumento de la TnI. Los valores de BNP aumentaron en 6 pacientes (32%), con diferencias significativas al mes postrasplante (basal: 13,6 1;6,1-30,9 vs. primer mes: 38,9 16,3-120,0 pg/ml, p = 0,036); con una mayor elevación en aquellos pacientes que recibieron antimetabolitos vs. otros fármacos (basal: 13,6 1;6,1-30,9 vs. al primer mes: 67,0 ;21,3-174,9 pg/ml, p = 0,039). El aumento de BNP no se asoció con el riesgo CV. Conclusión: la liberación de BNP posterior al TPH es un fenómeno frecuente (32% de los pacientes), alcanza un máximo al mes, independientemente de la FEVI. El subgrupo de pacientes que recibió antimetabolitos presentó una mayor liberación precoz de BNP.

Introduction: cardiovascular (CV) diseases are the leading cause of death in those who survive cancer. Although hematopoietic stem cell transplantation (HSCT) is associated with diverse grades of cardiotoxicity, these complications have been poorly characterized. Objective: to analyze the release profile of myocardial biomarkers as a potential subclinical marker of cardiotoxicity in patients undergoing HSCT. Material and method: descriptive, analytical, prospective, cross-sectional, single-center study, recruiting patients referred to the cardio-oncology polyclinic, with indication for HSCT in October 2018-March 2020. Clinical, ECG, biochemical and imaging controls were performed according to the algorithm of follow-up. The evolutionary values of serum biomarkers were compared using the Friedman test. Baseline LVEF was compared with that of 3 months after HSCT using the Wilcoxon test. Results: 19 patients were included, 37% women, aged 43.8 ± 15.7 years. No changes in LVEF were detected. In no case was an increase in TnI observed. BNP values increased in 6 patients (32%), with significant differences one month after transplantation (baseline: 13.6 ;6.1-30.9 vs. first month: 38.9 ;16.3-120.0, p = 0.036), detecting a greater elevation in those patients who received antimetabolites vs. other rugs (baseline: 13.6 ;6.1-30.9 vs. at the first month: 67.0 21.3-174.0, p = 0.039). The increase in BNP was not associated with CV risk. Conclusion: BNP release after HSCT is frequent (32% of our patients), reaching a maximum at one month, regardless of LVEF. The subgroup of patients who received antimetabolites had a greater early release of BNP.

Introdução: as doenças cardiovasculares (CV) são a principal causa de morte em pessoas que sobrevivem ao câncer. Embora o transplante de células-tronco hematopoéticas (TCTH) esteja associado à diverso grado de cardiotoxicidade, essas complicações têm sido mal caracterizadas. Objetivo: analisar o perfil de liberação de biomarcadores miocárdicos como potenciais marcadores subclínicos de cardiotoxicidade em pacientes submetidos ao TCTH. Material e método: estudo descritivo, analítico, prospectivo, transversal, unicéntrico, com recrutamento de pacientes encaminhados à policlínica de cardio-oncologia, com indicação de TCTH de outubro de 2018 a março de 2020. Foram realizados controles clínicos, eletrocardiográficos, bioquímicos e de imagem de acordo com o algoritmo de acompanhamento. Os valores evolutivos dos biomarcadores séricos foram comparados pelo teste de Friedman. A FEVE basal foi comparada com a de 3 meses após o TCTH usando o teste de Wilcoxon. Resultados: foram incluídos 19 pacientes, 37% mulheres, com idade de 43,8 ± 15,7 anos. Nenhuma mudança na LVEF foi detectada. Em nenhum caso foi observado um aumento de TnI. Os valores de BNP aumentaram um mês após o transplante (linha de base: 13,6 6,1-30,9; vs. primeiro mês: 38,9 16,3-120,0, p = 0,036), se detectou uma maior elevação nos pacientes que receberam antimetabólitos vs. outros medicamentos (linha de base: 13,6 ;6,1-30,9; vs. no primeiro mês: 67,0 ;21,3-174,0;, p = 0,039). O aumento do BNP não foi associado ao risco CV. Conclusão: a liberação do BNP após o TCTH é frequente (32% de nossos pacientes), podendo chegar a no máximo um mês, independente da FEVE. O subgrupo de pacientes que recebeu antimetabólitos apresentou maior liberação precoce de BNP.

Immune reconstitution after allogenic stem cell transplantation: An observational study in pediatric patients

Abstract Introduction The immune reconstitution (IR) after the allogenic hematopoietic stem cell transplantation (allo-HSCT) is a progressive process intrinsically correlated to the therapeutic success. It is essential to understand the interfering factors in IR to prevent the HSCT-related mortality. Methods We retrospectively evaluated the clinical outcomes, absolute lymphocyte counts (ALCs) and lymphocyte subtypes at different time-points of 111 pediatric patients with allogeneic HSCT for malignant and non-malignant diseases from 2013 to 2018. Results The ALCs gradually increased on D+30, D+100, and D+180 (medians 634/μL, 1022/μL and 1541/μL, respectively). On D+100, the CD3+CD8+ achieved the highest recovery rate (68%), followed by the CD16+CD56+ (47%), CD3+CD4+ (39%) and CD19+ (8%). The adequate ALC recovery was associated with age < 8 years, bone marrow grafts, myeloablative conditioning, non-use of serotherapy and non-haploidentical donors. The ALC and CD3+CD8+ on D+100 counts were higher in patients with the cytomegalovirus infection. The CD3+CD4+ recovery was associated with an age < 8 years, a non-malignant disease and a lower incidence of acute graft-versus-host disease ≥ grade 2. Furthermore, the ALC recovery on D+100 resulted in a higher overall survival, regardless of the disease type (HR 3.65, 1.05 - 12.71, p= 0.04). Conclusion Several factors influenced the IR after the allo-HSCT. The ALC ≥ 500/μL on D+100 was a simple IR predictor of survival, easily available to resource-limited centers.

Effects of a physiotherapeutic protocol in cardiorespiratory, muscle strength, aerobic capacity and quality of life after hematopoietic stem cell transplantation

Abstract Objective To analyze the effects of hospital cardiorespiratory physical therapy protocol on the functional capacity and quality of life of patients submitted to hematopoietic stem cell transplantation (HSCT). Methods From January to December 2019, bilateral dynamometry, Manovacuometry and Ventilometry, peak expiratory flow "Peak Flow", 6-min walk test (6MWT), SF-36 Quality of Life Questionnaire and Visual Analog Scale (VAS) were applied in patients who have undergone an allogeneic or autologous hematopoietic stem cell transplantation (HSCT), pre-conditioning (initial evaluation) and pre-discharge (final evaluation). The patients were submitted to an intervention protocol, consisting of aerobic training, muscle strengthening and respiratory muscle training, between the two assessments. Results 29 patients were enrolled in the study and 24 (83%) completed all procedure. Myeloablative and reduced intensity conditioning were performed in 89.6% and 10.4%, respectively; 17 (58%) patients have undergone an autologous HSCT; 10 (35%) identical related allogeneic HSCT, and 2 (7%) haploidentical allogeneic HSCT. The median number of interventions per patient was 3 (1-9). A decreasing in the right and left dynamometry (p ≤ 0.0001 and 0.002, respectively) and, also in the distance covered in the 6MWT (p = 0.004), was observed after HSCT. There was no significant difference in respiratory muscle strength, quality of life and fatigue sensation. Conclusion Cardiorespiratory rehabilitation can preserve functional capacity and quality of life.

Temporal trends in Hematopoietic Stem Cell Transplantation in Argentina: regional differences that mirror the global reality

Introduction: Hematopoietic stem cell transplantation is the only curative treatment for many disorders and international data shows a growing trend. Method: We aimed to evaluate the temporal trends in HSCT transplant rates in Argentina. A time-series analysis was performed for the period 2009 to 2018 using the national database from the National Central Coordinating Institute for Ablations and Implants. Crude and standardized transplant rates were calculated. A permutation joinpoint regression model analysis was used to identify significant changes over time. Results: Altogether, 8,474 transplants were reported to INCUCAI by 28 centers (autologous 67.5%); the main indication was multiple myeloma (30%). The WHO age-sex standardized HSCT rates for the entire country were 153.3 HSCT/10 million inhabitants (95% CI 141.7 −165.8) in 2009 and 260.1 HSCT/10 million inhabitants (95% CI 245.5−275.5) in 2018. There was a large gap in HSCT rates among the states and regions. The transplant rate was higher for autologous transplants throughout the years. Within the allogeneic group, the related donor transplant rate was higher than the unrelated donor transplant rate. The joinpoint regression analysis of HSCT rates for the whole country over time showed an observed annual percentage change of 6.3% (95% CI 5.4-7.3; p < 0.01). No changes were observed for unrelated donors during the study period. Conclusions: Age-sex standardized HSCT rates in Argentina are increasing, mainly due to autologous and family donor allogeneic transplants. A wide variation across the country was found, demonstrating differences in the access to transplantation among Argentine regions

Pacientes con enfermedad injerto contra huésped e infecciones asociadas al rechazo de trasplante / Patients with graft-versus-host disease and infections associated with transplant rejection

Introducción: durante muchos años las células madre hematopoyéticas han sido el tratamiento para muchos trastornos hematológicos, pero su eficacia está limitada por la enfermedad injerto contra huésped (EICH); una de las principales complicaciones del trasplante alogénico se encuentra asociado con morbilidad y mortalidad, por lo tanto, la prevención es importante para el éxito del trasplante alogénico. Objetivo: realizar una revisión acerca del reconocimiento clínico de una EICH para brindar el tratamiento correcto y evitar ciertas complicaciones, como infecciones que llevan al rechazo del injerto y ponen en riesgo la calidad de vida del paciente. En la mayoría de los casos las pruebas de laboratorio como biomarcadores y biopsias, son buenos predictores para procesos biológicos o patológicos que confirmen el diagnóstico y establezcan el estadio de la enfermedad. Metodología: se realizó una revisión bibliográfica en bases de datos, tales como Pubmed y ClinicalKey, con base en los siguientes términos MeSH: cirugía, mortalidad, patología, complicaciones, virología. Conclusión: la prevención y tratamiento de esta enfermedad predispone a infecciones y diferentes complicaciones que ponen en riesgo la vida del paciente.

Introduction: For many years hematopoietic stem cells have been the treatment for many hematological disorders, but their efficacy is limited by graft-versus-host disease (GVHD), one of the main complications of allogeneic transplantation associated with morbidity and mortality; therefore, prevention is important for the success of allogeneic transplantation. Objective: To review the clinical recognition of GVHD in order to provide the correct treatment and avoid certain complications, such as infections that lead to graft rejection and jeopardize the patient's quality of life. In most cases laboratory tests such as biomarkers and biopsies are good predictors of biological or pathological processes that confirm the diagnosis and establish the stage of the disease. Methodology: A bibliographic review was carried out in databases such as Pubmed and ClinicalKey based on the following MeSH terms: surgery, mortality, pathology, complications, virology. Conclusion: The prevention and treatment of this disease predisposes to infections and different complications that put the patient's life at risk.

Utilização de Cateter Central de Inserção Periférica para Realização de Transplante de Células-Tronco Hematopoiéticas / Use of Peripherally Inserted Central Catheter to Perform Hematopoietic Stem Cell Transplantation / Uso de Catéter Central de Inserción Periférica para Realizar Trasplante de Células Madre Hematopoyéticas

Introdução: O acesso venoso seguro é indispensável aos pacientes no transplante de células-tronco hematopoiéticas (TCTH), e o enfermeiro participa de todo o seu processo de decisão e manutenção. Objetivo: Sistematizar a experiência de enfermeiros na utilização do cateter central de inserção periférica (CCIP) para a realização de TCTH em um centro de transplante de medula óssea de uma instituição pública, referência nacional em oncologia. Método: Estudo descritivo elaborado a partir da sistematização criada por Oscar Jara Holliday. Resultados: A experiência de utilização do CCIP, para a realização de TCTH, no centro de transplante estudado, teve início em 2017. Durante a implementação da nova rotina, surgiram obstáculos relacionados à resistência da equipe, aceitação dos pacientes, disponibilidade de material adequado e profissionais habilitados. Pensando na assistência terapêutica endovenosa de qualidade e segura para o paciente, registrou-se a marca de 130 CCIP implantados nos últimos seis anos (2017-2022), o que representou 32% do total de cateteres utilizados no último ano para realização de transplantes autólogos, alogênicos aparentados, alogênicos não aparentados e haploidênticos. Outro dado referente ao sucesso desse procedimento nesse centro mostra que 80% dos CCIP foram retirados por motivo de alta e os outros 20% por trombose (2%); obstrução (8%); óbito (5%); e febre (5%). Conclusão: Observa-se que, apesar das dificuldades enfrentadas, a implementação e a utilização de CCIP para infusão de células-tronco hematopoiéticas têm apresentado bons resultados e contribuem para a prática de obtenção de acesso vascular seguro no TCTH

Introduction: Safe venous access is essential for patients undergoing hematopoietic stem cell transplantation (HSCT) and the nurse participates in the entire decision-making process and maintenance. Objective: To systematize the experience of nurses in using the peripherally inserted central catheter (PICC) to perform HSCT in a bone marrow transplant center of a public institution that is a national oncology reference. Method: Descriptive study based in Jara Holliday's systematization. Results: The experience of using PICC to perform HSCT at the transplant center investigated began in 2017. During the implementation of the new routine, obstacles related to the team's resistance, patient acceptance, availability of adequate material and qualified professionals were detected. Regarding quality and safe intravenous therapeutic assistance for the patient, 130 PICC have been implanted in the last six years (2017-2022), accounting for 32% of the total number of catheters used to perform autologous, related allogeneic, unrelated allogeneic and haploidentical transplants in the last year. 80% of PICC was removed due to hospital discharge and 20% due to thrombosis (2%), obstruction (8%), death (5%) and fever (5%) confirming the success of this procedure in the center investigated. Conclusion: Despite the difficulties, the implementation and use of PICC for the infusion of hematopoietic stem cells has shown good results and contributed to obtaining safe vascular access in HSCT

Introducción: El acceso venoso seguro es fundamental para los pacientes sometidos a trasplante de células madre hematopoyéticas (TCMH) y el personal de enfermería participa en todo el proceso de toma de decisiones y mantenimiento de este acceso. Objetivo: Sistematizar la experiencia de enfermeros en el uso del catéter central de inserción periférica (PICC) para realizar trasplante de células madre hematopoyéticas en un centro de trasplante de médula ósea de una institución pública de referencia nacional en oncología. Método: Estudio descriptivo elaborado a partir de la sistematización realizada por Oscar Jara Holliday. Resultados: La experiencia de utilizar el PICC para realizar el TCMH en el centro de trasplante estudiado se inició en 2017. Durante la implementación de la nueva rutina surgieron obstáculos relacionados con la resistencia del equipo, aceptación del paciente, disponibilidad de material adecuado y profesionales calificados. Pensando en una asistencia terapéutica intravenosa de calidad y segura para el paciente, nos lanzamos y elevamos la marca de 130 PICC implantados en los últimos seis años (2017-2022), lo que representó en el último año el 32% del total de catéteres utilizados para realizar trasplantes autólogos, alogénicos relacionados, alogénicos no relacionados y haploidénticos. Otro dato referente al éxito de este procedimiento en nuestro centro muestra que el 80% de los PICC se retiraron por alta y el otro 20% por trombosis (2%); obstrucción (8%); muerte (5%) y fiebre (5%). Conclusión: Al final de este informe, observamos que, a pesar de las dificultades enfrentadas, la implementación y el uso de PICC para la infusión de células madre hematopoyéticas mostró buenos resultados y contribuyó para la práctica de obtener un acceso vascular seguro en el TCMH

Can NLR be a biomarker for mucositis and gvhd in patients undergoing allogeneic HSCT?

Allogeneic hematopoietic stem cell transplantation (HSCT) is a treatment for many diseases; however, it can induce complications such as Oral Mucositis (OM) and Graft-versus- Host Disease (GVHD). The neutrophil-lymphocyte ratio (NLR) is a peripheral biomarker of systemic inflammation and an independent prognostic factor for several inflammatory diseases. Aim: This study aimed to evaluate the association of NLR with OM and GVHD in patients undergoing allogeneic HSCT. Methods: Patients who underwent allogeneic HSCT at the Bone Marrow Transplant Service of the Hospital de Clínicas Complex of the Federal University of Paraná were included in the study. Socio-demographic data and blood counts were collected from patients' medical records. The NLR was calculated and associated with OM and GVHD. Results: 45 patients were included in the study. Although NLR was higher in patients with OM and oral GVHD, no statistical difference was observed, and no relationship between OM and GVHD with NLR could be stated. Conclusion: Although both OM and GVHD are associated with an inflammatory response as well as the immune system, it was not associated with NLR. Further investigation considering other variables related to HSCT might find possible associations, as it could favor patient management and prevention

Cuidados de enfermagem no transplante autólogo de células-tronco hematopoiéticas em pacientes com mieloma múltiplo / Atención de enfermería en el trasplante autólogo de células madre hematopoyéticas en pacientes con mieloma múltiple / Nursing care in autologous hematopoietic stem cell transplantation in multiple myeloma patients

Objetivos: Identificar as evidências disponíveis sobre o cuidado de enfermagem no transplante autólogo de células-tronco hematopoiéticas em pacientes com mieloma múltiplo no ambiente hospitalar, relacionar os cuidados identificados na literatura às teorias de enfermagem, quais aspectos têm sido priorizados quanto ao cuidado da enfermagem e compreender em que fase do tratamento o enfermeiro é mais atuante. Método: Estudo de Revisão de Escopo, com busca realizada nas bases de dados LILACS e MEDLINE via PubMed, no mês de junho de 2021, 10 artigos foram identificados. Resultados: Tratam de cuidados relacionados a procedimentos específicos e demonstram a importância que o enfermeiro tem de atuar no controle do ambiente, bem-estar, apoio emocional e da educação em saúde com estímulo à autonomia e ao autocuidado. Conclusão: Observam-se bases teóricas que devem estar presentes em todo o processo de cuidar, guiando a atuação do enfermeiro, que se mostra relevante em todo o processo de tratamento.

Objectives: To identify the available evidence on nursing care in autologous hematopoietic stem cell transplantation in patients with multiple myeloma in the hospital environment, to relate the care identified in the literature to nursing theories, which aspects have been prioritized in terms of nursing care and understand at which stage of treatment the nurse is most active. Method: Scope Review Study, with search performed in LILACS and MEDLINE databases via PubMed, in June 2021, 10 articles were identified. Results: They deal with care related to specific procedures and demonstrate the importance that nurses have to act in the control of the environment, well-being, emotional support and health education, encouraging autonomy and self-care. Conclusion: There are theoretical bases that must be present throughout the care process, guiding the performance of nurses, which proves to be relevant throughout the treatment process.

Objetivos: Identificar la evidencia disponible sobre el cuidado de enfermería en el trasplante autólogo de células madre hematopoyéticas en pacientes con mieloma múltiple en el ámbito hospitalário y relacionar con las teorías de enfermería, aspectos que se han priorizado en cuanto al cuidado de enfermería y comprender en qué etapa del tratamiento la enfermera está más activa. Método: Estudio Scope Review, con búsqueda realizada en bases de datos LILACS y MEDLINE vía PubMed, en junio de 2021, se identificaron 10 artículos. Resultados: Abordan cuidados relacionados con procedimientos específicos y demuestran la importancia que tiene el enfermero de actuar en el control del entorno, el bienestar, el apoyo emocional y la educación para la salud, fomentando la autonomía y el autocuidado. Conclusión: Existen bases teóricas que deben estar presentes en todo el proceso de atención, orientando el desempeño de las enfermeras, las cuales son relevantes en todo el proceso de tratamiento.

Assessment of oral health condition in recipients of allogeneic hematopoietic cell transplantation

ABSTRACT Objective: The objective of this study was to evaluate the oral health condition and dental treatments performed in patients in pre-allogeneic HCT. Method: The records of patients treated during 2018 at a Brazilian HCT service were reviewed. The following oral health data were obtained: 1. Decayed, missing and filled teeth / correlated index for primary dentition (DMFT/dmft); 2. Quality of oral hygiene and 3. Dental pathologies: 3.1 Periodontal infectious focus, 3.2 Endodontic infectious focus and 3.3 Carie incidence. All dental procedures performed were surveyed. Results: Thirty-three patients were included, with a mean age of 28.42 (±16.37), 20 male (60%) and 13 female. The average DMFT/dmft found in this study was 10.24 (± 8.37), similar to the index found in the population in southeastern Brazil. The younger study population presented a DMFT/dmft considered high, when compared to the general population. A total of 27.2% of the patients had active caries lesions, 33.3%, foci of periodontal infection, 15.1%, endodontic infectious focus and 40%, poor oral hygiene. Almost half of the patients (48.4%) had to undergo dental intervention, 24.2% needing periodontal scaling, 21.2%, fillings and 12.1%, tooth extractions. Conclusion: We conclude that the studied population had an important incidence of dental pathologies and infectious conditions that could complicate throughout HCT, especially in younger patients, therefore presenting a high demand for dental treatment in the pre-HCT. Studies that assess the impact of dental conditioning on the outcomes of HCT with an emphasis on dental infectious complications, days of hospitalization and survival are necessary."

Alterações na qualidade de vida de adultos com câncer hematológico em cinco anos após o transplante de células-tronco hematopoéticas / Changes in the quality of life of adults with hematological cancer in five years after hematopoietic stem cell transplantation

Resumo: O objetivo desta tese foi avaliar as alterações da qualidade de vida relacionada à saúde dos pacientes adultos com câncer hematológico, submetidos ao transplante de células-tronco hematopoéticas, nos primeiros cinco anos após o procedimento. Trata-se de um estudo quantitativo, longitudinal, observacional e analítico, realizado em hospital público do sul do Brasil, referência na América Latina para esta modalidade de tratamento. Foram incluídos 55 participantes com idade igual ou superior a 18 anos, que se submeteram a esta terapia. A coleta de dados ocorreu de setembro de 2013 a janeiro de 2021, com avaliações em dez etapas: pré-transplante (antes de iniciar o condicionamento), pancitopenia, pré-alta hospitalar, após 100 dias, após 180 dias, Follow up 1 (após 360 dias), e anualmente até completar cinco anos da realização do procedimento. Foram aplicados um instrumento para coleta de dados sociodemográficos e clínicos e os questionários de Qualidade de vida relacionada à saúde Quality of life Questionnaire Core 30 e Functional Assessment of Cancer Therapy - Bone Marrow Transplant, ambos traduzidos, adaptados e validados para o português - Brasil. Em relação ao diagnóstico, as leucemias estão presentes em 65% dos casos; quanto à modalidade de tratamento, o transplante de células-tronco alogênico foi realizado em 71% dos pacientes. No que diz respeito aos óbitos, a causa de maior incidência foi por recidiva da doença (44%), e o maior número ocorreu no primeiro ano (37%). A qualidade de vida global (56,6/100) e geral (90,7/148) apresentou os menores escores na etapa de pancitopenia, com melhores índices no quinto ano, (80,4/100) e (116,1/148), respectivamente. A análise com o modelo linear generalizado misto evidenciou alterações significativas nos escores dos domínios de qualidade de vida relacionada à saúde entre as etapas ao longo do tempo. Foi comprovada a hipótese de que os pacientes com melhores escores nos domínios de qualidade de vida relacionada à saúde observados no início do tratamento têm maior sobrevida. Os resultados do estudo inferem as correlações entre os domínios mensurados e encontram, assim, sustentação no modelo conceitual teórico utilizado. As contribuições consistem em reafirmar a dimensionalidade do constructo qualidade de vida relacionada à saúde, além de agregar conhecimento acerca das alterações autopercebidas pelos pacientes durante o tratamento.

Abstract: The objective of this thesis was to evaluate the changes in the health-related quality of life of adult patients with hematological cancer undergoing hematopoietic stem cell transplantation in the first five years after the procedure. This is a quantitative, longitudinal, observational and analytical study carried out in a public hospital in southern Brazil, a reference in Latin America for this treatment modality. We included 55 participants aged 18 years and over, who underwent this therapy. Data collection took place from September 2013 to January 2021, with evaluations in ten stages: pre-transplantation (before starting conditioning), pancytopenia, pre-hospital discharge, after 100 days, after 180 days, Follow up 1 ( after 360 days), and annually until completing five years of the procedure. An instrument for collecting sociodemographic and clinical data and the Health-related Quality of life Questionnaire Core 30 and Functional Assessment of Cancer Therapy - Bone Marrow Transplant questionnaires were applied, both translated, adapted and validated for Portuguese - Brazil. Regarding diagnosis, leukemias are present in 65% of cases; regarding the treatment modality, allogeneic stem cell transplantation was performed in 71% of the patients. With regard to deaths, the cause of highest incidence was disease recurrence (44%), and the highest number occurred in the first year (37%). The global (56.6/100) and general (90.7/148) quality of life had the lowest scores in the pancytopenia stage, with better rates in the fifth year (80.4/100) and (116.1/148), respectively. The analysis with the mixed generalized linear model showed significant changes in the scores of the health-related quality of life domains between the stages over time. The hypothesis was confirmed that patients with better scores in the domains of health-related quality of life observed at the beginning of treatment have greater survival. The study results infer the correlations between the measured domains and thus find support in the theoretical conceptual model used. The contributions consist of reaffirming the dimensionality of the health-related quality of life construct, in addition to adding knowledge about the self-perceived changes by patients during treatment.

Resumen: El objetivo de esta tesis fue evaluar los cambios en la calidad de vida relacionada con la salud de pacientes adultos con cáncer hematológico, sometidos a trasplante de células madre hematopoyéticas, en los primeros cinco años después del procedimiento. Se trata de un estudio cuantitativo, longitudinal, observacional y analítico realizado en un hospital público del sur de Brasil, referencia en América Latina para esta modalidad de tratamiento. Se incluyeron 55 participantes mayores de 18 años que se sometieron a esta terapia. La recolección de datos ocurrió de septiembre de 2013 a enero de 2021, con evaluaciones en diez etapas: pretrasplante (antes de iniciar el acondicionamiento), pancitopenia, alta prehospitalaria, después de 100 días, después de 180 días, Seguimiento 1 (después de 360 días), y anualmente hasta completar cinco años del procedimiento. Se aplicó un instrumento de recolección de datos sociodemográficos y clínicos y los cuestionarios Health-related Quality of life Questionnaire Core 30 y Functional Assessment of Cancer Therapy - Bone Marrow Transplant, ambos traducidos, adaptados y validados para portugués - Brasil. En cuanto al diagnóstico, las leucemias están presentes en el 65% de los casos; en cuanto a la modalidad de tratamiento, se realizó trasplante alogénico de células madre en el 71% de los pacientes. En cuanto a las defunciones, la causa de mayor incidencia fue la recidiva de la enfermedad (44%) y el mayor número se produjo en el primer año (37%). La calidad de vida global (56,6/100) y general (90,7/148) tuvieron las puntuaciones más bajas en la etapa de pancitopenia, con mejores tasas en el quinto año (80,4/100) y (116,1/148), respectivamente. El análisis con el modelo lineal generalizado mixto mostró cambios significativos en las puntuaciones de los dominios de calidad de vida relacionada con la salud entre las etapas a lo largo del tiempo. Se confirmó la hipótesis de que los pacientes con mejores puntajes en los dominios de calidad de vida relacionada con la salud observados al inicio del tratamiento tienen mayor sobrevida. Los resultados del estudio infieren las correlaciones entre los dominios medidos y así encuentran apoyo en el modelo teórico conceptual utilizado. Los aportes consisten en reafirmar la dimensionalidad del constructo calidad de vida relacionada con la salud, además de sumar conocimientos sobre los cambios autopercibidos por los pacientes durante el tratamiento.

Reactivación de la enfermedad de Chagas después de trasplante autólogo de progenitores hematopoyéticos. Reporte de caso y revisión de la literatura / Chagas disease reactivation after autologous stem cell transplant. Case report and literature review

Introducción. La enfermedad de Chagas es una parasitosis endémica en Latinoamérica transmitida por triatominos. Está asociada a factores de riesgo como la pobreza y la ruralidad. Después de la infección aguda, un tercio de los pacientes presenta compromiso del corazón, el aparato digestivo o el sistema nervioso central, en tanto que los dos tercios restantes no presentan este tipo de compromiso secundario. La inmunosupresión farmacológica rompe el equilibrio entre el sistema inmunitario y el parásito, lo cual favorece su reactivación. Caso clínico. Se presenta el caso de un hombre de 58 años procedente de un área rural colombiana, con diagnóstico de mieloma múltiple resistente a los fármacos de primera línea de tratamiento, que requirió un nuevo esquema de quimioterapia y consolidación con trasplante autólogo de células madre. Después del trasplante, presentó neutropenia febril. Los estudios microbiológicos iniciales fueron negativos. En el frotis de sangre periférica, se demostraron tripomastigotes y se diagnosticó enfermedad de Chagas aguda posterior al trasplante. Se inició el tratamiento con benznidazol. La evolución del paciente fue satisfactoria. Conclusiones. La serología positiva para Chagas previa a un trasplante obliga a descartar la reactivación de la enfermedad en caso de neutropenia febril. Se requieren más estudios para determinar las herramientas que permitan estimar la probabilidad de reactivación de la enfermedad y decidir sobre la mejor opción de relación entre costo, riesgo y beneficio de la terapia profiláctica.

Introduction: Chagas disease is an endemic parasitic infection in Latin America transmitted by triatomines. It is associated with risk factors such as poverty and rurality. After acute infection, a third of patients will present target organ involvement (heart, digestive tract, central nervous system). The remaining two thirds remain asymptomatic throughout their life. Pharmacological immunosuppression breaks the balance between the immune system and the parasite, favoring its reactivation. Clinical case: We present the case of a 58-year-old man from a Colombian rural area with a diagnosis of multiple myeloma refractory to the first line of treatment who required a new chemotherapy scheme and consolidation with autologous stem cell transplant. During the post-transplant period, he suffered from febrile neutropenia. Initial microbiological studies were negative but the peripheral blood smear evidenced trypomastigotes in blood. With a diagnosis of acute Chagas disease in a post-transplant patient, benznidazole was started. The evolution of the patient was satisfactory. Conclusions: Positive serology prior to transplantation makes it necessary to rule out reactivation of the pathology in the setting of febrile neutropenia. More studies are required to determine tools for estimating the probability of reactivation of the disease and defining the best cost-risk-benefit relation for the prophylactic therapy.