Effective treatment of bone marrow diseases: A Synthesis of Findings from 20 Studies

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Original Abstract of the Article

Major research findings

Shwachman-Diamond syndrome (SDS) is a genetic disorder characterized by low levels of neutrophils, a type of white blood cell that fights infection. 5 showed that ataluren, a drug that can help cells produce a full-length protein, can improve the production of neutrophils and their ability to move towards infection in people with SDS. 13 found that people with SDS are more likely to develop myeloid malignancies, such as leukemia, which is a cancer of the blood-forming cells in the bone marrow. 16 reviewed current practices and future research directions in the diagnosis and treatment of inherited bone marrow failure syndromes (IBMFS), a group of rare genetic disorders that include SDS. 18 provided an overview of the pathophysiology and treatment of bone marrow failure. 19 established a mouse model of bone marrow suppression induced by cytarabine (Ara-C), a drug used to treat leukemia. 15 investigated a new approach to treat acute myeloid leukemia (AML) using a nanocomplex that targets the bone marrow. 7 described the clinical features and treatment strategy of degenerative atlantoaxial subluxation (DAAS), a condition that can cause spinal cord compression. provided a review of therapeutic options for acquired amegakaryocytic thrombocytopenic purpura (AATP), a rare bone marrow disorder characterized by low platelet counts. 1 described the Australian Aplastic Anaemia and other Bone Marrow Failure Syndromes Registry, which aims to improve outcomes for patients with BMFS. 20 reviewed short telomere syndrome (STS), a rare group of diseases caused by defects in telomere-maintenance genes. 17 showed that combined subchondral and intra-articular injections of bone marrow aspirate concentrate (BMAC) provided stable results for up to 24 months in patients with knee osteoarthritis. 11 reviewed the current research on bone marrow edema syndrome (BMES), a relatively uncommon condition. 2 discussed post-transplant complications in patients with IBMFS. 12 investigated the role of apoptosis of hematopoietic stem cells in bone marrow suppression following CAR-T therapy. 14 evaluated lung toxicity with lenalidomide using a pharmacovigilance database. 3 discussed the metabolic basis of inherited neutropenias. 8 reported a case of irreversible intrathecal chemotherapy-induced myelopathy in a patient with diffuse large B-cell lymphoma.

Treatment summary

5 showed that ataluren, a drug that can help cells produce a full-length protein, could improve neutrophil production and function in people with SDS. 16 noted that hematopoietic stem cell transplantation is an effective treatment for some bone marrow failure syndromes. 18 discussed hematopoietic stem cell transplantation and immunosuppressive therapy as treatment options for bone marrow failure. described the use of immunosuppressive therapy and platelet transfusions in the treatment of acquired amegakaryocytic thrombocytopenic purpura (AATP). 17 showed that combined subchondral and intra-articular injections of bone marrow aspirate concentrate (BMAC) provided stable results for up to 24 months in patients with knee osteoarthritis. 11 reviewed treatment options for bone marrow edema syndrome, which include symptomatic treatment, extracorporeal shock waves therapy, pulsed electromagnetic fields, hyperbaric oxygen, vitamin D, iloprost, bisphosphonates, denosumab, and surgery.

Benefits and risks

Benefit summary

These studies suggest that drugs like ataluren, hematopoietic stem cell transplantation, and immunosuppressive therapies can be effective treatments for bone marrow diseases. 5 showed that ataluren could improve neutrophil production and function in people with SDS. 16 discussed the effectiveness of hematopoietic stem cell transplantation for some bone marrow failure syndromes. 18 described the potential benefits of hematopoietic stem cell transplantation and immunosuppressive therapy for bone marrow failure. 17 showed that combined subchondral and intra-articular injections of bone marrow aspirate concentrate (BMAC) provided stable results for up to 24 months in patients with knee osteoarthritis.

Risk summary

These studies also suggest that treatments for bone marrow diseases can have side effects. 5 did not discuss any side effects associated with ataluren. 16 noted the risks of graft-versus-host disease (GVHD) and infections following hematopoietic stem cell transplantation. 18 discussed the increased risk of infections associated with immunosuppressive therapy. 17 indicated that combined subchondral and intra-articular injections of bone marrow aspirate concentrate (BMAC) can have a failure rate and may lead to complications.

Comparison between studies

Commonalities

These studies all agree that bone marrow diseases involve problems with the production and/or function of blood-forming cells in the bone marrow. They also indicate that hematopoietic stem cell transplantation and/or immunosuppressive therapies have the potential to be effective treatments for these diseases.

Differences

These studies focused on different bone marrow diseases and treatment approaches. 5 investigated the potential of ataluren to treat SDS. 16 reviewed the use of hematopoietic stem cell transplantation for bone marrow failure syndromes. 18 provided a general overview of bone marrow failure and treatment options, including hematopoietic stem cell transplantation and immunosuppressive therapies. 17 explored the effectiveness of combined subchondral and intra-articular injections of BMAC for knee osteoarthritis.

Consistency and contradiction of the results

These studies support the idea that hematopoietic stem cell transplantation and immunosuppressive therapies can be effective treatments for some bone marrow diseases. However, it's important to remember that each study focuses on specific diseases and treatment methods, so direct comparison of results can be challenging.

Considerations for real-life application

While research indicates the potential benefits of these treatments for bone marrow diseases, it's crucial to remember that they also carry risks. For example, hematopoietic stem cell transplantation can lead to GVHD and infections, while immunosuppressive therapies can increase the risk of infections. It's important to discuss these risks and benefits with your doctor before making decisions about treatment.

Limitations of current research

The number of patients included in some of these studies was relatively small, which limits the generalizability of the findings. Additionally, many of these studies did not assess the long-term effects of treatment, so more research is needed to understand the long-term outcomes of these therapies.

Future research directions

Further research is needed to develop new and improved treatments for bone marrow diseases. This includes developing new therapies, reducing side effects of existing treatments, and evaluating the long-term effectiveness of treatments.

Conclusion

These studies highlight the potential of treatments like hematopoietic stem cell transplantation and immunosuppressive therapies for addressing bone marrow diseases. While promising, it's crucial to be aware of their limitations and potential risks. Ongoing research is essential for developing safer and more effective treatments for these complex diseases.

Treatment list

Ataluren, hematopoietic stem cell transplantation, immunosuppressive therapy, extracorporeal shock waves therapy, pulsed electromagnetic fields, hyperbaric oxygen, vitamin D, iloprost, bisphosphonates, denosumab, surgery

Literature analysis of 20 papers
Positive Content
17
Neutral Content
0
Negative Content
3
Article Type
0
1
2
8
19
1.

The Australian Aplastic Anaemia and other Bone Marrow Failure Syndromes Registry.

Author: FoxLucy C, McQuiltenZoe K, FirkinFrank, FoxVanessa, BadouxXavier, BajelAshish, BarbaroPasquale, Cole-SinclairMerrole F, ForsythCecily, GibsonJohn, HiwaseDevendra K, JohnstonAnna, MillsAnthony, RoncolatoFernando, SutherlandRobyn, SzerJeff, TingStephen B, VilcassimShahla, YoungLauren, WatersNeil A, WoodErica M,

Improved treatment strategies

This study describes the Australian Aplastic Anaemia and other Bone Marrow Failure Syndromes Registry (AAR) and its aim to improve outcomes for patients with BMFS in Australia.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

3.

The metabolic basis of inherited neutropenias.

Author: OyarbideUsua, CraneGenevieve M, CoreySeth J

Neutropenia treatment

Neutrophils, the shortest-lived blood cells, require extensive proliferation and differentiation to maintain adequate numbers for rapid responses to infections. This review explores the role of metabolic pathways in neutrophil production and how disturbances in these pathways can lead to neutropenia. Understanding these metabolic vulnerabilities could pave the way for new treatments for neutropenias.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

4.

Efficacy and safety of immunosuppressive agents for adults with lupus nephritis: a systematic review and network meta-analysis.

Author: JiangNan, JinShangyi, YuChen, ZhaoJiuliang, WangQian, TianXinping, LiMengtao, ZengXiaofeng

Improved complete remission
Lupus nephritis remission
Reduced SLEDAI score
All-cause mortality
Cancer
ESRD
Herpes zoster
Infection
Myelosuppression
Ovarian failure
Relapse

This network meta-analysis compared the efficacy and safety of various immunosuppressive regimens for lupus nephritis (LN). The combination of tacrolimus (TAC), mycophenolate mofetil (MMF), and glucocorticoid (GC) was the best regimen for improving the total remission rate and SLEDAI score. The choice of regimen for specific outcomes should be carefully considered for high-risk patients.

Meta-AnalysisA statistical technique that integrates the results of multiple studies to draw more general conclusions about a particular issue.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

5.

Ataluren improves myelopoiesis and neutrophil chemotaxis by restoring ribosome biogenesis and reducing p53 levels in Shwachman-Diamond syndrome cells.

Author: CipolliMarco, BoniChristian, PenzoMarianna, VillaIsabella, BolampertiSimona, BaldisseriElena, FrattiniAnnalisa, PortaGiovanni, ApiMartina, SelicatoNora, RocciaPamela, PollutriDaniela, Marinelli BusilacchiElena, PoloniAntonella, CaporelliNicole, D'AmicoGiovanna, PegoraroAnna, CesaroSimone, OyarbideUsua, VellaAntonio, LippiGiuseppe, CoreySeth J, ValliRoberto, PoliniAlessandro, BezzerriValentino

SDS treatment

This study investigated the potential of ataluren, a translational readthrough-inducing drug, for treating Shwachman-Diamond syndrome (SDS) caused by a nonsense mutation in the SBDS gene. Ataluren restored full-length SBDS protein synthesis, improved 80S ribosome assembly, and restored myelopoiesis in vitro and ex vivo. These findings strengthen the rationale for a clinical trial of ataluren in SDS patients with the nonsense mutation.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Language : English

6.

Clinical features, epidemiology, and treatment of Shwachman-Diamond syndrome: a systematic review.

Author: HanXue, LuShuanglong, GuChangjuan, BianZhuli, XieXiaotian, QiaoXiaohong

SDS information
SDS

Shwachman-Diamond syndrome (SDS) is a rare autosomal recessive disorder characterized by inherited bone marrow failure, exocrine pancreatic dysfunction, and diverse clinical phenotypes. This study reviews international reports on SDS patients to summarize the clinical features, epidemiology, and treatment of this complex disorder.

Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

8.

Irreversible intrathecal chemotherapy-induced myelopathy in a patient with diffuse large B-cell lymphoma: A case report.

Author: HirakawaYuri, KitaoAkihito, WatanabeMarika, MatsumotoSakuya, KomakiRyohei, SakaiRina, MorimotoKohei, YakushijinKimikazu, MinamiHironobu

Lymphoma treatment
Myelopathy risk

A case of intrathecal chemotherapy-induced myelopathy is reported. Although rare, this complication can be irreversible.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Language : English

9.

Bone marrow edema of the hip: a narrative review.

Author: Di MartinoAlberto, BrunelloMatteo, VillariEleonora, CataldiPiergiorgio, D'AgostinoClaudio, FaldiniCesare

BME management
Hip pain relief
AVNH risk
Hip arthritis risk

Bone marrow edema (BME) of the hip can be primary or secondary. MRI is the gold standard for diagnosis. Treatment depends on the type of BME, ranging from conservative measures to surgery.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

10.

Acquired Amegakaryocytic Thrombocytopenic Purpura: A Review of Therapeutic Options.

Author: HussainSyed A, ZafarAneeqa, FaisalHafsa, Ud DinMohammad A

AATP thrombocytopenia

Acquired amegakaryocytic thrombocytopenic purpura (AATP) is a rare bone marrow disorder characterized by a decrease or absence of megakaryocytes. This review provides a comprehensive overview of current therapeutic options for AATP, which is based on case studies and expert opinions due to the rarity of the disease.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

11.

Research Advances in the Treatment of Bone Marrow Edema Syndrome.

Author: LiShihong, YuHuimin, LongShiwei, LiJian, HeYang, ZhengXiaolong, YangShuang, TangYingchao, XieQingyun, ZhengWei

Improved quality of life

Bone marrow edema syndrome (BMES) is an uncommon condition with limited reporting in the literature. This paper reviews the treatment options for BMES, including symptomatic treatment, extracorporeal shock waves therapy, pulsed electromagnetic fields, hyperbaric oxygen, vitamin D, iloprost, bisphosphonates, denosumab, and surgery. The aim is to inform clinicians about these options and improve patients' quality of life and disease duration.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

12.

Apoptosis of Hematopoietic Stem Cells Contributes to Bone Marrow Suppression Following Chimeric Antigen Receptor T Cell Therapy.

Author: ReadJay A, RouceRayne H, MoFeiyan, MamonkinMaksim, KingKatherine Y

Effective cancer treatment
Cytokine release syndrome
Delayed bone marrow recovery

CAR-T therapy can lead to cytokine release syndrome (CRS), which causes delayed bone marrow recovery. CRS increases levels of proinflammatory cytokines, leading to apoptosis and depletion of hematopoietic stem and progenitor cells (HSPCs).

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.
Research Support, N.I.H., ExtramuralResearch funding provided by the NIH to external research institutions and researchers.

Language : English

14.

Evaluation of Lung Toxicity With Lenalidomide Using the Pharmacovigilance Database.

Author: SatoJunya, ErenTsuchiya, MurataSaeko, ShimizuTadashi, UchidaMayako

LND lung toxicity

This study utilizes the Japanese Adverse Drug Event Report (JADER) database to investigate the incidence and onset timing of lenalidomide (LND)-related lung toxicity, analyzing its occurrence and outcomes.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Language : English

16.

Inherited bone marrow failure syndromes: a review of current practices and potential future research directions.

Author: DengJoseph, McReynoldsLisa J

Disease Understanding
Patient Outcomes

This review provides an overview of the current state of diagnosis and treatment for inherited bone marrow failure syndromes (IBMFS) in children and identifies areas for future research.

ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, N.I.H., IntramuralFunding provided by the NIH for research projects within the NIH.

Language : English

18.

Aplastic Anemia as a Roadmap for Bone Marrow Failure: An Overview and a Clinical Workflow.

Author: SolimandoAntonio G, PalumboCarmen, PragnellMary Victoria, BittrichMax, ArgentieroAntonella, KrebsMarkus

Improved BM Failure Diagnosis
Improved Myeloid Malignancy Diagnosis

This study reviews the complex interplay of factors involved in bone marrow (BM) failures and myeloid malignancy predisposition syndromes. The study highlights the importance of accurate diagnosis and explores potential new theragnostic windows for treating these diseases.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

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