Hepatitis-associated Aplastic Anaemia in Children

2020 ◽  
Vol 232 (03) ◽  
pp. 151-158
Author(s):  
Anne-Kathrin Böske ◽  
Annette Sander ◽  
Karl-Walter Sykora ◽  
Ulrich Baumann ◽  
Eva-Doreen Pfister
Keyword(s):  
Bone Marrow ◽  
Aplastic Anaemia ◽  
Bone Marrow Failure ◽  
Haematopoietic Stem Cell ◽  
Liver Transplants ◽  
Bone Marrow Hypoplasia ◽  
Development Therapy ◽  
Life Threatening ◽  
Haematological Remission

Abstract Background Children with idiopathic acute liver failure (IALF) are at a high risk of developing life-threatening bone marrow failure (BMF). The aim of the study was to describe the development, therapy and prognosis of this hepatitis-associated aplastic anaemia (HAAA) in comparison to isolated acquired aplastic anaemia. Results We retrospectively found 18 patients (9 female) of HAAA between 1984 and 2017 with an age of 1.4–16.4 years. Fifteen of them fulfilled the SAA criteria, 3 had a bone marrow hypoplasia. Eleven of these children received liver transplantation (LTx) (these were 11 of 42 (26%) children receiving LTx for IALF), 6 patients recovered without LTx. The first signs of BMF, thrombocytopaenia and leucocytopaenia, occurred before LTx in all cases. During the follow-up period 8 patients reached haematological remission, 6 received haematopoietic stem cell transplantation (HSCT). Seven children died in a median of 304 days after the first symptoms mostly because of bleedings and infections. To date, extensive investigations failed to detect a genetically, viral or immunological aetiology. No AA was diagnosed in the 41 patients receiving liver transplants during the same period for ALF of known aetiology. As a comparison group, we collected the data of patients with isolated SAA. 73% achieved a remission after Immunosuppressive therapy (IST) without HSCT, and none of them died during the follow-up period. Conclusion Blood counts should be examined early and regularly (0–22 days after onset) in patients with IALF. Aggressive treatment with LTx, IST and HSCT appears to improve the prognosis.

scholarly journals “Real World” Australian Experience of Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) in Adults with Severe Aplastic Anaemia.

2020 ◽  
Vol 4 (4) ◽  
Author(s):  
Courtney Tate ◽  
Jason P Butler ◽  
Cameron Curley ◽  
Siok-Keen Tey ◽  
Glen A Kennedy ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Stem Cell ◽  
Aplastic Anaemia ◽  
Bone Marrow Failure ◽  
Haematopoietic Stem Cell ◽  
Unrelated Donor ◽  
Cell Transplant ◽  
Severe Aplastic Anaemia ◽  
Hematopoietic Stem ◽  
Gvhd Prophylaxis

Acquired Severe Aplastic Anaemia (SAA) is a rare bone marrow failure syndrome, for which allogeneic haematopoietic stem cell transplant (HSCT) is a proven curative therapy. Despite excellent outcomes for matched sibling SAA recipients in terms of engraftment and survival, HSCT remains highly challenging in older matched-unrelated-donor (MUD) recipients, due to increased non-relapse mortality (NRM) from causes such as graft versus host disease (GVHD), organ failure and infection. We sought to evaluate our local HSCT outcomes for SAA, determine factors that predict for inferior outcomes, and benchmark local outcomes against international cohorts. To define outcomes for sibling and MUD HSCT in adults ≥17 years of age with SAA at a single Australian institution between 2002 and 2018 and compare these outcomes to internationally published cohorts. The primary outcome was 1-year overall survival (OS). Secondary outcomes included the incidences of engraftment, response, secondary graft failure, GVHD, and moderate to severe organ dysfunction. All 21 patients comprising 10 sibling and 11 MUD HSCTs, with a median age of 30 (range 17-64), received bone marrow grafts. At a median follow up of 3.5 years, 17 (81%) of 21 patients remained alive and in remission from SAA. 1-year OS, non-relapse mortality (NRM), and GVHD incidence were 85%, 15%, and 48% respectively. Two patients died prior to engraftment at Day+27 and Day+33. When comparing sibling and MUD HSCT recipients, incidences of survival, engraftment and post-HSCT complications were similar; however, MUD HSCT survivors experienced a higher incidence of chronic GVHD (67% vs. 11%; p=0.04). Age >40 years, AKI by Day+28, infection by Day+100 and deviation from intended GVHD prophylaxis predicted for worse OS. Our outcomes following HSCT for SAA are comparable to international cohorts with age >40 years, early onset infection, AKI and deviation from intended GVHD prophylaxis protocol negatively impacting OS. Further research is warranted to optimise MUD HSCT conditioning and GVHD prophylaxis protocols for SAA, particularly in older patients.

scholarly journals Nontransplant therapy for bone marrow failure

Hematology ◽  
2016 ◽  
Vol 2016 (1) ◽  
pp. 83-89 ◽  
Author(s):  
Danielle M. Townsley ◽  
Thomas Winkler
Keyword(s):  
Bone Marrow ◽  
Aplastic Anemia ◽  
Bone Marrow Failure ◽  
Clonal Evolution ◽  
Single Agent ◽  
Telomere Attrition ◽  
Long Term Follow Up ◽  
Treatment Naïve

Abstract Nontransplant therapeutic options for acquired and constitutional aplastic anemia have significantly expanded during the last 5 years. In the future, transplant may be required less frequently. That trilineage hematologic responses could be achieved with the single agent eltrombopag in refractory aplastic anemia promotes new interest in growth factors after years of failed trials using other growth factor agents. Preliminary results adding eltrombopag to immunosuppressive therapy are promising, but long-term follow-up data evaluating clonal evolution rates are required before promoting its standard use in treatment-naive disease. Danazol, which is traditionally less preferred for treating cytopenias, is capable of preventing telomere attrition associated with hematologic responses in constitutional bone marrow failure resulting from telomere disease.

scholarly journals Pancytopenia: A Clinico Hematological Study

2011 ◽  
Vol 3 (01) ◽  
pp. 015-020 ◽  
Author(s):  
Gayathri B N. ◽  
Kadam Satyanarayan Rao
Keyword(s):  
Bone Marrow ◽  
Megaloblastic Anemia ◽  
Hematological Parameters ◽  
Disease Process ◽  
Bone Marrow Aspiration ◽  
Drug Induced ◽  
Male Predominance ◽  
Bone Marrow Hypoplasia ◽  
Life Threatening ◽  
A Prospective Study

ABSTRACT Background: Pancytopenia is a relatively common hematological entity. It is a striking feature of many serious and life-threatening illnesses, ranging from simple drug-induced bone marrow hypoplasia, megaloblastic anemia to fatal bone marrow aplasias and leukemias. The severity of pancytopenia and the underlying pathology determine the management and prognosis. Thus, identification of the correct cause will help in implementing appropriate therapy. Objectives: To study the clinical presentations in pancytopenia due to various causes; and to evaluate hematological parameters, including bone marrow aspiration. Materials and Methods: It was a prospective study, and 104 pancytopenic patients were evaluated clinically, along with hematological parameters and bone marrow aspiration in Hematology Unit, Department of Pathology, JJMMC, Davanagere, during the period of September 2005 to September 2007. Results: Among 104 cases studied, age of patients ranged from 2 to 80 years with a mean age of 41 years, and male predominance. Most of the patients presented with generalized weakness and fever. The commonest physical finding was pallor, followed by splenomegaly and hepatomegaly. Dimorphic anemia was the predominant blood picture. Bone marrow aspiration was conclusive in all cases. The commonest marrow finding was hypercellularity with megaloblastic erythropoiesis. The commonest cause for pancytopenia was megaloblastic anemia (74.04%), followed by aplastic anemia (18.26%). Conclusion: The present study concludes that detailed primary hematological investigations along with bone marrow aspiration in cytopenic patients are helpful for understanding disease process and to diagnose or to rule out the causes of cytopenia. These are also helpful in planning further investigations and management.

scholarly journals Revesz syndrome revisited

2020 ◽  
Vol 15 (1) ◽  
Author(s):  
Michael Karremann ◽  
Eva Neumaier-Probst ◽  
Frank Schlichtenbrede ◽  
Fabian Beier ◽  
Tim H. Brümmendorf ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
English Literature ◽  
Bone Marrow Failure ◽  
Dyskeratosis Congenita ◽  
Kaplan Meier ◽  
Low Prevalence

Abstract Background Revesz syndrome (RS) is an extremely rare variant of dyskeratosis congenita (DKC) with only anecdotal reports in the literature. Methods To further characterize the typical features and natural course of the disease, we screened the English literature and summarized the clinical and epidemiological features of previously published RS cases. In addition, we herein describe the first recorded patient in central Europe. Results The literature review included 18 children. Clinical features are summarized, indicating a low prevalence of the classical DKC triad. All patients experienced early bone marrow failure, in most cases within the second year of life (median age 1.5 years; 95% CI 1.4–1.6). Retinopathy occurred typically between 6 and 18 months of age (median age 1.1 years; 95% CI 0.7–1.5). The incidence of seizures was low and was present in an estimated 20% of patients. The onset of seizures was exclusively during early childhood. The Kaplan–Meier estimate of survival was dismal (median survival 6.5 years; 95% CI 3.6–9.4), and none of the patients survived beyond the age of 12 years. Stem cell transplantation (SCT) was performed in eight children, and after a median of 22 months from SCT four of these patients were alive at the last follow up visit. Conclusion RS is a severe variant of DKC with early bone marrow failure and retinopathy in all patients. Survival is dismal, but stem cell transplantation may be performed successfully and might improve prognosis in the future.

Anaemia, cytopenias, and thrombosis in palliative medicine

2015 ◽  
Author(s):  
Nancy Y. Zhu ◽  
Cynthia Wu
Keyword(s):  
Bone Marrow ◽  
End Of Life ◽  
End Of Life Care ◽  
Bone Marrow Failure ◽  
Nutritional Deficiencies ◽  
Adverse Side Effects ◽  
Care Plans ◽  
Life Threatening ◽  
Expected Benefits

Many haematological issues can complicate end-of-life care, including cytopenias and venous thromboembolism (VTE). Anaemia is very common and can significantly impact quality of life; causes include haemorrhage, iron deficiency, nutritional deficiencies, and bone marrow infiltration. Neutropenia from bone marrow failure as a result of disease infiltration or from chemotherapy effects can result in life-threatening infections. Finally, VTE is commonly seen in cancer patients as well as those who require prolonged hospitalization. Symptoms can cause discomfort, mortality is increased, and treatment is associated with major bleeding. Understanding the therapeutic options and their adverse side effects is essential in the management of these complex problems. Despite the presence of effective therapies, it is also important to realize that events such as febrile neutropenia and pulmonary embolism are often seen at the end of life and intervention may not always impact prognosis. The risks of intervention should be weighed against expected benefits when developing appropriate palliative care plans.

scholarly journals Management of thrombosis in paroxysmal nocturnal hemoglobinuria: a clinician’s guide

2016 ◽  
Vol 8 (3) ◽  
pp. 119-126 ◽  
Author(s):  
Morag Griffin ◽  
Talha Munir
Keyword(s):  
Bone Marrow ◽  
Median Survival ◽  
Paroxysmal Nocturnal Hemoglobinuria ◽  
Bone Marrow Failure ◽  
Paroxysmal Nocturnal Haemoglobinuria ◽  
Orphan Disease ◽  
Life Threatening

Paroxysmal nocturnal haemoglobinuria (PNH), an ultra-orphan disease with a prevalence of 15.9 per million in Europe, is a life-threatening disorder, characterized by haemolysis, bone marrow failure and thrombosis. Patients with PNH prior to the availability of eculizumab had a median survival of between 10 and 22 years, with thrombosis accounting for 22–67% of deaths. 29–44% of patients had at least one thrombosis. This paper provides a clinician’s guide to the diagnosis, management and complications of PNH, with an emphasis on thrombosis.

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