Aplastic Anemia In Children Research Articles

Aplastic anemia in children: the current concept of differential diagnosis

Aplastic anemia in children: the current concept of differential diagnosis

Clinical Observation on Haploid Hematopoietic Stem Cells Combined with Umbilical Cord Blood Double Transplantation for Aplastic Anaemia in Children

To explore the efficacy and safety of haploidentical hematopoietic stem cell transplantation combined with umbilical cord blood infusion for the treatment of aplastic anaemia in children. Nine cases of children with aplastic anaemia treated with umbilical cord blood combined with haploidentical hematopoietic stem cell transplantation at the People's Hospital of Henan University of Chinese Medicine from January 1, 2021 to September 15, 2023 with a median age of 11(2-13) years and a median follow up of 18(7.5-21) months were included, and the clinical data were retrospectively analyzed. Hematopoiesis reconstitution, the incidence of graft-versus-host disease(GVHD), infections and survival of the patients were analyzed. All 9 children were successfully implanted. The median time to neutrophil and platelet implantation was 11.11±1.27 d and 12.44±3.36 d, respectively. One case developed acute gastrointestinal GVHD of degree I, which was improved after treatment, and the patient developed superficial gastritis and chronic gastrointestinal GVHD at a later stage, which is currently under clinical follow-up. Acute GVHD of II-IV degree was 0%. Hemorrhagic cystitis in 3 cases, CMV infection in 5 cases and bacterial and fungal infections in 5 cases improved with symptomatic treatment.All 9 children demonstrated complete donor chimerism within 1 month after transplantation, at two years of follow-up, all nine children survived without recurrence or development of grade II-IV GVHD, and there were no children with transplant-related deaths. Haploidentical hematopoietic stem cell transplantation combined with umbilical cord blood transfusion for aplastic anaemia in children has a low incidence and mild degree of GVHD, with significant efficacy, and can be used as a therapeutic option for children without an HLA full donor chimeric match.

Treatment of relapsed/refractory severe aplastic anemia in children: Evidence-based recommendations.

Severe aplastic anemia (SAA) is a rare potentially fatal hematologic disorder. Although overall outcomes with treatment are excellent, there are variations in management approach, including differences in treatment between adult and pediatric patients. Certain aspects of treatment are under active investigation in clinical trials. Because of the rarity of the disease, some pediatric hematologists may have relatively limited experience with the complex management of SAA. The following recommendations reflect an up-to-date evidence-based approach to the treatment of children with relapsed or refractory SAA.

Treatment of newly diagnosed severe aplastic anemia in children: Evidence-based recommendations.

Severe aplastic anemia (SAA) is a rare potentially fatal hematologic disorder. Although overall outcomes with treatment are excellent, there are variations in management approach, including differences in treatment between adult and pediatric patients. Certain aspects of treatment are under active investigation in clinical trials. Because of the rarity of the disease, some pediatric hematologists may have relatively limited experience with the complex management of SAA. The following recommendations reflect an up-to-date evidence-based approach to the treatment of children with newly diagnosed SAA.

Efficacy, Safety, and Population Pharmacokinetics of Eltrombopag in Children with Different Severities of Aplastic Anemia.

Eltrombopag was approved as a first-line treatment for patients older than 2 years old with severe aplastic anemia (SAA). However, data on eltrombopag in children with different types of aplastic anemia (AA), especially non-severe AA (NSAA), are limited. We performed a prospective, single-arm, and observational study to investigate eltrombopag's efficacy, safety, and pharmacokinetics in children with NSAA, SAA, and very severe AA (VSAA). The efficacy and safety were assessed every 3 months. The population pharmacokinetic (PPK) model was used to depict the pharmacokinetic profile of eltrombopag. Twenty-three AA children with an average age of 7.9 (range of 3.0-14.0) years were enrolled. The response (complete and partial response) rate was 12.5%, 50.0%, and 100.0% after 3, 6, and 12 months in patients with NSAA. For patients with SAA and VSAA, these response rates were 46.7%, 61.5%, and 87.5%. Hepatotoxicity occurred in one patient. Fifty-three blood samples were used to build the PPK model. Body weight was the only covariate for apparent clearance (CL/F) and volume of distribution. The allele-T carrier of adenosine triphosphate-binding cassette transporter G2 was found to increase eltrombopag's clearance. However, when normalized by weight, the clearance between the wild-type and variant showed no statistical difference. In patients with response, children with NSAA exhibited lower area under the curve from time zero to infinity, higher CL/F, and higher weight-adjusted CL/F than those with SAA or VSAA. However, the differences were not statistically significant. The results may support further individualized treatment of eltrombopag in children with AA.

Clinical Signs and Treatment of New-Onset Bone Marrow Failure Associated SARS-CoV-2 Infection in Children: A Single Institution Prospective Cohort Study.

Viral infections can cause direct and indirect damage to hematopoietic stem cells. The objectives of this study were to identify the frequency and severity of aplastic anemia in children infected with severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) as well as recognize the response to treatment. 13 children with newly diagnosed severe aplastic anemia were enrolled in this prospective clinical trial. Blood samples were obtained from all patients to detect SARS-CoV-2 antibodies, and nasopharyngeal swabs were collected for reverse-transcription Polymerase Chain Reaction to detect SARS-CoV-2 viruses. According to the laboratory results, patients were classified as having SARS-CoV-2 positive antibodies and SARS-CoV-2 negative antibodies. Both groups received combined cyclosporine (CsA) + Eltrombopag (E-PAG). The hematological response, either complete response (CR) or partial response (PR), no response (NR), and overall response (OR) rates of combined E-PAG + CsA treatment after 6 months were evaluated. Four children were recognized to have aplastic anemia and SARS-CoV-2 positive antibodies. Two patients fulfilled the hematological criteria for CR and no longer required transfusion of packed red blood cells (PRBCs) or platelets, and one had PR and was still PRBC transfusion-dependent but no longer required platelet transfusion. The remaining patient showed NR, and he had died before reaching the top of the HSCT waiting list. Moreover, six patients in the SARS-CoV-2 negative antibodies group had CR, while three patients had PR. The difference in ANC, Hg, and platelet counts between both groups was not significant. The SARS-CoV-2 virus is added to several viral infections known to be implicated in the pathogenesis of aplastic anemia. Studies are needed to establish a definitive association and determine whether the response of bone marrow failure to standard therapy differs from that of idiopathic cases.

Clinical characteristics of hepatitis-associated aplastic anemia in children.

To understand the current situation of hepatitis-related aplastic anemia (HAAA) in children, we analyzed the patients with HAAA admitted to our hospital in the past 5 years to understand the disease characteristics and prognosis.The clinical data of patients with HAAA admitted to our hospital from February 2017 to May 2022 were retrospectively analyzed.A total of 81 patients with HAAA, 56 males and 25 females. The median onset age was 5.9 years. The median time from hepatitis to occurrence of hemocytopenia was 30 days, and the median follow-up time was 2.77 years. There were 23 cases (28.5%) of severe aplastic anemia (SAA), 50 cases of very severe aplastic anemia (VSAA), and 8 cases of non-severe aplastic anemia (NSAA). At the beginning of the disease, cytotoxic T lymphocyte (CTL) was higher than normal in 60% of patients, and the median CD4/CD8 ratio was 0.2. As of follow-up, 72 children survived, 4 were lost, and 5 died. Thirty-four cases were treated with immunosuppressive therapy (IST), with a median follow-up time of 0.97 years. The total reaction rate was 73.5% (25/34), the complete reaction rate was 67.6% (23/34), and the nonreaction rate was 26.5% (9/34). Multivariate analysis suggested that co-infection was an independent risk factor affecting the efficacy of IST at 6 months, with an OR value of 16.76, 95% CI (1.23, 227.95), P=0.034. No independent influencing factors were found at the end of follow-up.The proportion of CTL cells in peripheral blood of children with HAAA is relatively increased, and IST is effective in 73.5% of children. Co-infection may prolongs the time to response to IST.

Efficacy, Safety, and Population Pharmacokinetics of Eltrombopag in Children with Different Severities of Aplastic Anemia

OBJECTIVES: Eltrombopag (EPAG) was initially approved by the U.S. Food and Drug Administration (FDA) as first-line treatment, combined with standard immunosuppressive therapy, for patients older than two years old with severe aplastic anemia (SAA). However, the use of EPAG in children with different types of aplastic anemia (AA), especially non-severe AA (NSAA) has been limited. Therefore, we decided to investigate the efficacy, safety, and pharmacokinetics of EPAG in children with NSAA, SAA, and very severe AA (VSAA). METHODS: A prospective, open-label, observational study was conducted at the Institute of Hematology and Blood Diseases Hospital, Tianjin, China. The efficacy including complete response (CR), partial response (PR), and no response (NR), as well as safety was assessed every three months after the start of treatment. A non-linear mixed-effects population pharmacokinetic (PPK) model was used to depict the pharmacokinetic profile of EPAG. We explored the exposure-response relationship of EPAG in patients with different types of AA. This study was registered at ClinicalTrials.gov (NCT03844360). RESULTS: A total of 23 AA children with an average age of 7.9 (range of 3.0 - 14.0) years were enrolled in this study for efficacy, safety, and PPK analysis. Fifteen patients had a treatment response (5 had CR and 10 had PR). The response rate was 12.5% after 3 months, 50.0% after 6 months, and 100% after 12 months of treatment for patients with NSAA. For patients with SAA and VSAA, these response rates were 46.7%, 61.5%, and 87.5%. Hepatotoxicity occurred in one patient. Fifty-three blood samples were collected and analyzed to build the PPK model using allometric scaling with fixed exponents. Body weight significantly affected EPAG's apparent clearance and volume of distribution. The mean (range) of the area under the concentration-time curve from time zero to infinity (AUC ∞) was 316 (131 - 809) μg×h /mL. Twenty patients with genotypes were included in the genotype analysis. The apparent clearance of EPAG was significantly higher in allele-T carriers of adenosine triphosphate-binding cassette G2 (ABCG2) (rs2231142, G>T) ( p=0.02). In patients with clinical response, children with NSAA exhibited lower AUC ∞, higher apparent clearance, and higher weight-adjusted apparent clearance than those with SAA or VSAA, although the differences were not significant. CONCLUSIONS: This study enriched the efficacy and safety of EPAG in children with different types of AA. Body weight and ABCG2 genotype significantly influence EPAG clearance. The results may support further individualized treatment of EPAG in children with AA.

Frequency of Aplastic Anemia in Children (1-15 years) with New-Onset Pancytopenia

Background: Pancytopenia is a medical disorder in which the usual quantities of red blood cells, white blood cells, and platelets are depleted. This condition can arise from various factors, including diminished production of hematopoietic cells, ineffective hematopoiesis, or the destruction of these cells in the peripheral blood. Objective: To determine the frequency of aplastic anemia in children (1-15 years) with new-onset pancytopenia presenting at Children Hospital Lahore. Study Design: Cross sectional study. Settings: Department of Pediatric Medicine, Children Hospital, Lahore Pakistan. Duration: From August 2022 January 2023. Methods: This research included 250 children, covering both genders, with ages ranging from 1 month to 15 years old. These children had all been diagnosed with pancytopenia within the past month. The primary focus of the study was to determine the prevalence of aplastic anemia within this group of children. This prevalence was then examined in relation to their age, gender, and socioeconomic backgrounds. Results: The patient group had a mean age of 6.74 years, primarily consisting of children aged 1-10 years (86.4%). Gender distribution was fairly balanced, with 55.2% males and 44.8% females. Most patients belonged to the middle class (48.8%), followed by the lower class (35.2%) and high class (16.0%). Pallor was the most common, with 86.36% of patients experiencing it. Fever and bruises/purpura were also frequent, affecting 64.55% and 59.55% of patients, respectively. Aplastic anemia was observed in 33.60% of the children, while 66.4% did not have this condition. Conclusion: In children who had recently developed pancytopenia, the incidence of aplastic anemia was determined to be 33.60%. Interestingly, no noteworthy disparities in the occurrence of aplastic anemia were observed with regard to age, gender, or socioeconomic status.

Recent advances in the diagnosis and treatment of pediatric acquired aplastic anemia.

Acquired aplastic anemia (AA) in children is a rare bone marrow failure that requires several special considerations for its diagnosis and treatment compared with that in adults. The most common issue is the differential diagnosis with refractory cytopenia of childhood and inherited bone marrow failure syndromes, which is crucial for making decisions on the appropriate treatment for pediatric AA. In addition to detailed morphological evaluation, a comprehensive diagnostic work-up that includes genetic analysis using next-generation sequencing will play an increasingly important role in identifying the underlying etiology of pediatric AA. When discussing treatment strategies for children with acquired AA, the long-term sequelae and level of hematopoietic recovery that affect daily or school life should also be considered, although the overall survival rate has reached 90% after immunosuppressive therapy or hematopoietic cell transplantation (HCT). Recent advances in HCT for pediatric patients with acquired AA have been remarkable, with the successful use of upfront bone marrow transplantation from a matched unrelated donor, unrelated cord blood transplantation or haploidentical HCT as salvage treatment, and fludarabine/melphalan-based conditioning regimens. This review discusses current clinical practices in the diagnosis and treatment of acquired AA in children based on the latest data.

Real world scenario of aplastic anemia in children – Single centre experience

Real world scenario of aplastic anemia in children – Single centre experience

Impact of Conditioning Regimen and Graft-versus-Host Disease Prophylaxis on The Outcome of Haploidentical Peripheral Blood Stem Cell Transplantation for High-Risk Severe Aplastic Anemia in Children and Young Adults: A Report from the Pediatric Severe Aplastic Anemia Consortium of India

Allogenic hematopoietic cell transplantation (HCT) is the best curative approach for patients with severe aplastic anemia (SAA). The outcomes of HCT from haploidentical family donors (HFDs) have improved, making it a feasible option for patients lacking an HLA-identical donor. However, data on HFD-HCT for younger patients with SAA is sparse. In this multicenter retrospective study, we evaluated the outcomes of 79 patients undergoing HFD-HCT for SAA. All the patients were heavily pretransfused, the median time to HCT was >12 months, and 67% had failed previous therapies. Conditioning was based on fludarabine (Flu)-cyclophosphamide (Cy)-antithymocyte globulin (ATG)/total body irradiation (TBI) with or without thiotepa/melphalan (TT/Mel). Post-transplantation Cy (PTCy) and calcineurin inhibitors (CNIs)/sirolimus were used as graft-versus-host disease (GVHD) prophylaxis with or without abatacept. The rate of primary graft failure (PGF) was 16.43% overall, lower in patients conditioned with TT/Mel. The incidences of acute and chronic GVHD were 26.4% and 18.9%, respectively. At a median follow-up of 48 months, the overall survival (OS) and event-free survival (EFS) were 61.6% and 58.1%, respectively. Both OS and EFS were better in the TT/Mel recipients and with abatacept as GVHD prophylaxis. On multivariate analysis, the use of abatacept was found to favorably impact the outcome variables, including GVHD and EFS. Our study suggests that PTCy-based HFD-HCT is a reasonable option for young patients with high-risk SAA, in whom optimization of conditioning and GVHD prophylaxis might further improve outcomes.

The impact of pretransplant serum ferritin on haploidentical hematopoietic stem cell transplant for acquired severe aplastic anemia in children and adolescents.

Haploidentical hematopoietic stem cell transplant (haplo-HSCT) provides an important alternative for children and adolescents with acquired severe aplastic anemia (SAA) lacking matched donors. To test whether pretransplant serum ferritin (SF) represents a candidate predictor for survival and a potential biomarker for graft-versus-host disease (GvHD) in pediatric haplo-HSCT, we retrospectively evaluated 147 eligible patients with SAA who underwent haplo-HSCT. The patients were divided into the low-SF group(<1000 ng/mL) and the high-SF group (≥ 1000 ng/mL). We found that SF ≥1000 ng/mL independently increased the risk of grade II-IV aGvHD (HR=2.596; 95% CI, 1.304-5.167, P=0.007) and grade III-IV aGvHD (HR=3.350; 95% CI, 1.162-9.658, P=0.025). Similar probabilities of transplant-related mortality at 100 days were observed in the two groups (6.19 ± 2.45%vs 8.00 ± 3.84%, P=0.168). The two-year overall survival (85.29 ± 3.89%vs 92.00% ± 3.84%, P=0.746) and failure-free survival (83.23% ± 4.08%vs 83.37% ± 6.27%, P=0.915) were comparable. GvHD-/failure-free survival were 60.06 ± 5.10% and 75.56 ± 6.87%, respectively (P=0.056). In conclusion, elevated pretransplant SF level is associated with higher incidences of grade II-IV aGvHD and grade III-IV aGvHD. However, it is not associated with worse survival after haplo-HSCT for children and adolescent patients with SAA.

P802: PATTERNS OF T-CELL AUTOREACTIVITY DIFFER BETWEEN PEDIATRIC APLASTIC ANEMIA AND HEPATITIS-ASSOCIATED BONE MARROW FAILURE

Background: Both idiopathic aplastic anemia (AA) and hepatitis-associated bone marrow failure (HABMF) are considered to be autoreactive T cell mediated diseases. This is based on indirect evidence, most often the success of immunosuppression or oligoclonality with T cell repertoire restriction. Aims: Our questions were: Does the composition of T cells and T-cell receptor beta (TRB) repertoire differ between HABMF, BMF without preceding hepatitis (nonHABMF) and normal bone marrow (BM)? Are there any shared clonotypes suggesting common antigenic stimulus? How does immunosuppressive therapy affect T-cells and does this correlate with outcome? Methods: In total 22 pediatric patients diagnosed in 2004-2021 with HABMF were included in the study. Sixteen HABMF patients were treated with immunosuppressive therapy (IST) as a frontline treatment, of which 8 patients did not respond. As a control group, we included 10 nonHABMF patients with AA (n=6) and refractory cytopenia of childhood (n=4), all treated with IST with poor response in 6 patients, as well as 10 samples of healthy donor BM grafts and 8 newborn peripheral blood (NBPB) samples. We performed flow cytometry immunophenotyping at diagnosis (D0) and on day 120 (D120) after initiation of IST in HABMF and nonHABMF patients. We performed sequencing of the TRB gene rearrangements according to the EuroClonality-NGS working group protocols using DNA isolated from these samples and normalized DNA input for TRB library preparation to the equivalent of 20 000 CD3+ cells per sample based on flow cytometry data, if possible. Whole exome sequencing (WES) was performed from diagnostic samples of HABMF patients. Results: Patients with HABMF had a significantly lower proportion of CD3+ T cells in their BM compared to the nonHABMF with the predominance of CD8+ T cells and their activation by expression of HLA-DR observed with flow cytometry. The analysis of TRB repertoire was performed in 22 D0 and 10 corresponding D120 samples of HABMF patients that had undergone IST and we compared the diversity and clonotype composition with the nonHABMF patients as well as the BM graft samples and NBPB. Expansion of individual TRB clonotypes (>5% of all reads) at D0 was observed more frequently in HABMF patients (9 out of 22) compared to the nonHABMF patients (1 out of 10). None of these expanded clonotypes were shared among more patients based on their nucleotide or amino acid sequence. There was no correlation of expanded T cell clone dynamics and response to IST. The diversity of TRB repertoire was reduced in the nonHABMF group after IST, however we did not observe any correlation between the clinical response to the IST and initial TRB diversity in either group of patients. WES did not reveal any pathogenic variants in genes associated with immune dysregulation. Summary/Conclusion: Both the proportion of T cells and T-cell composition in the BM differed between HABMF and nonHABMF patients at diagnosis. Although we observed an expected reduction in the CD3+ cells after IST both in the HABMF and nonHABMF group, there was no significant difference in the TRB repertoire diversity in the HABMF patients between D0 and D120. We did not observe any correlation between the repertoire diversity and clonotype evolution and the clinical response to IST. Our data further support the hypothesis that T-cells play a significant role in both AA and HABMF, but suggest that the pathogenetic mechanism of both entities is different. Supported by NV20-03-00284, NV19-05-00332, UNCE/MED/015, NU20J-07-00028 and GAUK 534120.

Childhood aplastic anaemia with paroxysmal nocturnal haemoglobinuria clones: A retrospective single-centre study in South Africa.

BackgroundParoxysmal nocturnal haemoglobinuria (PNH) clones in children are rare but commonly associated with aplastic anaemia (AA) and myelodysplasia.ObjectiveThis study aimed to determine the prevalence of PNH clones in paediatric patients with idiopathic AA, identify differences in clinical and laboratory features and outcomes, and determine the impact of clone size on clinical presentation.MethodsPatients with confirmed idiopathic AA who were tested for PNH between September 2013 and January 2018 at the Inkosi Albert Luthuli Central Hospital, Durban, KwaZulu-Natal, South Africa, were included. PNH clones were detected in neutrophils and monocytes by flow cytometry using fluorescent aerolysin, CD24, CD66b and CD14.ResultsTwenty-nine children with AA were identified and 11 were excluded. Ten patients (10/18, 55.6%) had PNH clones ranging from 0.11% to 24%. Compared to the PNH-negative group, these children were older (median: 10 years vs 4 years, p = 0.02) and had significantly lower total white cell counts (median 1.7 × 109/L vs 3.2 × 109/L; p = 0.04). There was no difference in median absolute neutrophil count or haemoglobin concentration. Four patients in each group received immunosuppressive therapy (IST). At six months, all four patients with PNH clones had responded, compared to one in the PNH-negative group.ConclusionMore than half of children with AA had a PNH clone. The size of the clone did not impact clinical severity; however, IST use may positively impact prognosis. We recommend early initiation of IST in patients with AA to avoid delays associated with human leukocyte antigen typing.

Correlation between Serum G-CSF Level and Immune Function in Children with Aplastic Anemia

To observe the occurrence of immune dysfunction in children with aplastic anemia (AA) and the factors that may lead to immune dysfunction, analyze the relationship between the expression of granulocyte colony stimulating factor (G-CSF) and immune dysfunction. A total of 34 children with AA treated in our hospital from December 2016 to September 2018 were selected. All the children received immunosuppressive therapy (IST) for 6 months. According to whether the children had immune dysfunction after 6 months of treatment, they were divided into occurrence group and non occurrence group. General information and laboratory indices were compared between the two groups, and serum G-CSF level was tested, the relationship between serum G-CSF level and immune dysfunction in AA children after treatment with IST was observed and analyzed. After treatment with IST for 6 months, 12 cases developed immune dysfunction (35.29%). Serum interferon (IFN)-γ level of the occurrence group was higher but G-CSF level was lower than those of the non occurrence group (P<0.05), while the difference of other baseline data was not statistically significant (P>0.05). Multiple regression analysis showed that overexpression of serum IFN-γ and low expression of G-CSF were both the influencing factors of immune dysfunction in AA children after IST treatment (OR>1, P<0.05). ROC curve was drawn, and the result showed that the area under the curve (AUC) of serum G-CSF level predicted the risk of immune dysfunction after IST was 0.843>0.80, when the index cut-off value was set at 6.614 pg/ml, the predictive value was ideal. AA children have a higher risk of immune dysfunction after IST, which may be related to the low expression of serum G-CSF. The detection of serum G-CSF expression can be considered to predict the risk of immune dysfunction in AA children after IST, so as to guide early clinical intervention.

A New Immunosuppressive Therapy for Very Severe Aplastic Anemia in Children with Autoantibodies.

At present, a number of very severe aplastic anemia (VSAA) patients cannot receive hematopoietic stem cell transplantation (HSCT) or standard immunosuppressive therapy (IST) due to the high cost of therapy, shortage of sibling donors, and lack of resources to support the HSCT. In addition, some VSAA patients with autoantibodies have no life-threatening infections or bleeding at the time of initial diagnosis. Considering the disease condition, economics and other factors, the present study designed a new and relatively mild treatment strategy: cyclosporine A plus pulsed high-dose prednisone (CsA+HDP). The present study retrospectively analyzed 11 VSAA patients, who were treated with CsA+HDP in our hospital from August 2017 to August 2019. The median follow-up time for these patients was 24.9 months. The overall response rate was 54.5% (6/11) at six months after the initiation of IST and 81.8% (9/11) at deadline. Five patients achieved complete remission and four patients met the criteria for partial response at the last follow-up. The median time to response for responders was 110 days. Three patients underwent HSCT due to the poor effect of CsA+HDP or to find a suitable transplant donor. Recurrence and clonal evolution were not found in any of these patients. The estimated 3-year overall survival rate and 3-year failure-free survival rate were 100.0% and 72.7%, respectively. In addition, the results revealed that the cyclosporine-prednisone-associated toxicity was mild and well-tolerated by most patients. The novel CsA+HDP regimen has good therapeutic effect and safety for VSAA patients with autoantibodies, who have no serious life-threatening infections or bleeding at the time of initial diagnosis.

553 - Thiotepa Based Reduced Toxicity Conditioning for Heavily Transfused Acquired Severe Aplastic Anemia in Children and Young Adults: Exceptional Outcomes of a Pilot Study

553 - Thiotepa Based Reduced Toxicity Conditioning for Heavily Transfused Acquired Severe Aplastic Anemia in Children and Young Adults: Exceptional Outcomes of a Pilot Study

130 - Ptcy-Based Haploidentical Hematopoietic Cell Transplant for Acquired Severe Aplastic Anemia in Children and Young Adults: A Report from Pediatric Severe Aplastic Anemia Consortium of India

130 - Ptcy-Based Haploidentical Hematopoietic Cell Transplant for Acquired Severe Aplastic Anemia in Children and Young Adults: A Report from Pediatric Severe Aplastic Anemia Consortium of India

Clinical-epidemiological aspects of acquired aplastic anemia in children in the Kyrgyz Republic

Clinical-epidemiological aspects of acquired aplastic anemia in children in the Kyrgyz Republic