Clinical profile of thrombocytopenia in tropical infectious diseases

Background: In an infant or child thrombocytopenia can occur due to large spectrum of illness ranging from tropical infection to malignancy or bone marrow failure. Management is decided by the severity of thrombocytopenia, associated risk factors and underlying illness. Children with thrombocytopenia may be asymptomatic – detected by complete blood count for some other clinical issue or symptomatic-presenting with mucosal and/or cutaneous bleeding and rarely central nervous system bleed. Aim of this research is to study the distribution of patients with thrombocytopenia, their grading according to platelet counts and etiology with special focus to infective causes, other complications in these infections and recovery from thrombocytopenia.Methods: This is an observational analytical retrospective study. 100 randomly selected pediatric patients (6 months to 12 years) admitted in pediatric ward with documented thrombocytopenia (platelet count <150,000/ul) on admission or at any point of time during hospitalisation are enrolled and analyzed.Results: 91% patients have thrombocytopenia associated with infective causes, of which 44% have dengue. 7 patients in study have bleeding manifestations and 3 required platelet transfusion. 50% patients with dengue with thrombocytopenia have leucopenia and 2% have pancytopenia. 57.1% patients with enteric fever with thrombocytopenia show elevated alanine aminotransferase (ALT) levels. Mean platelet recovery time is 2 to 4 days for various infections.Conclusions: Majority of patients do not have bleeding manifestations, and they are mainly seen with severe thrombocytopenia associated with infections. Requirement of platelet transfusion is not common and is seen only in patients with severe thrombocytopenia with significant bleeding manifestation.

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Adipose-Derived Mesenchymal Stem Cells with Potential to Ameliorate Platelet Recovery and Enhance Megakaryopoiesis in a Mouse Model of Radiation-Induced Thrombocytopenia Via the PI3k/Akt Pathway

Blood ◽

10.1182/blood.v124.21.2931.2931 ◽

2014 ◽

Vol 124 (21) ◽

pp. 2931-2931

Author(s):

Xiao-Hui Zhang ◽

Jia-Min Zhang ◽

Fei-Er Feng ◽

Qian-Ming Wang ◽

Xiao-Lu Zhu ◽

...

Keyword(s):

Bone Marrow ◽

Platelet Transfusion ◽

Treated Group ◽

Saline Group ◽

Akt Pathway ◽

Severe Thrombocytopenia ◽

Platelet Recovery ◽

Phosphorylated Akt ◽

Radiation Induced

Abstract *XHZ and JMZ contribute equally to this work # Co-correspondence to: Xiao-hui Zhang and Xiao-Jun Huang E-mail: [email protected] Introduction: Substantial damage to the bone marrow can be caused by exposure to radiation, which can then develop into pancytopenia, especially severe thrombocytopenia. Severe thrombocytopenia can be a major, life-threatening event in untreated individuals. Recent studies have highlighted the role that radiation-induced thrombocytopenia plays in radiation mortality. However, studies focused on mitigating radiation-induced thrombocytopenia have rarely been reported. Among all alternative therapies available to patients experiencing radiation-induced thrombocytopenia, platelet transfusion is most effective. As a supportive therapy, platelet transfusion cannot substantially reconstitute the damaged hematopoietic properties of the bone marrow. Adipose-derived mesenchymal stem cells (ADSCs) are capable of migrating to injured tissue sites for damage repair. Moreover, it was demonstrated that ADSCs could support hematopoiesis both in vitro and in vivo. However, the therapeutic effects of ADSCs in radiation-induced thrombocytopenia as well as the underlying mechanism remain unknown. In this study, we hypothesized that administration of ADSCs may mitigate thrombocytopenia after radiation exposure. We investigated the in vivo impact of ADSCs on megakaryopoiesis and platelet recovery, and whether this amelioration effect was mediated through the activation of PI3K/Akt pathway in irradiated mice. Methods: The mouse model of radiation-induced thrombocytopenia was established by first irradiating mice with a 4Gy dose. Then, 15 mice were immediately injected with suspended ADSCs (1×106cells in 0.3 ml) and another 15 mice with equivalent saline. Ten unirradiated mice served as a control group. Platelet counts and white blood cell (WBC) counts in the peripheral blood were detected every week. Total colony formation units (CFU), megakaryocyte colony formation units (MK-CFU) and CD41+ cells in the bone marrow were assessed 21 days after irradiation. Bone marrow cellularity was determined by hematoxylin and eosin (HE) staining, and apoptosis was detected by terminal-deoxynucleotidyl transferase-mediated nick end-labeling (TUNEL) assay. Western blots were performed with anti-phosphorylated Akt, anti-Bcl-2 and anti-bax antibodies. Results: ADSCs obtained from the inguinal adipose exhibited a fibroblast-like morphology. Cultured cells were positive for CD29 (99.79%) and CD90 (97.82%), but not CD34 (3.08%), CD45 (1.04%) and CD31 (3.08%). Radiation markedly reduced peripheral blood counts, with a nadir on day 7. Recovery of both platelets (546.33±62.99 vs 375.48±50.33×109/L, P<0.05) and WBCs (4.23±0.51 vs 2.46±3.10×109/L, P<0.05) were better in the ADSCs-treated group compared with the saline group, respectively, 21 days after irradiation. A significant increase in total CFU (34.55±4.21 vs 12.86±2.15, P<0.05) and MK-CFU (6.28±0.74 vs 2.54±0.39, P<0.05) after irradiation were observed in the ADSCs group compared with the saline group, respectively. Further, the proportion of CD41+ cells in the ADSCs group was significantly higher than that in the saline group (4.2%±0.54% vs 1.21%±0.11%, P<0.05). A radioprotective effect was shown in the ADSCs-treated group, especially in the megakaryocytic lineage, by HE staining. In the ADSCs group, the number of apoptotic cells was significantly lower than that in the saline group (3.52±0.42 vs 13.48±2.15 per field, P<0.05). Administration of ADSCs up-regulated protein expression of phosphorylated Akt and Bcl-2, whereas the expression of Bax, a protein related to apoptosis, was significantly lower in the ADSCs group. Conclusion:For the first time, we demonstrated that ADSCs were capable of promoting platelet recovery and improving megakaryopoiesis in irradiated mice. This protective function of ADSCs is likely to be mediated via the PI3K/Akt pathway, which would thus provide a new therapeutic alternative for mitigating radiation-induced thrombocytopenia. Disclosures No relevant conflicts of interest to declare.

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Complement Inhibition Using Eculizumab Overcomes Platelet Transfusion Refractoriness in Allo-Immunized Patients Receiving HLA Mismatched Platelets

Blood ◽

10.1182/blood.v128.22.3840.3840 ◽

2016 ◽

Vol 128 (22) ◽

pp. 3840-3840 ◽

Cited By ~ 1

Author(s):

Phuong T. Vo ◽

Enkhtsetseg Purev ◽

Kamille A West ◽

Michael Hochman ◽

Emily C. McDuffee ◽

...

Keyword(s):

Platelet Transfusion ◽

Median Number ◽

Response To Therapy ◽

Bleeding Complications ◽

Severe Thrombocytopenia ◽

Cell Transplant ◽

Platelet Recovery ◽

Complement Inhibition ◽

Hla Antibody ◽

Platelet Transfusions

Abstract Background: 30-50% of heavily platelet transfused patients will ultimately develop HLA allo-immunization. Although HLA-matched platelets can be lifesaving for these patients, a substantial proportion of patients receiving partially HLA-matched platelet products develop platelet transfusion refractoriness, putting them at risk for serious bleeding complications. Recent data suggest compliment activation leading to the destruction of platelets bound by HLA allo-antibodies may play a pathophysiologic role in platelet refractoriness. Eculizumab is a monoclonal antibody that binds and inhibits C5 compliment, blocking both the classic and alternative pathways of compliment. Here we investigated whether eculizumab treatment could be used to overcome platelet transfusion refractoriness in HLA allo-immunized patients. Methods: We conducted a one armed; phase II pilot trial (NCT02298933) in which HLA allo-immunized patients with platelet transfusion refractoriness received a single infusion of eculizumab at a dose of 1200mg. Eligible patients included adults age 18-75 with severe thrombocytopenia who had detectable HLA class I antibodies associated with platelet transfusion refractoriness (defined as a post-transfusion corrected count increment (CCI) of <7500/ul and <5000/ul at 10-60 mins and 18-24 hrs respectively that occurred following at least 2 consecutive platelet transfusions). Patients were defined to respond to therapy if one of the first 2 platelet transfusions following eculizumab infusion resulted in a CCI>7500/uL at 10-60 mins and CCI>5000/uL at 18-24 hrs post transfusion. Subjects were taken off study 14 days following eculizumab treatment. Responding patients who developed recurrent platelet refractoriness after planned study withdrawal were eligible to re-enroll on study. All patients were required meningococcal vaccination before eculizumab infusion +/- antibiotic prophylaxis to cover N. meningitidis. Results: As of 8/2016, 8 Eculizumab infusions were administered to 7 HLA allo-immunized subjects (median age 40 yrs, range 24-71) with severe thrombocytopenia associated with platelet transfusion refractoriness. Patients had an underlying diagnosis of SAA (n=3), refractory/relapsed AML (n=2; 1 patient had just finished conditioning for a haplo-identical transplant), relapsed ALL (n=1; post MUD transplant) and high risk MDS (n=1). Compliment, total (CH50) was the most reliable and consistent measure for complement inhibition. After Eculizumab infusion, CH50 decreased to <20U/ml in all treated patients. 3 of 8 (43%) patients had a response to therapy with platelet refractoriness resolving following 4/8 (50%) eculizumab administrations (Figures 1-3). Subject 1 received an additional 2nd treatment with eculizumab 2 months after her 1st response and again had resolution of platelet refractoriness (Figure 1A&1B). In 3 out of 4 cases where platelet refractoriness was overcome, the administered platelet product given immediately after eculizumab treatment expressed an HLA allele for which an HLA antibody had been detected in the patient's serum. Resolution of platelet refractoriness resulted in a clinically meaningful reduction in the requirement for platelet transfusions: the median number of platelet transfusion given 2 weeks before and 2 weeks after the eculizumab infusion in responding patients was 8.5 (range 3-10) and 4.5 (range 3-5) transfusions respectively. For the non-responders, the median number of platelet transfusions given 2 weeks before and 2 weeks after the eculizumab infusion was 8 (range 7-10 ) and 9.5 (range 6-15) transfusions respectively. Conclusions: The preliminary data from this ongoing trial suggest eculizumab may have efficacy in overcoming HLA antibody-associated platelet transfusion refractoriness. Remarkably, we observed eculizumab overcame platelet refractoriness in a subset of HLA allo-immunized patients receiving HLA mismatched platelet products that expressed an HLA allele for which an HLA antibody was detectable in the patient's serum. These data raise the possibility that eculizumab administration could be of therapeutic benefit in patients with platelet refractoriness as a consequence of newly discovered HLA antibodies, buying time for transfusion medicine departments to acquire HLA matched platelets or until platelet recovery occurs following chemotherapy or stem cell transplant. Disclosures No relevant conflicts of interest to declare.

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Accuracy of platelet counting haematology analysers in severe thrombocytopenia and potential impact on platelet transfusion

British Journal of Haematology ◽

10.1111/j.1365-2141.2004.05352.x ◽

2005 ◽

Vol 128 (4) ◽

pp. 520-525 ◽

Cited By ~ 91

Author(s):

H. C. Segal ◽

C. Briggs ◽

S. Kunka ◽

A. Casbard ◽

P. Harrison ◽

...

Keyword(s):

Platelet Transfusion ◽

Severe Thrombocytopenia ◽

Potential Impact

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The study of clinical profile and vitamin B 12 in dengue fever patients

Asian Journal of Medical Sciences ◽

10.3126/ajms.v12i8.36311 ◽

2021 ◽

Vol 12 (7) ◽

pp. 47-51

Author(s):

Mamatha Tittamegalapalya Ramalingaiah ◽

Jeetendra Kumar Jogihalli Mood ◽

Satyanarayana Narayanashetty ◽

Rashmi Madappa Bhuvaneshappa

Keyword(s):

Vitamin B12 ◽

Hospital Stay ◽

Dengue Fever ◽

Platelet Transfusion ◽

Clinical Profile ◽

Study Patient ◽

Vitamin B ◽

Severe Thrombocytopenia ◽

Duration Of Hospital Stay ◽

Vitamin B 12

Background: Dengue is an acute infectious febrile illness characterised by thrombocytopenia and platelet dysfunction leading to bleeding manifestations. Vitamin B12 is required for platelet production in the bone marrow. So, deficiency of vitamin B12 in dengue patients can have severe thrombocytopenia. Aims and Objective: Study was aimed to know the clinical profile of dengue fever patients and to correlate serum vitamin B12 level with severity of thrombocytopenia, platelet transfusion and duration of hospital stay. Materials and Methods: This observational study was done on dengue patients for period of 3 months January 2020 to March 2020. Confirmed cases of dengue fever with NS 1 Ag positive & Ig M antibody positive were included in the study. Patient with sepsis, underlying malignancy, autoimmune disorder, hematological disorder, drugs causing thrombocytopenia were excluded from the study. Appropriate statistical methods were applied. Results: Total 50 subjects were included. Majority of subjects were in the age group 31 to 40 years. 50% were males and females respectively. Most common clinical feature was fever and bleeding manifestations. Mean Vitamin B12 was significantly lower among those with severe thrombocytopenia and highest among those with no thrombocytopenia., there was significant difference in mean Platelet transfusion and duration of hospital stay with respect to severity of Platelet count. Conclusion: Dengue fever patients with vitamin B 12 deficiency had moderate to severe thrombocytopenia and more bleeding manifestations. Those patients required more platelet transfusion and increased duration of hospital stay.

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Ethylenediaminetetraacetic Acid‑Dependent Pseudothrombocytopenia in Patient Previously Treated as Dengue Fever

Berkala Kedokteran ◽

10.20527/jbk.v17i1.10250 ◽

2021 ◽

Vol 17 (1) ◽

pp. 65

Author(s):

Hendra Wana Nur’amin ◽

Muhammad Darwin Prenggono ◽

Wivina Riza Devi

Keyword(s):

Bone Marrow ◽

Platelet Count ◽

Dengue Fever ◽

Complete Blood Count ◽

Ethylenediaminetetraacetic Acid ◽

Bone Marrow Aspiration ◽

Normal Platelet ◽

Low Platelet Count ◽

Bleeding Manifestation ◽

Secondary Hospital

Abstract: One of the most widely used anticoagulants for a complete blood count is ethylenediaminetetraacetic acid (EDTA). Pseudothrombocytopenia (PTCP) may be caused by EDTA, this condition may lead to inappropriate diagnosis and treatment. We report a 25-year-old female with unspecific headache and joint pain with very low platelet count since 1 month before hospital admission. She was diagnosed with Dengue fever infection and got some platelet transfusion from the previous secondary hospital. She was carried out for a blood test with another anticoagulant (sodium citrate) and bone marrow aspiration. The results showed that she had normal platelet count and bone marrow cellularity. When a patient was identified with thrombocytopenia without any bleeding manifestation, hematology disease, and family history, PTCP should be taken into consideration to prevent unnecessary intervention. Keywords: platelet, pseudothrombocytopenia, ethylenediaminetetraacetic acid, Dengue fever

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Phase II Study of Eltrombopag in Subjects with Fanconi Anemia

Blood ◽

10.1182/blood-2019-123151 ◽

2019 ◽

Vol 134 (Supplement_1) ◽

pp. 3753-3753

Author(s):

Daisuke Araki ◽

Ma Evette Barranta ◽

Fariba Chinian ◽

Julie Erb-Alvarez ◽

Thomas Winkler ◽

...

Keyword(s):

Phase Ii ◽

Fanconi Anemia ◽

Platelet Transfusion ◽

Phase Ii Study ◽

Bone Marrow Failure ◽

Genetic Disorder ◽

Study Entry ◽

Extension Phase ◽

Therapeutic Modality ◽

Hematopoietic Stem

Background. Fanconi anemia (FA) is a rare genetic disorder that often presents with progressive bone marrow failure (BMF) due to an impaired DNA damage response and chronic exposure to elevated levels of proinflammatory cytokines. To date, hematopoietic stem/progenitor cell (HSPC) transplantation remains the only curative treatment for FA-associated BMF. However, donor availability, graft failure, and FA-specific transplant toxicities remain significant hurdles. Androgens have been successfully used but side effects often prevent prolonged therapy. Attempts at genetic correction of FA are underway but clinical efficacy has not yet been demonstrated. In this clinical trial, we investigate whether eltrombopag (EPAG), an FDA-approved mimetic of thrombopoietin that promotes trilineage hematopoiesis in subjects with acquired BMF (Olnes, NEJM 2012; Townsley, NEJM 2017), may offer a novel therapeutic modality for subjects with FA. Our pre-clinical studies indicate that EPAG evades blockade of signal transduction from c-MPL induced by inflammatory cytokines (Alvarado, Blood 2019). Additionally, we found that EPAG enhances DNA repair activity in human HSPCs (Guenther, Exp Hematol 2019). Thus, EPAG may positively influence two of the main known mechanisms leading to BMF in FA. Study Design. This is a non-randomized, phase II study of EPAG given to subjects with FA (NCT03204188). Subjects receive EPAG for 6 months at an oral daily dose adjusted for age and ethnicity. Subjects who cannot tolerate the medication or fail to respond by 6 months are taken off study drug. Subjects who respond at 6 months are invited in the extension phase for an additional 3 years. They continue on the same dose of EPAG until they reach robust count criteria (platelets > 50K/μL, hemoglobin (Hgb) > 10 g/dL in the absence of transfusions, and absolute neutrophil count (ANC) > 1K/uL for > 8 weeks) or until they reach steady state response (defined as stable counts for 6 months). Drug dose is tapered slowly to the lowest dose that maintains a stable platelet count and eventually discontinued until they meet off study criteria or the study is closed. Eligibility Assessment. Inclusion criteria: (1) Confirmed diagnosis of FA by a biallelic mutation in a known FANC gene and/or by positive chromosome breakage analysis in lymphocytes and/or skin fibroblasts; (2) One or more of the following cytopenias: platelets ≤ 30K/μL or platelet transfusion dependence in the 8 weeks prior to study entry, ANC ≤ 500/μL, Hgb ≤ 9.0 g/dL or red blood cell (RBC) transfusion dependence in the 8 weeks prior to study entry; (3) Failed or declined treatment with androgens; 4) Age > 4 years. Exclusion criteria: (1) Evidence of MDS or AML; (2) Cytogenetic abnormalities associated with poor prognosis in FA; (3) Known biallelic mutations in BRCA2; (4) Active malignancy or likelihood of recurrence of malignancies within 12 months; (5) Treatment with androgens ≤ 4 weeks prior to initiating EPAG. Primary Endpoints. The primary efficacy endpoint is the proportion of drug responders at 6 months. Response to EPAG is defined by one or more of the following criteria: (1) Platelets increase by 20K/μL above baseline, or platelet transfusion independence; (2) Hgb increase by > 1.5g/dL or a reduction in the units of RBC transfusions by at least 50%; (3) At least a 100% increase in ANC for subjects with a pretreatment ANC of < 0.5 x 109/L, or an ANC increase > 0.5 x 109/L. The primary safety endpoint is the toxicity profile assessed at 6 months using the CTCAE criteria. Sample Size and Statistical Methods. Simon's Two-Stage Minimax Design is used, with a response probability of ≤ 20% to terminate the treatment. In the first stage, 12 subjects will be accrued. The study will be stopped if no more than 2 subjects respond to the treatment within 6 months. If 3 or more subjects respond within 6 months in the first stage, then an additional 13 subjects will be accrued, for a total of 25 subjects. Enrollment. Two subjects have been enrolled to date. No drug-related adverse events have been observed. Subject #1 (7YO female) did not respond to 6 months of EPAG, likely due to limited HSPC reserve in the context of profound cytopenias (ANC = 100/µL, Hgb = 6g/dL, Plt = 0K/µL). In contrast, subject #2 (49YO female) showed response to EPAG at 3 months and will continue on the extension phase of the study. Conclusion. This study will provide important clinical information on safety and efficacy of EPAG in subjects with FA. Disclosures Winkler: Agios: Employment.

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A randomized, placebo-controlled trial of intravenous gammaglobulin in alloimmunized thrombocytopenic patients

Blood ◽

10.1182/blood.v75.1.313.313 ◽

1990 ◽

Vol 75 (1) ◽

pp. 313-316 ◽

Cited By ~ 45

Author(s):

T Kickler ◽

HG Braine ◽

S Piantadosi ◽

PM Ness ◽

JH Herman ◽

...

Keyword(s):

Platelet Transfusion ◽

Controlled Trial ◽

Parameter Estimate ◽

Control Group ◽

Platelet Recovery ◽

Mean Values ◽

Blinded Study ◽

Before And After ◽

Study Population ◽

Intravenous Gammaglobulin

Abstract In a placebo-controlled, randomized blinded study, we evaluated the efficacy of intravenous gammaglobulin (IV-IgG) in alloimmunized thrombocytopenic patients. IV-IgG was administered at a dose of 400 mg/kg for 5 days. An incompatible platelet transfusion from the same donor was used before and after treatment. Seven patients received IV- IgG and five patients received placebo. Although platelet recovery in 1 to 6 hours was satisfactory in five patients after IV-IgG treatment, 24- hour survival was not improved in most patients. None of the patients receiving the placebo achieved satisfactory 1-hour platelet-corrected count increments (CCIs). By t test, the posttreatment mean values 1 hour after transfusion CCIs in the IV-IgG group were significantly greater than in the control group (8,413 v 1,050, P less than .007). Using a regression model to adjust for any distributional assumptions of the study population, the parameter estimate for IV-IgG treatment was positive, indicating that IV-IgG treatment is associated with higher CCIs. Although IV-IgG may improve 1-hour platelet recovery, clinical benefit was not demonstrated since 24-hour survival was not improved. IV-IgG treatment before unmatched platelet transfusions should not be considered as a replacement for HLA-compatible platelets in alloimmunized patients.

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How well do platelets prevent bleeding?

Hematology ◽

10.1182/hematology.2020000136 ◽

2020 ◽

Vol 2020 (1) ◽

pp. 518-522

Author(s):

Darrell J. Triulzi

Keyword(s):

Platelet Transfusion ◽

Anticoagulation Therapy ◽

Outpatient Setting ◽

Severe Thrombocytopenia ◽

Transfusion Threshold ◽

Spontaneous Bleeding ◽

Risk Of Bleeding ◽

Increased Risk ◽

High Incidence ◽

Platelet Transfusions

Abstract Prophylactic platelet transfusions are used to reduce the risk of spontaneous bleeding in patients with treatment- or disease-related severe thrombocytopenia. A prophylactic platelet-transfusion threshold of <10 × 103/µL has been shown to be safe in stable hematology/oncology patients. A higher threshold and/or larger or more frequent platelet doses may be appropriate for patients with clinical features associated with an increased risk of bleeding such as high fevers, sepsis, disseminated intravascular coagulation, anticoagulation therapy, or splenomegaly. Unique factors in the outpatient setting may support the use of a higher platelet-transfusion threshold and/or dose of platelets. A prophylactic platelet-transfusion strategy has been shown to be associated with a lower risk of bleeding compared with no prophylaxis in adult patients receiving chemotherapy but not for autologous transplant recipients. Despite the use of prophylactic platelet transfusions, a high incidence (50% to 70%) of spontaneous bleeding remains. Using a higher threshold or larger doses of platelets does not change this risk. New approaches to reduce the risk of spontaneous bleeding, including antifibrinolytic therapy, are currently under study.

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Lusutrombopag is effective and safe in patients with chronic liver disease and severe thrombocytopenia: a multicenter retrospective study

BMC Gastroenterology ◽

10.1186/s12876-020-01573-9 ◽

2020 ◽

Vol 20 (1) ◽

Author(s):

Hiroaki Nomoto ◽

Naoki Morimoto ◽

Kouichi Miura ◽

Shunji Watanabe ◽

Yoshinari Takaoka ◽

...

Keyword(s):

Platelet Count ◽

Liver Disease ◽

Chronic Liver Disease ◽

Platelet Transfusion ◽

Chronic Liver ◽

Severe Thrombocytopenia ◽

Invasive Procedures ◽

Significance Level ◽

Group A ◽

Group B

Abstract Background Chronic liver disease (CLD) is often complicated by severe thrombocytopenia (platelet count < 50,000/µL). Platelet transfusion has been a gold standard for increasing the platelet count to prevent hemorrhagic events in such patients. Lusutrombopag, a thrombopoietin receptor agonist, can increase the platelet count in such patients when invasive procedures are scheduled. Former studies on lusutrombopag included patients with a platelet count of > 50,000/µL at baseline: the proportions of patients who did not require platelet transfusion were 84–96%, which might be overestimated. Methods The efficacy and safety of lusutrombopag were retrospectively investigated in CLD patients with platelet count of < 50,000/µL, a criterion for platelet transfusion, in real-world settings. We examined the proportion of patients who did not require platelet transfusion in 31 CLD patients, which exceeded a minimum required sample size (21 patients) calculated by 80% power at a significance level of 5%. Lusutrombopag, 3 mg once daily, was administered 8–18 days before scheduled invasive procedures. Results Among 31 patients who received lusutrombopag, 23 patients (74.2%) patients showed a platelet count of ≥ 50,000/µL (Group A) and did not require platelet transfusion. The remaining 8 patients (25.8%) did not reached platelet ≥ 50,000/µL (Group B). The means of platelet increase were 38,000/µL and 12,000/µL in groups A and B, respectively. A low platelet count at baseline was a characteristic of patients in group B. Among 13 patients who repeatedly used lusutrombopag, lusutrombopag significantly increased the platelet count as the initial treatment. When all repeated uses of lusutrombopag were counted among these 13 patients, platelet transfusion was not required in 82.1% (23/28) of treatments. Although one patient showed portal thrombosis after lusutrombopag treatment, the thrombosis was disappeared by anticoagulant treatment for 35 days. The degree of platelet increase with lusutrombopag was larger than that in their previous platelet transfusion. Conclusions The proportion of patients who did not require platelet transfusion was 74.2%, which is smaller than that in former studies which included CLD patients with a platelet count of > 50,000/µL. However, lusutrombopag is effective and safe for CLD patients with a platelet count of < 50,000/µL.

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Anesthetic Management of a Patient With Fanconi Anemia

Anesthesia Progress ◽

10.2344/anpr-66-02-06 ◽

2019 ◽

Vol 66 (4) ◽

pp. 218-220

Author(s):

Saki Nagano ◽

Masanori Tsukamoto ◽

Takeshi Yokoyama

Keyword(s):

Fanconi Anemia ◽

Platelet Transfusion ◽

Autosomal Recessive ◽

Dental Treatment ◽

Bone Marrow Failure ◽

Anesthetic Management ◽

Bone Marrow Failure Syndrome ◽

Hematopoietic Stem ◽

Dental Surgery ◽

Preoperative Examination

Fanconi anemia (FA) is a type of bone marrow failure syndrome based on an autosomal recessive inherited trait with increased predisposition for other cancers. It is extremely rare and is characterized by short stature, polydactyly, and pancytopenia. At present, the only effective treatment for FA is allogeneic hematopoietic stem cell transplantation (SCT). Chemotherapy is necessary prior to allogeneic SCT. Dental treatment is usually performed before chemotherapy to reduce potential infections. We experienced the anesthetic management of a 4-year-old boy diagnosed with FA, who underwent extensive dental extractions before chemotherapy for SCT. In the preoperative examination, the platelet count was decreased to less than 3.0 × 104 cells/μL because of chronic pancytopenia. The patient received 20 units of platelet transfusion over 3 days prior to anesthesia. Dental surgery and multiple dental extractions were successfully completed under general anesthesia with sevoflurane, fentanyl, and remifentanil, and chemotherapy started 3 days postoperatively.

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