Quality of life and changes in the social dimension of hematopoietic stem cell transplants recipients.

OBJECTIVES: to assess/correlate health-related quality of life with the social dimension of hematopoietic, autologous and allogeneic stem cell transplant patients in the three years post-transplant. METHODS: longitudinal, observational study with 55 patients, in a reference hospital in Latin America, from September 2013 to February 2019, using the Quality of Life Questionnaire-Core and Functional Assessment Cancer Therapy Bone Marrow Transplantation. RESULTS: A total of 71% underwent allogeneic transplantation. The social dimension had low averages since the baseline stage (55, 21) and low scores (56) for quality of life in pancytopenia. There was a significant positive correlation between social dimension, quality of life in pancytopenia (p<0.01) and follow-up after hospital discharge (p<0.00). There is a significant difference (p<0.00) throughout the stages, however, not in terms of the type of transplant (p>0.36/0.86). CONCLUSIONS: patients with better assessments in the social dimension have a better quality of life. Interventions focusing on the multidimensionality of the quality of life construct are necessary.

Quality of life and changes in the social dimension of hematopoietic stem cell transplants recipients / Calidad de vida y cambios en el dominio social de los receptores de trasplantes de células madre hematopoyéticas / Qualidade de vida e alterações no domínio social de transplantados de células-tronco hematopoéticas

ABSTRACT Objectives: to assess/correlate health-related quality of life with the social dimension of hematopoietic, autologous and allogeneic stem cell transplant patients in the three years post-transplant. Methods: longitudinal, observational study with 55 patients, in a reference hospital in Latin America, from September 2013 to February 2019, using the Quality of Life Questionnaire-Core and Functional Assessment Cancer Therapy Bone Marrow Transplantation. Results: A total of 71% underwent allogeneic transplantation. The social dimension had low averages since the baseline stage (55, 21) and low scores (56) for quality of life in pancytopenia. There was a significant positive correlation between social dimension, quality of life in pancytopenia (p<0.01) and follow-up after hospital discharge (p<0.00). There is a significant difference (p<0.00) throughout the stages, however, not in terms of the type of transplant (p>0.36/0.86). Conclusions: patients with better assessments in the social dimension have a better quality of life. Interventions focusing on the multidimensionality of the quality of life construct are necessary.

RESUMEN Objetivo: evaluar/correlacionar la calidad de vida relacionada a la salud con el ámbito social de pacientes trasplantados de células madre hematopoyéticas, autólogas y alogénicas, tres años post trasplante. Métodos: se trata de un estudio longitudinal, observacional, llevado a cabo en un hospital referencial de América Latina con 55 pacientes entre septiembre de 2013 y febrero de 2019, utilizando la Escala de Calidad de Vida-Núcleo y la Evaluación funcional de la terapia del cáncer: trasplante de médula ósea. Resultados: el 71% realizó un trasplante alogénico; el ámbito social presentó promedios bajos desde la etapa inicial (55, 21) y puntuaciones bajas (56) en cuanto a la calidad de vida en la pancitopenia. Se observó una correlación positiva significativa entre el ámbito social, la calidad de vida en la pancitopenia (p<0,01) y el seguimiento después del alta hospitalaria (p<0,00). Hubo una diferencia significativa (p<0,00) a lo largo de los estadios, pero no en lo que respecta a la modalidad del trasplante (p>0,36/0,86). Conclusiones: aquellos pacientes con mejor evaluación en el ámbito social presentan mejor calidad de vida. Es necesario realizar intervenciones que se centren en la multidimensionalidad de la construcción de la calidad de vida.

RESUMO Objetivo: avaliar/correlacionar a qualidade de vida relacionada à saúde com o domínio social de pacientes transplantados de células-tronco hematopoéticas, autólogo e alogênico, nos três anos pós-transplante. Métodos: estudo longitudinal, observacional, com 55 pacientes, num hospital referência da América Latina, de setembro de 2013 a fevereiro de 2019, com o Quality of Life Questionnarie-Core e Functional Assessment Cancer Therapy Bone Marrow Transplantation. Resultados: 71% realizaram transplante alogênico. O domínio social apresentou médias baixas desde a etapa basal (55, 21) e escores baixos (56) para a qualidade de vida na pancitopenia. Houve correlação positiva significativa entre domínio social, qualidade de vida na pancitopenia (p<0,01) e acompanhamento após alta hospitalar (p<0,00). Observa-se diferença significativa (p<0,00) ao longo das etapas, porém, não quanto à modalidade de transplante (p>0,36/0,86). Conclusões: pacientes com melhor avaliação no domínio social apresentam melhor qualidade de vida. Intervenções com foco na multidimensionalidade do constructo qualidade de vida são necessárias.

Living donor liver transplantation for idiopathic portal hypertension with extrahepatic portal vein stenosis and splenic artery aneurysms: a case report and review of the literature.

BACKGROUND: Idiopathic portal hypertension (IPH) generally has a good prognosis and rarely results in liver transplantation. Furthermore, there are few reports of living donor liver transplantation (LDLT) for IPH with extrahepatic portal vein stenosis. CASE PRESENTATION: We report the case of a 51-year-old female patient diagnosed with IPH more than 20 years ago. She suffered severe jaundice, massive ascites, and encephalopathy at the time of her visit to our hospital. The patient's extrahepatic portal vein showed a scar-like stenosis, and the portal flow was completely hepatofugal. Collateral circulation such as the splenorenal shunt was well developed, and multiple splenic artery aneurysms up to 2 cm were observed in the splenic hilum. Her Model for End-Stage Liver Disease score increased to over 40 because of renal dysfunction, requiring temporary dialysis. We performed LDLT using her husband's right lobe graft and splenectomy. The extrahepatic stenotic portal vein was completely resected, and the superficial femoral vein (SFV) graft collected from the recipient's right leg was used for portal reconstruction as an interposition graft. Although the clinical course after LDLT had many complications, the patient was discharged on postoperative day 113 and has been fine for 2 years after LDLT. Histopathologically, the explanted liver had obliterative portal venopathy, nodular regenerative hyperplasia, and incomplete septal cirrhosis. CONCLUSION: This case showed that severe IPH is occasionally associated with extrahepatic portal vein stenosis and can be treated with LDLT with portal vein reconstruction using an interposition graft. It was also suggested that the SFV is a useful choice for the interposition graft.

Individualized concept for the treatment of autosomal recessive polycystic kidney disease with end-stage renal disease.

Management of children with autosomal recessive polycystic kidney disease (ARPKD) who develop end-stage renal disease (ESRD) remains challenging because of concomitant liver disease. Patients with recurrent cholangitis are candidates for liver-kidney transplantation, while the treatment for patients with splenomegaly and pancytopenia due to portal hypertension is controversial. Herein, we report 7 children who were treated using an individualized treatment strategy stratified by liver disease. Two patients with recurrent cholangitis underwent sequential liver-kidney transplantation, while 4 patients with splenomegaly and pancytopenia but without recurrent cholangitis underwent splenectomy followed by isolated kidney transplantation. The remaining patient, who did not have cholangitis and pancytopenia, underwent isolated kidney transplantation. Blood cell counts were normalized after splenectomy was performed at the median age of 8.7 (range, 7.4-11.7) years. Kidney transplantation was performed at the median age of 8.8 (range, 1.9-14.7) years in all patients. Overwhelming post-splenectomy infections and cholangitis did not occur during the median follow-up period of 6.3 (range, 1.0-13.2) years. The estimated glomerular filtration rate at the last follow-up was 53 (range, 35-107) mL/min/1.73 m2 . No graft loss occurred. Our individualized treatment strategy stratified by recurrent cholangitis and pancytopenia can be a feasible strategy for children with ARPKD who develop ESRD and warrants further evaluation.

Bone marrow failure and extramedullary hematopoiesis in McCune-Albright syndrome.

In fibrous dysplasia/McCune-Albright syndrome (FD/MAS), bone and bone marrow are, to varying degrees, replaced by fibro-osseous tissue typically devoid of hematopoietic marrow. Despite the extensive marrow replacement in severely affected patients, bone marrow failure is not commonly associated with FD/MAS. We present a 14-year-old girl with FD/MAS, who developed pancytopenia and extramedullary hematopoiesis (EMH) with no identified cause, in the setting of iatrogenic thyrotoxicosis and hyperparathyroidism. Pancytopenia, requiring monthly blood transfusions, persisted despite multiple strategies to correct these endocrinopathies. Due to worsening painful splenomegaly, likely as a result of sequestration, splenectomy was performed. Following splenectomy, pancytopenia resolved and patient has since been transfusion-independent. We report the first detailed case of bone marrow failure and EMH in FD/MAS. The etiology of marrow failure is likely multifactorial and related to the loss of marrow reserve due to extensive polyostotic FD, exacerbated by iatrogenic thyrotoxicosis and hyperparathyroidism. Mini Abstract: A patient with fibrous dysplasia developed bone marrow failure and extramedullary hematopoiesis. The etiology likely involved loss of hematopoetic marrow space and uncontrolled endocrinopathies. Splenectomy was therapeutic.

[Role of splenectomy in the treatment of non-cirrhotic portal hypertension: about 3 cases]. / Place de la splénectomie dans la prise en charge de l'hypertension portale non cirrhotique: à propos de 3 cas.

Non-cirrhotic portal hypertension was first described by Guido BANTI in 1898 as a condition characterized by the association of portal hypertension with splenomegaly, anemia and healthy liver. The diagnosis was based on abdominal ultrasound, splenoportography and liver biopsy. Our study aimed to evaluate the role of splenectomy in non-cirrhotic portal hypertension. We conducted a retrospective study of 3 patients (2 women and 1 man) treated by our staff over the period January 2010 -September 2016. The diagnosis of idiopathic portal hypertension was based on the following criteria: portal hypertension, the presence of oesophageal varices associated with splenomegaly, the absence of cirrhosis or of other liver disorders responsible of portal hypertension. All patients underwent splenectomy. Outcome after splenectomy was marked by the standardization of clinical, radiological and biological signs of this disease associated with the absence of oesophageal varices recurrence. Splenectomy associated with ligation of oesophageal varices may be sufficient to treat this syndrome and especially its consequences without using splenorenal bypass.

Adverse Late and Long-Term Treatment Effects in Adult Allogeneic Hematopoietic Stem Cell Transplant Survivors.

Hematopoietic stem cell transplantation (HSCT) has become the standard of care for many malignant and nonmalignant hematologic diseases that don't respond to traditional therapy. There are two types: autologous transplantation (auto-HSCT), in which an individual's stem cells are collected, stored, and infused back into that person; and allogeneic transplantation (allo-HSCT), in which healthy donor stem cells are infused into a recipient whose bone marrow has been damaged or destroyed. There have been numerous advancements in this field, leading to marked increases in the number of transplants performed annually. This article--the first of several on cancer survivorship--focuses on the care of adult allo-HSCT survivors because of the greater complexity of their posttransplant course. The author summarizes potential adverse late and long-term treatment-related effects, with special focus on the evaluation and management of several cardiovascular disease risk factors that can occur either independently or concurrently as part of the metabolic syndrome. These risk factors are potentially modifiable with appropriate nursing interventions and lifestyle modifications.

Modified Sugiura Operation for Idiopathic Portal Hypertension with Bleeding Oesophageal Varices. A Case Report.

A case of a 36 years old man presenting massive upper GI bleeding due to oesophageal varices developed in the context of an idiopathic portal cavernoma and extensive porto-splenic thrombosis is discussed. He underwent a successful modified Sugiura operation (oesophago-gastric devascularisation and splenectomy [OGDS]) completed with interventional endoscopic treatment of residual oesophageal varices. The benefit of the modified Sugiura procedure proposed for the treatment of upper GI variceal bleeding developed in the context of splanchnic venous thrombosis is discussed. The procedure is a valid therapy in the treatment of symptomatic extra-hepatic hypertension when other options are inapplicable.

(99m)Tc disphosphonate uptake due to splenosis: incidental finding 60 years after splenectomy.

A 72-year-old man was referred for a triphasic bone scintigraphy to evaluate painful prosthetic implant of the knee. In all the study phases, images showed abnormal uptake close to the tibial portion of the implant. Interestingly, later whole-body images revealed high radiopharmaceutical accumulation in the left hypochondrium. Subsequent radiolabeled leukocyte scintigraphy confirmed radiotracer accumulation in the same localizations. As an important background the patient underwent splenectomy owing to Banti syndrome 60 years earlier. A Tc-labeled heat-damaged red blood cell scintigraphy was then performed, which confirmed radiopharmaceutical accumulation in the left hypochondrium being consistent with splenosis.

Primary follicular lymphoma of the spleen incidentally found in a patient with alcohol- and hepatitis C-related liver cirrhosis.

Primary splenic lymphoma is rare as non-Hodgkin lymphomas. Splenic infiltration of lymphoma cells may cause splenomegaly in many cases. However, splenomegaly is caused not only by tumor involvement but also by non-tumorous disorders. One of the most prevalent non-neoplastic causes is portal hypertension mostly due to liver cirrhosis. On the other hand, liver cirrhosis may underlie various extrahepatic manifestations including development of B-cell non-Hodgkin lymphomas. Here, we report a case of primary follicular lymphoma of the spleen in a patient with liver cirrhosis related to hepatitis C and alcohol. The lymphoma was incidentally found in an enlarged spleen resected palliatively to alleviate symptomatic pancytopenia of the patient. The main characteristic of our case is an incidental finding of a rare situation brought by careful pathological examination. Our case illustrates the importance to recognize a possibility of co-occurrence of chronic liver disease and extrahepatic lymphoma.

Transient effect of anti-CD20 therapy in a child with 22q11.2 deletion syndrome and severe steroid refractory cytopenias: a case report.

We report on the development of steroid-refractory recurrent cytopenias in a child with 22q11.2 deletion syndrome. His first hematological complication was autoimmune hemolytic anemia at 3 months of age. Thereafter, he developed severe autoimmune cytopenias of all 3 hematological lineages with poor response to steroids and intravenous immunoglobulin. At the age of 2½ years, a course of anti-CD20 therapy (Rituximab) was given with transient hematological recovery. Because of persistent symptoms, bone marrow transplantation from a matched unrelated donor was performed. Although the data in the use of anti-CD20 therapy in children with 22q11.2 deletion syndrome and autoimmune cytopenias are limited, our experience suggests its potential benefit.

[A case of laparoscopic partial hepatectomy and splenectomy for hepatocellular carcinoma and pancytopenia].

A 69-year-old woman with chronic hepatitis B and esophageal varices was admitted to our hospital because of a hepatocellular carcinoma( HCC) measuring 3 cm in segment S3. Computed tomography( CT) scan revealed splenomegaly, and the platelet count was 6.0×104/µL. Partial hepatectomy and splenectomy were performed sequentially under laparoscopic guidance in a right half-lateral decubitus position, using 7 working ports. The operation time was 237 min, and the amount of bleeding was 26 mL. Her postoperative course was uneventful, and she was discharged on the 10th day after the operation.

Successful second bone marrow transplantation in Omenn's syndrome after bone marrow aplasia: a case report.

Omenn's syndrome is a rare inherited variant of SCID. It is inevitably fatal, unless treated by bone marrow or stem cell transplantation. However, treatment-related complications and graft rejection are major obstacles to the success of transplantation. In this report, we describe an eight-month-old baby with Omenn's syndrome and disseminated BCGosis, who underwent allogenic BMT from his HLA-matched sister using anti-GVHD prophylaxis but without a conditioning regimen. Ten days after BMT, he developed acute GVHD involving the skin only (grade 1) with a flare of local BCGosis. Engraftment of 55% was demonstrated by FISH as early as the 11th day post-BMT. On day +48 post-BMT, he developed GVHD that was complicated by refractory pancytopenia, resistant to three doses of rituximab. Patient was re-transplanted (from the same donor) after receiving fludarabine and ATG, with successful engraftment and complete recovery from pancytopenia.

Effect of splenectomy on pancytopenia after a peripheral blood stem cell transplantation.

To analyze the effect of a splenectomy on pancytopenia after a peripheral blood stem cell transplantation (PBSCT) in chronic myeloid leukemia (CML) patients. Three CML patients diagnosed with splenomegaly in our department from 2002 to 2006 received a splenectomy after PBSCT. Patient 1, a 42-year-old male in chronic phase (CP), received an HLA-identical sibling allo-PBSCT. Patient 2, a 28-year-old female in aggressive phase (AP), received a PBSCT from her twin. Patient 3, a 26-year-old male in chronic phase (CP), received a PBSCT from an unrelated donor. The conditioning regimen included busulfan and cyclophosphamide (BU/CY2). Patients 1, 2, and 3 received splenectomies on days 168, 51, and 114, respectively. Bcr-abl transcripts were detected using the polymerase chain reaction (PCR). Chimerism was documented by PCR amplification of a variable number of tandem repeat (VNTR) polymorphrisms. Neither metrisone (2 mg/kg/day for 7 days), mycophenolic acid (MMF) (0.5 g twice daily for 1 month), high-dose γ-globulin (0.4 g/kg/day for 5 days), granulocyte colony-stimulating factor (G-CSF) therapy, nor erythropoietin (EPO) therapy had produced post-PBSCT hematopoiesis recovery. Patients 1 and 2 had 5% Ph-positive chromosomes while patient 3 exhibited graft-versus-host disease (GVHD). After receiving the splenectomy, all three had a rapid hematopoeitic recovery with no evidence of Ph-positive chromosomes, and patients 1 and 3 showed complete donor chimerism (CDC). Patient 1 developed chronic GVHD (cGVHD) on day 210 postsplenectomy that was treated successfully with prednisone. Patient 2 had acute GVHD on day 55 that was treated successfully with dexamethasone (10 mg), administered intravenously once a day for 3 days with good clinical response. To date, patients 1, 2, and 3 have survived postprocedure for 85, 49, and 25 months, respectively. Splenectomy is an effective option for the patients who have pancytopenia after PBSCT and the patients recovered a good graft function after splenectomy without procedure-related complication and with long-time survival. GVHD can develop in both allo-PBSCT and syngeneic PBSCT. A splenectomy after PBSCT may increase the risk of GVHD, enhance the effect of graft versus leukemia (GVL), promote CDC formation.

Focal nodular hyperplasia of the liver in patients previously treated for pediatric neoplastic diseases.

SUMMARY: The discovery of a liver nodule during postcancer follow-up suggests malignancy recurrence. However, patients previously treated for pediatric cancers are at greater risk of developing benign hepatic tumors, raising the problems of getting an accurate noninvasive diagnosis. Here we report 3 cases of focal nodular hyperplasia of the liver in patients previously treated for pediatric neoplastic diseases. Radiologic findings and needle-biopsies remained insufficient in 2 cases to rule out metastasis or a potentially threatening tumor. Only surgical resection led to positive diagnosis and prevented complications. However, a review of the literature shows that if the nodule presents with typical features of focal nodular hyperplasia a close imaging follow-up is all that is recommended.

Splenomegaly, hypersplenism and peripheral blood cytopaenias in patients with classical Anderson-Fabry disease.

A 39-year-old male with classical Anderson-Fabry disease (AFD) and long-standing idiopathic splenomegaly, who had been on haemodialysis since the age of 24, was splenectomised for symptomatic pancytopaenia. Spleen enlargement was first noted at clinical presentation, at age 16, but despite thorough investigation its cause remained unclear. Anaemia, leukopaenia and thrombocytopaenia were first observed a few years thereafter, but well before the start of dialytic treatment. On gross pathological examination the spleen weighed 700 g and had a fibrocongestive appearance. Histologically, it showed expansion of the red pulp and decreased white pulp. Some histiocytes and many of the endothelial cells lining the sinusoids had vacuolated cytoplasm with argyrophilic material within, suggesting their involvement in the storage pathology of AFD. In a retrospective review of our cohort of patients with classical AFD (n = 10), complete blood counts showing anaemia, leukopaenia or thrombocytopaenia were found in five, two and four patients, respectively, including a 6-year-old boy, whose spleen was also enlarged. Data from AFD international registries show that peripheral blood cytopaenias, particularly anaemia, are prevalent among these patients. Sinusoidal endothelial involvement resulting in compromise of splenic blood flow may be the cause of congestive splenomegaly and hypersplenism in classical AFD.