Generic formulations of imatinib for treatment of Philadelphia chromosome–positive leukemia in pediatric patients

Suttorp, Meinolf; Metzler, Markus; Millot, Frédéric; Shimada, Hideaki; Bansal, Deepak; Güneş, Adalet Meral; Kałwak, Krzysztof; Sedláček, Petr; Baruchel, André; Biondi, Andrea; Hijiya, Nobuko; Schultz, Kirk R.; Schrappe, Martin

doi:10.1002/pbc.27431

Cited by 12 publications

(6 citation statements)

References 27 publications

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“…These restrictions may be challenging for young children and teenagers, depending on lifestyle. Generic imatinib is much less expensive than the branded product 32 and 2G-TKIs cost significantly more than imatinib. 33 In randomized studies in adults, 2G-TKIs induced a significantly faster and deeper molecular remission than imatinib, although no survival advantage was observed.…”

Section: Casementioning

confidence: 99%

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How I treat chronic myeloid leukemia in children and adolescents

Hijiya

Suttorp

2019

Blood

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112

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Evidence-based recommendations have been established for treatment of chronic myeloid leukemia (CML) in adults treated with tyrosine kinase inhibitors (TKIs), but the rarity of this leukemia in children and adolescents makes it challenging to develop similar recommendations in pediatrics. In addition to imatinib, which was approved for pediatric CML in 2003, the second-generation TKIs dasatinib and nilotinib were recently approved for use in children, expanding the therapeutic options and pushing allogeneic stem cell transplantation to a third-line treatment of most pediatric cases. Yet, without sufficient data on efficacy and safety specific to pediatric patients, the selection of a TKI continues to rely on clinical experience in adults. Here, we present 4 case scenarios highlighting common yet challenging issues encountered in the treatment of pediatric CML (suboptimal response, poor treatment adherence, growth retardation, and presentation in advanced phases). Limited experience with very young children, the transition of teenagers to adult medicine, and the goal of achieving treatment-free remission for this rare leukemia are additional significant obstacles that require further clinical investigation through international collaboration.

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Section: Casementioning

confidence: 99%

“…The decision to stop TKI treatment may also be influenced by the financial burden of these expensive drugs; this issue may vary among different families and countries depending on the insurance system and the availability and price of imatinib generics. 32,61,62 Case 3…”

Section: Optimal Responsementioning

confidence: 99%

How I treat chronic myeloid leukemia in children and adolescents

Hijiya

Suttorp

2019

Blood

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112

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“…3 As observed with generic imatinib, not all generic products are substandard. 4 This then behoves us to identify products suitable for therapeutic use among the variety of generic options in the market. We believe three approaches allow us to address this challenge.…”

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confidence: 99%

Reply to: Comment on: Unsatisfactory quality of E. coli asparaginase biogenerics in India—Implications for clinical outcomes in acute lymphoblastic leukaemia

Sidhu

Saha

Krishnan

2021

Pediatric Blood & Cancer

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We thank Drs. Cecconello and Michalowski for their comments.We acknowledge the exceptional efforts of Brazil's physicians and scientists to identify and address the problem of inferior quality asparaginase in their country. 1 We share their concern that the asparaginase experience is potentially representative of a wider global problem of access to quality-assured generic cancer medicines. 2 This concern must be balanced against the requirement of generic products and biosimilars to meet the need for off-patent essential medicines at affordable cost. 3 As observed with generic imatinib, not all generic products are substandard. 4 This then behoves us to identify products suitable for therapeutic use among the variety of generic options in the market. We believe three approaches allow us to address this challenge. The first approach is to establish collaborative multi-institutional clinical groups to standardise disease management and evaluate treatment strategies systematically. 5 As shown by Dr.Cecconello and colleagues, 1 this enables us to begin addressing unmet needs in access to cancer medicines that are safe, suitably tolerated, effective, available in child-friendly formulations and at affordable cost. The second approach is to develop partnerships with pharmaceutical companies to evaluate pharma products, including conducting clinical studies to monitor drug safety and efficacy. This requires companies to recognise the long-term advantages of such a partnership, which includes developing the evidence base for product use in clinical practice and identifying opportunities for innovation. 6 The third approach is to engage patient advocacy groups to raise awareness and support for clinical studies that evaluate generic medicines and to identify generic products suitable for therapeutic use. This latter approach has received a fillip with launch of the World Health Organisation's Global Initiative for Childhood Cancer in 2018. 7 Collectively, we believe these approaches bear the promise of ensuring access to high-quality generic medicines for paediatric cancers.

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“…From a global viewpoint, children with CML and their families face major inequity between countries in terms of access to innovative drugs[ 65 ]. New drug development pathways could efficiently address the challenges with innovative methods and trial designs, investment in biology and preclinical research, new models of partnership and funding including public-private partnerships and precompetitive research consortia.…”

Section: Future Tasks and Outlookmentioning

confidence: 99%

Horn of plenty: Value of the international registry for pediatric chronic myeloid leukemia

Suttorp

Metzler

Millot

2020

WJCO

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Chronic myeloid leukemia (CML) in minors is a rare disease which can be effectively treated by tyrosine kinase inhibitors (TKIs) since the year 2000. A majority of pediatricians will encounter one or two CML patients in the course of their careers and will typically have to rely on written information along with their own intuition to provide care. Knowledge of response to TKIs and of age-specific side effects has an impact on the design of pediatric CML trials in many ways aiming to contribute toward greater predictability of clinical improvements. Information from a registry on a rare disease like CML offers the enormous benefit of enabling treating physicians to interact and share their collective experience. The International Registry on Pediatric CML (IR-PCML) was founded at Poitiers/France almost 10 years ago. Since then, the number of collaboration centers and in parallel of registered patients continuously increased (> 550 patients as of December 2019). Ideally, from a given treatment center in a country data are transferred to a national coordinator who interacts with the IR-PCML. In the sense of quality assurance, the registry can offer dissemination of knowledge on state-of-the-art diagnostics (including reference appraisal), optimal treatment approaches, and follow-up procedures within a network that is exerting its strength via participation. With continuous growth during the recent years, very rare subgroups of patients could be identified ( e.g ., CML diagnosed at age < 3 years, children presenting with specific problems at diagnosis or during course of treatment) which had not been described before. Publications coming from the IR-PCML disseminated this useful information derived from patients who robustly participate and share information about their disease, among themselves and with their caregivers and clinicians. Patient input driving the collection of data on this rare leukemia is the basis for the considerable success of bringing new therapeutics into clinical use.

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Generic formulations of imatinib for treatment of Philadelphia chromosome–positive leukemia in pediatric patients

Cited by 12 publications

References 27 publications

How I treat chronic myeloid leukemia in children and adolescents

How I treat chronic myeloid leukemia in children and adolescents

Reply to: Comment on: Unsatisfactory quality of E. coli asparaginase biogenerics in India—Implications for clinical outcomes in acute lymphoblastic leukaemia

Horn of plenty: Value of the international registry for pediatric chronic myeloid leukemia

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